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Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02489344
Recruitment Status : Completed
First Posted : July 3, 2015
Results First Posted : December 9, 2019
Last Update Posted : March 23, 2021
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Drug: GZ/SAR402671
Enrollment 8
Recruitment Details Participants who successfully completed 26 weeks of treatment in prior ACT13739 study (NCT02228460) were eligible to continue their treatment for up to 30 additional months in this extension study LTS14116. Eleven participants had enrolled and were treated in ACT13739 study, and 9 completed study. Of these 9 participants, 8 entered extension study.
Pre-assignment Details In extension study, participants continued on same dose regimen they had received in initial study. Two participants in LTS14116 completed treatment and did not discontinue early, but are counted as not completed study due to no record of completion.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Period Title: ACT13739 (Initial Study): 26 Weeks
Started [1] 11
Completed 9
Not Completed 2
Reason Not Completed
Adverse Event             1
Lost to Follow-up             1
[1]
Enrolled and treated.
Period Title: LTS14116 (Extension Study): 31 Months
Started [1] 8
Completed [2] 5
Not Completed 3
Reason Not Completed
Adverse Event             1
Treatment completed/Study not completed             2
[1]
Enrolled and treated.
[2]
2 participant completed,not discontinued early,but counted not completed due to no completion record
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
Analysis performed on all participants who were initially enrolled in the ACT13739 study.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants
26.5  (7.6)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Female
0
   0.0%
Male
11
 100.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 11 participants
Caucasian/White
8
  72.7%
Other
3
  27.3%
1.Primary Outcome
Title Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Hide Description Any untoward medical occurrence in a participant who received study drug was considered an adverse event (AE) without regard to possibility of causal relationship with this treatment. TEAEs were defined as AEs that developed or worsened during TEAE period (period from the first administration of study drug in ACT13739 through the last administration of the study drug in the combined ACT13739/LTS14116 treatment period plus 1 month or end of study participation for participant, whichever occurred first). For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of investigational medicinal product (IMP) during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
9
2.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Hematological Parameters
Hide Description

Criteria for potentially clinically significant abnormalities:

  • Hemoglobin: less than or equal to (<=) 115 grams per liter (g/L); greater than or equal to (>=)185 g/L; decreased from baseline (DFB) >=20 g/L
  • Hematocrit: <=0.37 volume/volume (v/v); >=0.55 v/v
  • Erythrocytes: >=6 Tera/L
  • Platelets: lesser than (<) 100 Giga/L; >=700 Giga/L
  • Leukocytes: <3.0 Giga/L (Non-Black [NB]) or <2.0 Giga/L (Black [B]); >=16.0 Giga/L
  • Neutrophils: <1.5 Giga/L (NB) or <1.0 Giga/L (B);
  • Lymphocytes: greater than (>) 4.0 Giga/L
  • Monocytes: >0.7 Giga/L
  • Basophils: >0.1 Giga/L
  • Eosinophils: >0.5 Giga/L or >upper limit of normal (ULN) (if ULN >=0.5 Giga/L) For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Hemoglobin <=115 g/L
1
   9.1%
Hemoglobin >=185 g/L
0
   0.0%
Hemoglobin DFB >=20 g/L
0
   0.0%
Hematocrit <=0.37 v/v
6
  54.5%
Hematocrit >0.55 v/v
0
   0.0%
Erythrocytes: >=6 Tera/L
0
   0.0%
Platelets <100 Giga/L
0
   0.0%
Platelets >=700 Giga/L
0
   0.0%
Leukocytes <3.0 Giga/L (NB) or <2.0 Giga/L (B)
0
   0.0%
Leukocytes >=16.0 Giga/L
0
   0.0%
Neutrophils <1.5 Giga/L (NB) or <1.0 Giga/L (B)
0
   0.0%
Lymphocytes >4.0 Giga/L
0
   0.0%
Monocytes >0.7 Giga/L
2
  18.2%
Basophils >0.1 Giga/L
1
   9.1%
Eosinophils >0.5 Giga/L or >ULN (ULN >=0.5 Giga/L)
0
   0.0%
3.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Electrolytes
Hide Description

Criteria for potentially clinically significant abnormalities:

  • Sodium: <=129 millimoles (mmol)/L; >=160 mmol/L
  • Potassium: <3 mmol/L; >=5.5 mmol/L
  • Chloride: <80 mmol/L; >115 mmol/L.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Sodium <=129 mmol/L
0
   0.0%
Sodium >=160 mmol/L
0
   0.0%
Potassium <3 mmol/L
0
   0.0%
Potassium >=5.5 mmol/L
0
   0.0%
Chloride <80 mmol/L
0
   0.0%
Chloride >115 mmol/L
0
   0.0%
4.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Liver Function Parameters
Hide Description

Criteria for potentially clinically significant abnormalities:

  • Alanine Aminotransferase (ALT): >3 ULN; >5 ULN; >10 ULN and >20 ULN
  • Aspartate aminotransferase (AST): >3 ULN; >5 ULN; >10 ULN and >20 ULN
  • Alkaline phosphatase: >1.5 ULN
  • Bilirubin: >1.5 ULN; >2 ULN.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
ALT >3 ULN
0
   0.0%
ALT >5 ULN
0
   0.0%
ALT >10 ULN
0
   0.0%
ALT >20 ULN
0
   0.0%
AST >3 ULN
0
   0.0%
AST >5 ULN
0
   0.0%
AST >10 ULN
0
   0.0%
AST >20 ULN
0
   0.0%
Alkaline Phosphatase >1.5 ULN
0
   0.0%
Bilirubin >1.5 ULN
0
   0.0%
Bilirubin >2 ULN
0
   0.0%
5.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Metabolic Parameters
Hide Description

Criteria for potentially clinically significant abnormalities:

  • Glucose: <=3.9 mmol/L and < lower limits of normal (LLN); >=11.1 mmol/L (unfasted [unfas]) or >=7 mmol/L (fasted [fas])
  • Lipase: >= 3 ULN
  • C Reactive Protein (CRP): > 2 ULN or > 10 milligrams (mg)/L (if ULN not provided).
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Glucose <=3.9 mmol/L and <LLN
3
  27.3%
Glucose >=11.1 mmol/L (unfas) or >=7 mmol/L (fas)
1
   9.1%
Albumin <=25 g/L
0
   0.0%
Lipase >=3 ULN
0
   0.0%
CRP >2 ULN or >10 mg/L (if ULN not provided)
2
  18.2%
6.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Renal Function Parameters
Hide Description

Criteria for potentially clinically significant abnormalities:

  • Creatinine: >=150 micromoles per liter (mcmol/L) (Adults); >=30% change from baseline; >= 100% change from baseline
  • Blood urea nitrogen: >=17 mmol/L
  • Urate: <120 mcmol/L; >408 mcmol/L For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Creatinine >=150 mcmol/L
0
   0.0%
Creatinine >=30% change from baseline
2
  18.2%
Creatinine >=100% change from baseline
0
   0.0%
Blood Urea Nitrogen >=17 mmol/L
0
   0.0%
Urate <120 mcmol/L
0
   0.0%
Urate >408 mcmol/L
5
  45.5%
7.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Laboratory Abnormalities: Urinalysis
Hide Description

Criteria with potentially clinically significant urine abnormalities:

pH: <= 4.6; pH: >= 8.0

Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
pH <= 4.6
0
   0.0%
pH >= 8.0
0
   0.0%
8.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Vital Signs Abnormalities
Hide Description

Criteria for potentially clinically significant vital sign abnormalities:

  • Systolic blood pressure (SBP) supine: <=95 millimeters of mercury (mmHg) and DFB >=20 mmHg; >=160 mmHg and increase from baseline (IFB) >=20 mmHg
  • Diastolic blood pressure (DBP) supine: <=45 mmHg and DFB >=10 mmHg; >=110 mmHg and IFB >=10 mmHg
  • Heart rate (HR) supine: <=50 beats per minute (bpm) and DFB >=20 bpm; >=120 bpm and IFB >=20 bpm
  • Weight: >=5% DFB; >=5% IFB For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
SBP (supine) <=95 mmHg and DFB >=20 mmHg
0
   0.0%
SBP (supine) >=160 mmHg and IFB >=20 mmHg
0
   0.0%
DBP (supine) <=45 mmHg and DFB >=10 mmHg
0
   0.0%
DBP (supine) >=110 mmHg and IFB >=10 mmHg
0
   0.0%
HR (supine) <=50 bpm and DFB >= 20 bpm
1
   9.1%
HR (supine) >=120 bpm and IFB >=20 bpm
0
   0.0%
Weight >=5% DFB
3
  27.3%
Weight >=5% IFB
2
  18.2%
9.Primary Outcome
Title Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Abnormalities
Hide Description

Criteria for potentially clinically significant ECG abnormalities:

  • ECG mean HR: <30 bpm; <30 bpm and DFB >=20 bpm; <40 bpm; <40 bpm and DFB >=20 bpm; <50 bpm; <50 bpm and DFB >=20 bpm; >90 bpm; <90 bpm and DFB >=20 bpm; >100 bpm; <100 bpm and DFB >=20 bpm; >120 bpm; <120 bpm and DFB >=20 bpm
  • PR Interval: >200 milliseconds (ms); >200 ms and IFB >=25%; >220 ms; >220 ms and IFB >=25%; >240 ms; >240 ms and IFB >=25%
  • QRS duration: >110 ms; >110 ms and IFB >=25%; >120 ms; >120 ms and IFB >=25%
  • QTc Bazett (QTcB) interval: >450 ms; >480 ms; >500 ms; IFB >30 and <=60 ms, IFB >60 ms
  • QTc Fridericia (QTc F): >450 ms; >480 ms; >500 ms; IFB >30 and <=60 ms; IFB >60 ms
  • QT Interval: >500 ms For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
ECG Mean HR <30 bpm
0
   0.0%
ECG Mean HR <30 bpm and DFB >=20 bpm
0
   0.0%
ECG Mean HR <40 bpm
0
   0.0%
ECG Mean HR <40 bpm and DFB >=20 bpm
0
   0.0%
ECG Mean HR <50 bpm
1
   9.1%
ECG Mean HR <50 bpm and DFB >=20 bpm
0
   0.0%
ECG Mean HR >90 bpm
1
   9.1%
ECG Mean HR <90 bpm and DFB >=20 bpm
0
   0.0%
ECG Mean HR >100 bpm
0
   0.0%
ECG Mean HR <100 bpm and DFB >=20 bpm
0
   0.0%
ECG Mean HR >120 bpm
0
   0.0%
ECG Mean HR <120 bpm and DFB >=20 bpm
0
   0.0%
PR interval >200 ms
2
  18.2%
PR interval >200 ms and IFB >=25%
0
   0.0%
PR interval >220 ms
0
   0.0%
PR interval >220 ms and IFB >=25%
0
   0.0%
PR >240 ms
0
   0.0%
PR interval >240 ms and IFB >=25%
0
   0.0%
QRS duration >110 ms
1
   9.1%
QRS duration >110 ms and IFB >=25%
0
   0.0%
QRS duration >120 ms
0
   0.0%
QRS duration >120 ms and IFB >=25%
0
   0.0%
QTcB interval >450 ms
0
   0.0%
QTcB interval >480 ms
0
   0.0%
QTc B interval >500 ms
0
   0.0%
QTcB interval IFB >30 and <=60 ms
1
   9.1%
QTcB interval IFB >60 ms
0
   0.0%
QTcF interval >450 ms
0
   0.0%
QTc F interval >480 ms
0
   0.0%
QTcF interval >500 ms
0
   0.0%
QTcF interval IFB >30 and <=60 ms
0
   0.0%
QTcF interval IFB >60 ms
0
   0.0%
QT interval >500 ms
0
   0.0%
10.Secondary Outcome
Title Change From Baseline in Plasma Globotriaosylceramide (GL-3) Concentration at Weeks 26, 52, 104, and 156
Hide Description Change from baseline in plasma GL-3 was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: micrograms per milliliter (mcg/mL)
Week 26 Number Analyzed 9 participants
-3.62  (1.07)
Week 52 Number Analyzed 7 participants
-5.06  (1.04)
Week 104 Number Analyzed 7 participants
-6.32  (2.53)
Week 156 Number Analyzed 7 participants
-6.97  (2.27)
11.Secondary Outcome
Title Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso GL-3) Concentration at Weeks 26, 52, 104, and 156
Hide Description Change from baseline in plasma GL-3 was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: nanograms per mL (ng/mL)
Week 26 Number Analyzed 9 participants
-30.99  (22.83)
Week 52 Number Analyzed 7 participants
-37.10  (20.69)
Week 104 Number Analyzed 7 participants
-39.84  (18.12)
Week 156 Number Analyzed 7 participants
-48.13  (15.65)
12.Secondary Outcome
Title Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration At Weeks 26, 52, 104, and 156
Hide Description Change from baseline in plasma GL-1 was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mcg/mL
Week 26 Number Analyzed 9 participants
-3.26  (1.43)
Week 52 Number Analyzed 7 participants
-3.58  (0.85)
Week 104 Number Analyzed 7 participants
-3.70  (0.85)
Week 156 Number Analyzed 7 participants
-3.23  (1.11)
13.Secondary Outcome
Title Change From Baseline in Plasma Monosialodihexosylganglioside (GM3) Concentration At Weeks 26, 52, 104, and 156
Hide Description Change from baseline in plasma GM3 was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. Concentration of GM3 in plasma was determined using a validated LC-MS/MS method. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mcg/mL
Week 26 Number Analyzed 9 participants
-10.77  (6.02)
Week 52 Number Analyzed 7 participants
-8.84  (4.55)
Week 104 Number Analyzed 7 participants
-9.92  (3.26)
Week 156 Number Analyzed 7 participants
-8.12  (5.37)
14.Secondary Outcome
Title Change From Baseline in Urine GL-3 Concentration At Weeks 26, 52, 104, and 156
Hide Description Change from baseline in urine GL-3 was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mg/mmol creatinine (Cr)
Week 26 Number Analyzed 8 participants
-0.25  (0.19)
Week 52 Number Analyzed 6 participants
-0.20  (0.22)
Week 104 Number Analyzed 7 participants
-0.18  (0.22)
Week 156 Number Analyzed 7 participants
-0.18  (0.27)
15.Secondary Outcome
Title Change From Baseline in High Sensitivity Cardiac Troponin T At Weeks 26, 52, 104, and 156
Hide Description Change from baseline in high sensitivity cardiac troponin T was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104, and 156. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mcg/L
Week 26 Number Analyzed 9 participants
0.0000  (0.0000)
Week 52 Number Analyzed 5 participants
0.0105  (0.0241)
Week 104 Number Analyzed 7 participants
-0.0015  (0.0000)
Week 156 Number Analyzed 7 participants
0.0006  (0.0027)
16.Secondary Outcome
Title Change From Baseline in Podocyturia Counts (Per Milligram of Creatinine) At Weeks 12, 26, and 156
Hide Description Change from baseline in podocyturia was obtained by subtracting baseline value from post-baseline value at Weeks 12, 26, and 156. Urine samples were processed to identify podocyte (podocalyxin, PCX) and parietal cell (claudin 1, CL1) markers. PCX +/CL1 negative cells were identified as podocytes and PCX +/CL1 positive cells as parietal cells with podocyte phenotype. All counts were corrected for urine Cr. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 12, 26, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: Count of podocytes/mg Cr
Week 12 Number Analyzed 9 participants
-1.40  (2.69)
Week 26 Number Analyzed 7 participants
-1.65  (2.97)
Week 156 Number Analyzed 4 participants
-2.73  (3.97)
17.Secondary Outcome
Title Summary of Shifts From Baseline in Skin GL-3 Score in Superficial Capillary Endothelial Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score
Hide Description Skin biopsies were performed for the scoring of GL-3 accumulation/inclusions by light microscopy. Three independent pathologists scored GL-3 clearance by using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from baseline GL-3 score to Weeks 12, 26, 52, and 156 GL-3 score. Shift to lower score from baseline indicated less severe condition at that respective time point. Any shift category of Baseline score/Week score that was not observed (no participant had data in the category) was not reported. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score:1/Week 12 Score:1 Number Analyzed 9 participants
4
  44.4%
Baseline Score:1/Week 12 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:2/Week 12 Score:1 Number Analyzed 9 participants
3
  33.3%
Baseline Score:2/Week 12 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:1/Week 26 Score:1 Number Analyzed 9 participants
4
  44.4%
Baseline Score:1/Week 26 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:2/Week 26 Score:1 Number Analyzed 9 participants
3
  33.3%
Baseline Score:2/Week 26 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:1/Week 52 Score:1 Number Analyzed 6 participants
3
  50.0%
Baseline Score:2/Week 52 Score:1 Number Analyzed 6 participants
2
  33.3%
Baseline Score:2/Week 52 Score:2 Number Analyzed 6 participants
1
  16.7%
Baseline Score:1/Week 156 Score:0 Number Analyzed 6 participants
2
  33.3%
Baseline Sore:1/Week 156 Score:1 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:1 Number Analyzed 6 participants
3
  50.0%
18.Secondary Outcome
Title Summary of Shifts From Baseline in Skin GL-3 Score in Deep Vessels Endothelial Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score
Hide Description Skin biopsies were performed for the scoring of GL-3 accumulation/inclusions by light microscopy. Three independent pathologists scored GL-3 clearance by using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from baseline GL-3 score to Weeks 12, 26, 52, and 156 GL-3 score. Shift to lower score from baseline indicated less severe condition at that respective time point. Any shift category of Baseline score/Week score that was not observed (no participant had data in the category) was not reported. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score:1/Week 12 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:2/Week 12 Score:1 Number Analyzed 9 participants
2
  22.2%
Baseline Score:2/Week 12 Score:2 Number Analyzed 9 participants
6
  66.7%
Baseline Score:1/Week 26 Score:1 Number Analyzed 9 participants
1
  11.1%
Baseline Score:2/Week 26 Score:1 Number Analyzed 9 participants
2
  22.2%
Baseline Score:2/Week 26 Score:2 Number Analyzed 9 participants
6
  66.7%
Baseline Score:2/Week 52 Score:1 Number Analyzed 6 participants
3
  50.0%
Baseline Score:2/Week 52 Score:1.5 Number Analyzed 6 participants
1
  16.7%
Baseline Sore:2/Week 52 Score:2 Number Analyzed 6 participants
2
  33.3%
Baseline Score:1/Week 156 Score:1 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:0.5 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:1 Number Analyzed 6 participants
3
  50.0%
Baseline Score:2/Week 156 Score:2 Number Analyzed 6 participants
1
  16.7%
19.Secondary Outcome
Title Summary of Shifts From Baseline in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score
Hide Description Skin biopsies were performed for the scoring of GL-3 accumulation/inclusions by light microscopy. Three independent pathologists scored GL-3 clearance by using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from baseline GL-3 score to Weeks 12, 26, 52, and 156 GL-3 score. Shift to lower score from baseline indicated less severe condition at that respective time point. Any shift category of Baseline score/Week score that was not observed (no participant had data in the category) was not reported. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score:1.5/Week 12 Score:2 Number Analyzed 9 participants
2
  22.2%
Baseline Score:2/Week 12 Score:2 Number Analyzed 9 participants
7
  77.8%
Baseline Score:1.5/Week 26 Score:1.5 Number Analyzed 9 participants
2
  22.2%
Baseline Score:2/Week 26 Score:2 Number Analyzed 9 participants
7
  77.8%
Baseline Score:1.5/Week 52 Score:2 Number Analyzed 5 participants
1
  20.0%
Baseline Score:2/Week 52 Score:2 Number Analyzed 5 participants
4
  80.0%
Baseline Score:1.5/Week 156 Score:2 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:1 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:1.5 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:2 Number Analyzed 6 participants
3
  50.0%
20.Secondary Outcome
Title Summary of Shifts From Baseline in Skin GL-3 Score in Perineurium Cells Over Time: Number of Participants in Categories of Shift in GL-3 Score
Hide Description Skin biopsies were performed for the scoring of GL-3 accumulation/inclusions by light microscopy. Three independent pathologists scored GL-3 clearance by using an inclusion severity score of 0 (none/trace), 1 (mild), 2 (moderate), and 3 (severe), where higher score indicated more severe condition. A single score per participant per time point was derived by taking the score rated by a majority of the pathologists; if a majority score could not be derived, the median score was used. Data were summarized and reported in terms of number of participants with shift from baseline GL-3 score to Weeks 12, 26, 52, and 156 GL-3 score. Shift to lower score from baseline indicated less severe condition at that respective time point. Any shift category of Baseline score/Week score that was not observed (no participant had data in the category) was not reported. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 12, 26, 52, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score:1/Week 12 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:2/Week 12 Score:2 Number Analyzed 9 participants
8
  88.9%
Baseline Score:1/Week 26 Score:2 Number Analyzed 9 participants
1
  11.1%
Baseline Score:2/Week 26 Score:2 Number Analyzed 9 participants
8
  88.9%
Baseline Score:1/Week 52 Score:2 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 52 Score:1 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 52 Score:1.5 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 52 Score:2 Number Analyzed 6 participants
3
  50.0%
Baseline Score:1/Week 156 Score:1 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:1.5 Number Analyzed 6 participants
1
  16.7%
Baseline Score:2/Week 156 Score:2 Number Analyzed 6 participants
4
  66.7%
21.Secondary Outcome
Title Change From Baseline in Mental Component Summary and Physical Component Summary of the Short Form-36 (SF-36) Health Survey at Weeks 26, 52, 104 and 156
Hide Description The SF-36 health survey is a participant-reported survey to measure participant's health. It is a 36-item questionnaire used to measure 8 various aspects of health (vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, mental health). The score range for each of the 8 aspects was from 0 (maximum disability) to 100 (no disability), higher scores indicating good health condition. Responses on the SF-36 were also used to calculate 2 summary scores: Physical component score (PCS) and mental component score (MCS). The score range for each of these 2 summary scores was from 0 (maximum disability) to 100 (no disability), where higher score indicated less disability or good health condition. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: units on a scale
Physical Component Summary: Week 26 Number Analyzed 9 participants
8.39  (6.88)
Physical Component Summary: Week 52 Number Analyzed 7 participants
7.70  (7.76)
Physical Component Summary: Week 104 Number Analyzed 6 participants
9.72  (9.02)
Physical Component Summary: Week 156 Number Analyzed 6 participants
5.30  (11.83)
Mental Component Summary: Week 26 Number Analyzed 9 participants
-3.31  (20.34)
Mental Component Summary: Week 52 Number Analyzed 7 participants
1.69  (8.27)
Mental Component Summary: Week 104 Number Analyzed 6 participants
6.48  (8.14)
Mental Component Summary: Week 156 Number Analyzed 6 participants
2.87  (14.17)
22.Secondary Outcome
Title Gastrointestinal (GI) Symptoms: Number of Participants With Abdominal Pain at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to report the presence of abdominal pain in past 10 days (before each of the specified time points). Participants answered the question: "Do you currently suffer from abdominal (tummy) pain? [Yes/No]". For this analysis, baseline was defined as initial ACT13739 study.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 11 participants
No 5
Yes 6
Week 2 Number Analyzed 11 participants
No 6
Yes 5
Week 4 Number Analyzed 11 participants
No 7
Yes 4
Week 8 Number Analyzed 11 participants
No 8
Yes 3
Week 12 Number Analyzed 10 participants
No 8
Yes 2
Week 18 Number Analyzed 9 participants
No 7
Yes 2
Week 26 Number Analyzed 9 participants
No 6
Yes 3
Week 52 Number Analyzed 7 participants
No 5
Yes 2
Week 104 Number Analyzed 7 participants
No 5
Yes 2
Week 156 Number Analyzed 7 participants
No 5
Yes 2
23.Secondary Outcome
Title Gastrointestinal Symptoms: Abdominal Pain Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to mark the severity of the abdominal pain in past 10 days (before each of the specified time points) on a visual analogue scale (VAS). The scale ranged from 0% (no pain) to 100% (very severe), where higher score indicated more severity. For this analysis, baseline was defined as initial ACT13739 study baseline. Standard deviation (SD) can only be calculated when there are more than 1 participant with data available. Thereby, applicable fields were left blank when SD was not calculable.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a 0-100 percent scale
Baseline Number Analyzed 6 participants
52.50  (20.50)
Week 2 Number Analyzed 5 participants
35.60  (18.47)
Week 4 Number Analyzed 4 participants
29.75  (20.07)
Week 8 Number Analyzed 3 participants
29.00  (21.28)
Week 12 Number Analyzed 2 participants
40.00  (21.21)
Week 18 Number Analyzed 2 participants
43.00  (29.70)
Week 26 Number Analyzed 3 participants
31.33  (32.81)
Week 52 Number Analyzed 1 participants
21.00
Week 104 Number Analyzed 1 participants
21.00
Week 156 Number Analyzed 2 participants
15.00  (7.07)
24.Secondary Outcome
Title Gastrointestinal Symptoms: Number of Days With Abdominal Pain Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to report the number of days they had abdominal pain in past 10 days (before each of the specified time points). Number of days with abdominal pain score was achieved by multiplying number of days with pain * 10. The score ranges from 10 to 100, where higher score signifies more number of days with pain. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a scale
Baseline Number Analyzed 6 participants
38.33  (31.89)
Week 2 Number Analyzed 5 participants
26.00  (20.74)
Week 4 Number Analyzed 4 participants
25.00  (20.82)
Week 8 Number Analyzed 4 participants
25.00  (23.80)
Week 12 Number Analyzed 2 participants
35.00  (7.07)
Week 18 Number Analyzed 2 participants
35.00  (21.21)
Week 26 Number Analyzed 3 participants
30.00  (34.64)
Week 52 Number Analyzed 3 participants
7.00  (12.12)
Week 104 Number Analyzed 2 participants
5.00  (7.07)
Week 156 Number Analyzed 2 participants
20.00  (0.00)
25.Secondary Outcome
Title Gastrointestinal Symptoms: Number of Participants With Abdominal Distension at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to report the presence of abdominal distention in past 10 days (before each of the specified time points). Participants answered the question: "Do you currently suffer from abdominal distension (bloating, swelling or tight tummy)? [Yes/No]". For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 11 participants
No 9
Yes 2
Week 2 Number Analyzed 11 participants
No 8
Yes 3
Week 4 Number Analyzed 11 participants
No 7
Yes 4
Week 8 Number Analyzed 11 participants
No 9
Yes 2
Week 12 Number Analyzed 10 participants
No 9
Yes 1
Week 18 Number Analyzed 9 participants
No 8
Yes 1
Week 26 Number Analyzed 9 participants
No 8
Yes 1
Week 52 Number Analyzed 7 participants
No 7
Yes 0
Week 104 Number Analyzed 7 participants
No 7
Yes 0
Week 156 Number Analyzed 7 participants
No 6
Yes 1
26.Secondary Outcome
Title Gastrointestinal Symptoms: Abdominal Distension Severity Score at Baseline and Weeks 2, 4, 8, 12, 18, 26, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to mark the severity of the abdominal distension in past 10 days (before each of the specified time points) on a VAS. The scale ranged from 0% (no distention) to 100% (very severe), where higher score indicated more severity. For this analysis, baseline was defined as initial ACT13739 study baseline. The SD can only be calculated when there are more than 1 participant with data available. Thereby, applicable fields were left blank when SD was not calculable.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a 0-100 percent scale
Baseline Number Analyzed 3 participants
45.33  (24.01)
Week 2 Number Analyzed 3 participants
38.67  (38.42)
Week 4 Number Analyzed 4 participants
21.00  (26.15)
Week 8 Number Analyzed 2 participants
35.00  (48.08)
Week 12 Number Analyzed 1 participants
72.00
Week 18 Number Analyzed 1 participants
69.00
Week 26 Number Analyzed 1 participants
80.00
Week 156 Number Analyzed 1 participants
5.00
27.Secondary Outcome
Title Gastrointestinal Symptoms: Number of Participants in Categories of Response Regarding Eating Less Due to Abdominal Pain/Bloating at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants responded to question "How often do you eat less during meals due to abdominal pain and/or bloating?" in past 10 days (before each of the specified time points) in the categories as 'never', 'occasionally' or 'often'. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 11 participants
Never 4
Occasionally 5
Often 2
Week 2 Number Analyzed 11 participants
Never 6
Occasionally 3
Often 2
Week 4 Number Analyzed 11 participants
Never 6
Occasionally 3
Often 2
Week 8 Number Analyzed 11 participants
Never 7
Occasionally 3
Often 1
Week 12 Number Analyzed 10 participants
Never 5
Occasionally 3
Often 2
Week 18 Number Analyzed 9 participants
Never 4
Occasionally 3
Often 2
Week 26 Number Analyzed 9 participants
Never 4
Occasionally 3
Often 2
Week 52 Number Analyzed 7 participants
Never 3
Occasionally 3
Often 1
Week 104 Number Analyzed 7 participants
Never 4
Occasionally 3
Often 0
Week 156 Number Analyzed 7 participants
Never 4
Occasionally 3
Often 0
28.Secondary Outcome
Title Gastrointestinal Symptoms: Satisfaction Over Bowel Habits at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to mark their satisfaction over bowel habits in past 10 days (before each of the specified time points) on a VAS. The scale ranged from 0% (very happy) to 100% (very unhappy), where higher percentage indicated less satisfaction. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a 0-100 percent scale
Baseline Number Analyzed 11 participants
26.55  (19.09)
Week 2 Number Analyzed 11 participants
30.82  (21.17)
Week 4 Number Analyzed 11 participants
36.09  (28.68)
Week 8 Number Analyzed 11 participants
34.27  (27.02)
Week 12 Number Analyzed 10 participants
21.50  (17.83)
Week 18 Number Analyzed 9 participants
23.89  (21.29)
Week 26 Number Analyzed 9 participants
35.56  (29.37)
Week 52 Number Analyzed 6 participants
19.00  (17.05)
Week 104 Number Analyzed 5 participants
18.00  (15.18)
Week 156 Number Analyzed 6 participants
34.02  (36.67)
29.Secondary Outcome
Title Gastrointestinal Symptoms: Frequency of Bowel Movements - Most Number of Times Bowel Movement Per Day at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to report the frequency of their bowel movement (per day or per week or per month) in past 10 days (before each of the specified time points) by answering the question "What is the most number of times you move your bowels per day/week/month?". Participants selected their preferred time unit (e.g., per day). Response provided by participants was converted to number of times per day for reporting the results. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: number of bowel movements per day
Baseline Number Analyzed 11 participants
2.36  (1.03)
Week 2 Number Analyzed 11 participants
2.00  (0.63)
Week 4 Number Analyzed 11 participants
2.00  (1.00)
Week 8 Number Analyzed 11 participants
1.74  (1.07)
Week 12 Number Analyzed 10 participants
2.04  (1.08)
Week 18 Number Analyzed 9 participants
2.17  (1.05)
Week 26 Number Analyzed 9 participants
2.16  (1.07)
Week 52 Number Analyzed 7 participants
2.08  (1.00)
Week 104 Number Analyzed 7 participants
1.96  (1.05)
Week 156 Number Analyzed 7 participants
1.65  (0.61)
30.Secondary Outcome
Title Gastrointestinal Symptoms: Frequency of Bowel Movements - Least Number of Times Bowel Movement Per Day at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to report the frequency of their bowel movement (per day or per week or per month) in past 10 days (before each of the specified time points). Participants answered the question "What is the least number of times you move your bowels per day/week/month?". Participants selected their preferred time unit (e.g., per day). Response provided by participants was converted to number of times per day for reporting the results. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: number of bowel movements per day
Baseline Number Analyzed 11 participants
0.78  (0.56)
Week 2 Number Analyzed 11 participants
0.66  (0.43)
Week 4 Number Analyzed 11 participants
0.65  (0.62)
Week 8 Number Analyzed 11 participants
0.62  (0.64)
Week 12 Number Analyzed 10 participants
0.67  (0.65)
Week 18 Number Analyzed 9 participants
0.73  (0.65)
Week 26 Number Analyzed 9 participants
0.75  (0.40)
Week 52 Number Analyzed 7 participants
0.76  (0.72)
Week 104 Number Analyzed 7 participants
0.61  (0.49)
Week 156 Number Analyzed 7 participants
0.92  (0.62)
31.Secondary Outcome
Title Gastrointestinal Symptoms: Influence of GI Symptoms of Fabry Disease on Life at Baseline and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156
Hide Description Participants assessed their GI symptoms (abdominal pain, abdominal distention, bowel movements) by completing a questionnaire (modified version of the inflammatory bowel severity scoring system). Participants were asked to mark the influence of their GI symptoms of Fabry disease on life in past 10 days (before each of the specified time points) on a VAS. The scale ranged from 0% (no at all) to 100% (completely), where higher percentage indicated more influence of the GI symptoms of the disease on life. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 2, 4, 8, 12, 18, 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a 0-100 percent scale
Baseline Number Analyzed 11 participants
34.36  (26.47)
Week 2 Number Analyzed 11 participants
26.73  (25.22)
Week 4 Number Analyzed 11 participants
21.27  (19.88)
Week 8 Number Analyzed 11 participants
22.09  (19.77)
Week 12 Number Analyzed 10 participants
17.50  (19.92)
Week 18 Number Analyzed 9 participants
24.89  (23.34)
Week 26 Number Analyzed 9 participants
36.44  (30.18)
Week 52 Number Analyzed 6 participants
20.33  (22.04)
Week 104 Number Analyzed 5 participants
26.50  (23.31)
Week 156 Number Analyzed 6 participants
19.82  (21.14)
32.Secondary Outcome
Title Gastrointestinal Symptoms: Number of Participants With Stool Consistency Assessment by Bristol Stool Scale Scoring at Baseline and Weeks 26, 52, 104, and 156
Hide Description Participants were asked to rate their stool consistency in past 10 days (before each of the specified time points) on a 7-point Bristol stool scale, according to the following types: 1 = separate hard lumps, 2 = sausage shaped but lumpy, 3 = sausage-like with cracks on the surface, 4 = sausage-like but smooth and soft, 5 = soft blobs with clear cut edges, 6 = fluffy pieces with ragged edges, and 7 = watery with no solid pieces. Types 1 and 2 indicate constipation, types 3 and 4 indicate "ideal stools" (easiest to defecate), and 5-7 tending towards diarrhea. Frequency of each stool type was categorized as 'never', occasionally', or 'often'. For this analysis, baseline was defined as the initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Stool Type 1: Baseline Number Analyzed 11 participants
Never 11
Occasionally 0
Often 0
Stool Type 1: Week 26 Number Analyzed 9 participants
Never 8
Occasionally 1
Often 0
Stool Type 1: Week 52 Number Analyzed 7 participants
Never 6
Occasionally 0
Often 1
Stool Type 1: Week 104 Number Analyzed 7 participants
Never 6
Occasionally 0
Often 1
Stool Type 1: Week 156 Number Analyzed 7 participants
Never 5
Occasionally 1
Often 1
Stool Type 2: Baseline Number Analyzed 11 participants
Never 6
Occasionally 5
Often 0
Stool Type 2: Week 26 Number Analyzed 9 participants
Never 6
Occasionally 0
Often 3
Stool Type 2: Week 52 Number Analyzed 7 participants
Never 3
Occasionally 4
Often 0
Stool Type 2: Week 104 Number Analyzed 7 participants
Never 4
Occasionally 2
Often 1
Stool Type 2: Week 156 Number Analyzed 7 participants
Never 2
Occasionally 3
Often 2
Stool Type 3: Baseline Number Analyzed 11 participants
Never 1
Occasionally 4
Often 6
Stool Type 3: Week 26 Number Analyzed 9 participants
Never 1
Occasionally 3
Often 5
Stool Type 3: Week 52 Number Analyzed 7 participants
Never 0
Occasionally 3
Often 4
Stool Type 3: Week 104 Number Analyzed 7 participants
Never 0
Occasionally 6
Often 1
Stool Type 3: Week 156 Number Analyzed 7 participants
Never 0
Occasionally 4
Often 3
Stool Type 4: Baseline Number Analyzed 11 participants
Never 1
Occasionally 8
Often 2
Stool Type 4: Week 26 Number Analyzed 9 participants
Never 2
Occasionally 4
Often 3
Stool Type 4: Week 52 Number Analyzed 7 participants
Never 3
Occasionally 1
Often 3
Stool Type 4: Week 104 Number Analyzed 7 participants
Never 2
Occasionally 3
Often 2
Stool Type 4: Week 156 Number Analyzed 7 participants
Never 3
Occasionally 2
Often 2
Stool type 5: Baseline Number Analyzed 11 participants
Never 1
Occasionally 9
Often 1
Stool type 5: Week 26 Number Analyzed 9 participants
Never 4
Occasionally 4
Often 1
Stool type 5: Week 52 Number Analyzed 7 participants
Never 5
Occasionally 2
Often 0
Stool type 5: Week 104 Number Analyzed 7 participants
Never 4
Occasionally 1
Often 2
Stool type 5: Week 156 Number Analyzed 7 participants
Never 3
Occasionally 3
Often 1
Stool Type 6: Baseline Number Analyzed 11 participants
Never 5
Occasionally 5
Often 1
Stool Type 6: Week 26 Number Analyzed 9 participants
Never 4
Occasionally 4
Often 1
Stool Type 6: Week 52 Number Analyzed 7 participants
Never 4
Occasionally 2
Often 1
Stool Type 6: Week 104 Number Analyzed 7 participants
Never 3
Occasionally 3
Often 1
Stool Type 6: Week 156 Number Analyzed 7 participants
Never 4
Occasionally 3
Often 0
Stool Type 7: Baseline Number Analyzed 11 participants
Never 5
Occasionally 5
Often 1
Stool Type 7: Week 26 Number Analyzed 9 participants
Never 5
Occasionally 4
Often 0
Stool Type 7: Week 52 Number Analyzed 7 participants
Never 5
Occasionally 2
Often 0
Stool Type 7: Week 104 Number Analyzed 7 participants
Never 4
Occasionally 3
Often 0
Stool Type 7: Week 156 Number Analyzed 7 participants
Never 3
Occasionally 3
Often 1
33.Secondary Outcome
Title Change From Baseline in Beck Depression Inventory (BDI) Total Score at Weeks 26, 104, and 156
Hide Description The BDI-II Scale was a 21-item scoring tool which measures the existence and severity of symptoms of depression. Each of the 21 items on BDI-II tool represent a depressive symptom. Each symptoms were scored on a 4-point scale of 0 to 3 (0=symptom not present); (3=symptom very intense). Scores for each symptom were added up to obtain the total scores for all 21 items, which were interpreted as follows: Scores of 0-13: minimal depression; 14-19: mild depression; 20-28: moderate depression and 29-63: severe depression, where higher scores indicated more depression. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: score on a scale
Week 26 Number Analyzed 9 participants
-0.89  (8.13)
Week 104 Number Analyzed 4 participants
2.00  (3.37)
Week 156 Number Analyzed 7 participants
-3.43  (7.09)
34.Secondary Outcome
Title Change From Baseline in Albumin/Creatinine Ratio (ACR) and Protein/Creatinine Ratio (PCR) at Weeks 26, 52, 104, and 156
Hide Description For each scheduled visit for this assessment, 3 timed overnight urine samples were collected between 4 to 7 days of each other. All urine samples were collected within a 16-day period. ACR and PCR were determined for each collection. The median of the values determined for the 3 collections/visit was used for analysis. Baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mg/g
ACR: Week 26 Number Analyzed 9 participants
-26.22  (26.75)
ACR: Week 52 Number Analyzed 7 participants
1.00  (57.23)
ACR: Week 104 Number Analyzed 7 participants
12.21  (71.82)
ACR: Week 156 Number Analyzed 7 participants
-1.14  (54.80)
PCR: Week 26 Number Analyzed 9 participants
-38.33  (24.40)
PCR: Week 52 Number Analyzed 7 participants
0.33  (62.11)
PCR: Week 104 Number Analyzed 7 participants
-12.88  (76.97)
PCR: Week 156 Number Analyzed 7 participants
-34.38  (71.40)
35.Secondary Outcome
Title Number of Participants in Categories of Echocardiogram (ECHO) Results at Baseline and at Weeks 26, 52, 104, and 156
Hide Description The summary statistics of all continuous echocardiogram variables were calculated for each visit. The overall interpretation of the readings were summarized in 3 categories: normal, abnormal but not clinically significant (NCS), and abnormal but clinically significant (CS) categories. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 8 participants
Normal 6
Abnormal NCS 2
Abnormal CS 0
Week 26 Number Analyzed 9 participants
Normal 6
Abnormal NCS 3
Abnormal CS 0
Week 52 Number Analyzed 7 participants
Normal 5
Abnormal NCS 2
Abnormal CS 0
Week 104 Number Analyzed 7 participants
Normal 4
Abnormal NCS 3
Abnormal CS 0
Week 156 Number Analyzed 7 participants
Normal 5
Abnormal NCS 2
Abnormal CS 0
36.Secondary Outcome
Title Number of Participants in Categories of Brain Magnetic Resonance Imaging (MRI) Results at Baseline and Weeks 26, and 156
Hide Description All continuous MRI variables were summarized using descriptive statistics for each visit. The overall interpretation of the readings were summarized in 2 categories as: normal, and abnormal. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 10 participants
Normal
6
  60.0%
Abnormal
4
  40.0%
Week 26 Number Analyzed 9 participants
Normal
7
  77.8%
Abnormal
2
  22.2%
Week 156 Number Analyzed 7 participants
Normal
4
  57.1%
Abnormal
3
  42.9%
37.Secondary Outcome
Title Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) at Weeks 26, 52, 104, and 156
Hide Description Estimated glomerular filtration rate was used to measure level of kidney function and determine the stage of kidney disease. Change from baseline in eGFR was obtained by subtracting baseline value from post-baseline value at Weeks 26, 52, 104 and 156. For this analysis, baseline was defined as initial ACT13739 study baseline.
Time Frame Baseline of ACT13739 study and Weeks 26, 52, 104, and 156 post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set: all participants who received at least 1 dose of IMP during the ACT13739 study. All data collected during ACT13739 and LTS14116 studies were included in analysis. Here, 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: mL/min/1.73m^2
Week 26 Number Analyzed 7 participants
-3.43  (8.64)
Week 52 Number Analyzed 7 participants
-5.57  (11.07)
Week 104 Number Analyzed 7 participants
-5.71  (10.23)
Week 156 Number Analyzed 6 participants
-4.83  (17.54)
38.Secondary Outcome
Title Chitotriosidase (Chit1) Plasma Concentration Levels at Weeks 52, 104,156 and 160 (End of Treatment Follow-up)
Hide Description Plasma concentrations of Chit1 over time were determined using mass spectrometry (MS)-based assay. For the analysis, 52 ng/mL was considered as the lower limit of quantification. Although identified in protocol as a secondary endpoint, plasma Chit1 is also an exploratory measure. Only LTS14116 timepoints were analyzed and are presented. Data summarized are the measured values at each time point (not change from baseline). Here, measured value '0.000' denotes no chitotriosidase detected in plasma for the 5 participants at Week 156 reported by laboratory for all evaluable participants.
Time Frame Weeks 52, 104, 156 and 160 (End of Treatment Follow-up) post-ACT13739 baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis population included participants in LTS14116 study with evaluable plasma Chit1 data. Only data collected during LTS14116 study were included in analysis. Here, 'overall number of participants analyzed' = participants evaluable for this outcome measure and 'number analyzed' = participants with available data for each specified category.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: ng/mL
Week 52 Number Analyzed 7 participants
35.965  (46.520)
Week 104 Number Analyzed 7 participants
12.785  (20.993)
Week 156 Number Analyzed 5 participants
0.000  (0.000)
Week 160 (End of Treatment Follow-up) Number Analyzed 4 participants
6.483  (6.309)
Time Frame From baseline of ACT13739 study up to 37 months post-ACT13739 baseline.
Adverse Event Reporting Description Reported AEs were TEAEs, defined as AEs that developed/worsened during TEAE period (period from the first administration of study drug in ACT13739 through the last administration of the study drug in the combined ACT13739/LTS14116 treatment period plus 1 month or end of study participation for participant, whichever occurs first). Analysis was performed on safety population.
 
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description Participants received GZ/SAR402671 15 mg once daily orally for 36 months during combined ACT13739/LTS14116 treatment period.
All-Cause Mortality
GZ/SAR402671
Affected / at Risk (%)
Total   0/11 (0.00%) 
Hide Serious Adverse Events
GZ/SAR402671
Affected / at Risk (%)
Total   3/11 (27.27%) 
Blood and lymphatic system disorders   
Haemolysis  1  1/11 (9.09%) 
Ear and labyrinth disorders   
Deafness unilateral  1  1/11 (9.09%) 
Vertigo  1  1/11 (9.09%) 
Musculoskeletal and connective tissue disorders   
Floppy infant  1  1/11 (9.09%) 
Nervous system disorders   
Ischaemic stroke  1  1/11 (9.09%) 
Psychiatric disorders   
Depressed mood  1  1/11 (9.09%) 
Intentional self-injury  1  1/11 (9.09%) 
1
Term from vocabulary, MedDRA 21.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
GZ/SAR402671
Affected / at Risk (%)
Total   9/11 (81.82%) 
Cardiac disorders   
Atrial fibrillation  1  1/11 (9.09%) 
Atrioventricular block first degree  1  1/11 (9.09%) 
Cardiomyopathy  1  2/11 (18.18%) 
Sinus bradycardia  1  1/11 (9.09%) 
Ear and labyrinth disorders   
Deafness unilateral  1  1/11 (9.09%) 
Ear pain  1  1/11 (9.09%) 
Middle ear effusion  1  1/11 (9.09%) 
Tinnitus  1  3/11 (27.27%) 
Vertigo  1  1/11 (9.09%) 
Eye disorders   
Blepharitis  1  1/11 (9.09%) 
Chalazion  1  1/11 (9.09%) 
Diplopia  1  1/11 (9.09%) 
Eye pain  1  1/11 (9.09%) 
Lacrimation increased  1  1/11 (9.09%) 
Lenticular opacities  1  1/11 (9.09%) 
Retinal vascular disorder  1  2/11 (18.18%) 
Vision blurred  1  1/11 (9.09%) 
Gastrointestinal disorders   
Abdominal pain upper  1  1/11 (9.09%) 
Constipation  1  1/11 (9.09%) 
Diarrhoea  1  1/11 (9.09%) 
Dry mouth  1  1/11 (9.09%) 
Gastrooesophageal reflux disease  1  2/11 (18.18%) 
Nausea  1  2/11 (18.18%) 
Oesophageal discomfort  1  1/11 (9.09%) 
Toothache  1  1/11 (9.09%) 
Vomiting  1  2/11 (18.18%) 
General disorders   
Fatigue  1  2/11 (18.18%) 
Feeling hot  1  2/11 (18.18%) 
Influenza like illness  1  1/11 (9.09%) 
Non-cardiac chest pain  1  1/11 (9.09%) 
Oedema  1  1/11 (9.09%) 
Oedema peripheral  1  1/11 (9.09%) 
Pain  1  2/11 (18.18%) 
Pyrexia  1  2/11 (18.18%) 
Temperature intolerance  1  1/11 (9.09%) 
Infections and infestations   
Conjunctivitis  1  1/11 (9.09%) 
Ear infection  1  1/11 (9.09%) 
Herpes simplex  1  1/11 (9.09%) 
Influenza  1  1/11 (9.09%) 
Nasopharyngitis  1  4/11 (36.36%) 
Periodontitis  1  1/11 (9.09%) 
Pharyngitis  1  1/11 (9.09%) 
Pharyngitis streptococcal  1  1/11 (9.09%) 
Rhinitis  1  2/11 (18.18%) 
Upper respiratory tract infection  1  3/11 (27.27%) 
Upper respiratory tract infection bacterial  1  1/11 (9.09%) 
Injury, poisoning and procedural complications   
Arthropod bite  1  1/11 (9.09%) 
Contusion  1  1/11 (9.09%) 
Joint injury  1  1/11 (9.09%) 
Limb injury  1  1/11 (9.09%) 
Meniscus injury  1  1/11 (9.09%) 
Muscle strain  1  2/11 (18.18%) 
Tooth fracture  1  1/11 (9.09%) 
Wound  1  1/11 (9.09%) 
Investigations   
Electrocardiogram T wave abnormal  1  1/11 (9.09%) 
Electrocardiogram abnormal  1  1/11 (9.09%) 
Glomerular filtration rate decreased  1  1/11 (9.09%) 
Nuclear magnetic resonance imaging brain abnormal  1  1/11 (9.09%) 
Vibration test abnormal  1  1/11 (9.09%) 
Metabolism and nutrition disorders   
Decreased appetite  1  1/11 (9.09%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/11 (9.09%) 
Back pain  1  1/11 (9.09%) 
Muscle twitching  1  1/11 (9.09%) 
Musculoskeletal chest pain  1  1/11 (9.09%) 
Myalgia  1  2/11 (18.18%) 
Neck pain  1  1/11 (9.09%) 
Pain in extremity  1  1/11 (9.09%) 
Rotator cuff syndrome  1  1/11 (9.09%) 
Nervous system disorders   
Amnesia  1  1/11 (9.09%) 
Balance disorder  1  1/11 (9.09%) 
Burning sensation  1  1/11 (9.09%) 
Carpal tunnel syndrome  1  1/11 (9.09%) 
Cerebral ischaemia  1  1/11 (9.09%) 
Dizziness  1  2/11 (18.18%) 
Headache  1  4/11 (36.36%) 
Hemiparesis  1  1/11 (9.09%) 
Hypoaesthesia  1  2/11 (18.18%) 
Memory impairment  1  1/11 (9.09%) 
Migraine  1  1/11 (9.09%) 
Monoparesis  1  1/11 (9.09%) 
Neuralgia  1  1/11 (9.09%) 
Paraesthesia  1  1/11 (9.09%) 
Presyncope  1  1/11 (9.09%) 
Tremor  1  2/11 (18.18%) 
White matter lesion  1  1/11 (9.09%) 
Psychiatric disorders   
Anxiety  1  1/11 (9.09%) 
Depressed mood  1  1/11 (9.09%) 
Depression  1  1/11 (9.09%) 
Nervousness  1  1/11 (9.09%) 
Renal and urinary disorders   
Micturition frequency decreased  1  1/11 (9.09%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  2/11 (18.18%) 
Dry throat  1  1/11 (9.09%) 
Dyspnoea  1  2/11 (18.18%) 
Hiccups  1  1/11 (9.09%) 
Nasal discomfort  1  1/11 (9.09%) 
Oropharyngeal pain  1  1/11 (9.09%) 
Sinus pain  1  1/11 (9.09%) 
Skin and subcutaneous tissue disorders   
Acne  1  1/11 (9.09%) 
Angiokeratoma  1  3/11 (27.27%) 
Dermatitis contact  1  1/11 (9.09%) 
Hyperhidrosis  1  1/11 (9.09%) 
1
Term from vocabulary, MedDRA 21.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Trial Transparency Team
Organization: Sanofi
Phone: 800-633-1610 ext 1#
EMail: Contact-US@sanofi.com
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Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT02489344    
Other Study ID Numbers: LTS14116
2014-004995-49 ( EudraCT Number )
U1111-1165-9049 ( Other Identifier: UTN )
First Submitted: June 30, 2015
First Posted: July 3, 2015
Results First Submitted: November 18, 2019
Results First Posted: December 9, 2019
Last Update Posted: March 23, 2021