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Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease

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ClinicalTrials.gov Identifier: NCT02228460
Recruitment Status : Completed
First Posted : August 29, 2014
Results First Posted : December 17, 2019
Last Update Posted : December 17, 2019
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 11
Recruitment Details Participants were enrolled in the study at 8 centers in 5 countries between 11 November 2014 and 06 September 2016. A total of 14 participants were screened in the study.
Pre-assignment Details Out of 14 screened participants, 11 participants were enrolled and treated in the study.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description GZ/SAR402671 15 milligram (mg) once daily, orally for 26 weeks.
Period Title: Overall Study
Started 11
Completed 9
Not Completed 2
Reason Not Completed
Adverse Event             1
Lost to Follow-up             1
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment.
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%
1.Primary Outcome
Title Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
Hide Description Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy 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 Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on full analysis set (FAS) that included all participants who received at least 1 dose of study treatment. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score: 1 / Week 26 Score: 1
4
  44.4%
Baseline Score: 1 / Week 26 Score: 2
1
  11.1%
Baseline Score: 2 / Week 26 Score: 1
3
  33.3%
Baseline Score: 2 / Week 26 Score: 2
1
  11.1%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection GZ/SAR402671
Comments A McNemar test was used to test the treatment effect based on paired pre-treatment and post-treatment (Week 26) frequencies of skin GL-3 score grouped into the categories (0 to <2; 2 to 3).
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3173
Comments Threshold for significance at 0.05 level.
Method McNemar Test
Comments [Not Specified]
2.Primary Outcome
Title Mean Change From Baseline at Week 26 in Skin GL-3 Score in Superficial Skin Capillary Endothelium
Hide Description Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy 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. Change from Baseline in GL-3 score was obtained by subtracting Baseline value from post-baseline value at Week 26. A negative change from Baseline indicates less severe condition at Week 26.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (95% Confidence Interval)
Unit of Measure: score on a scale
-0.22
(-0.73 to 0.29)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection GZ/SAR402671
Comments Wilcoxon signed rank test was used to assess the mean change from Baseline to Week 26 in skin GL-3 score.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.625
Comments Threshold for significance at 0.05 level.
Method Wilcoxon signed rank test
Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in Plasma GL-3 Concentration at Week 26
Hide Description Change from Baseline in plasma GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in plasma was determined using a validated liquid chromatography tandem mass spectrometry (LC-MS/MS) method.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: mcg/mL
-3.62  (1.07)
4.Secondary Outcome
Title Change From Baseline in Plasma Lyso Globotriaosylceramide (Lyso-GL-3) Concentration at Week 26
Hide Description Change from Baseline in plasma Lyso-GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of lyso-GL-3 in plasma was determined using a validated LC-MS/MS method.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: ng/mL
-30.99  (22.83)
5.Secondary Outcome
Title Change From Baseline in Plasma Glucosylceramide (GL-1) Concentration at Week 26
Hide Description Change from Baseline in plasma GL-1 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-1 in plasma was determined using a validated LC-MS/MS method.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: mcg/mL
-3.26  (1.43)
6.Secondary Outcome
Title Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Endothelial Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
Hide Description Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy 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 Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score: 1 / Week 26 Score: 1
1
  11.1%
Baseline Score: 2 / Week 26 Score: 1
2
  22.2%
Baseline Score: 2 / Week 26 Score: 2
6
  66.7%
7.Secondary Outcome
Title Change From Baseline at Week 26 in Skin GL-3 Score in Deep Vessels Smooth Muscle Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
Hide Description Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy 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 Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score: 1.5 / Week 26 Score: 1.5
2
  22.2%
Baseline Score: 2 / Week 26 Score: 2
7
  77.8%
8.Secondary Outcome
Title Change From Baseline at Week 26 in Skin GL-3 Score in Perineurium Cells: Number of Participants in Categories of Shift in GL-3 Score From Baseline to Week 26
Hide Description Skin biopsies taken at Baseline and Week 26 were analyzed for cellular GL-3 accumulation (inclusions) by light microscopy. Three independent pathologists evaluated each biopsy 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 Week 26 GL-3 score. Any shift category of Baseline score/Week 26 score that was not observed is not reported. Shift to lower score from Baseline to Week 26 indicates less severe condition at Week 26.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Score: 1 / Week 26 Score: 2
1
  11.1%
Baseline Score: 2 / Week 26 Score: 2
8
  88.9%
9.Secondary Outcome
Title Change From Baseline in Urine Globotriaosylceramide (GL-3) Concentration at Week 26
Hide Description Change from Baseline in urine GL-3 was obtained by subtracting Baseline value from post-baseline value at Week 26. Concentration of GL-3 in urine was determined using a validated LC-MS/MS method.
Time Frame Baseline, Week 26
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on FAS. Here, 'overall number of participants analyzed' = participants with available data at both Baseline and Week 26.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 8
Mean (Standard Deviation)
Unit of Measure: mg/mmol Cr
-0.25  (0.19)
10.Secondary 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 on-treatment period (period from the first administration of study drug through the last administration of the study drug plus 30 days or end of study participation for participant, whichever occurs first).
Time Frame From Baseline up to 212 days
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on safety population which included all enrolled participants who received at least 1 dose of study drug.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
9
  81.8%
11.Secondary Outcome
Title Pharmacokinetics (PK): Maximum Plasma Drug Concentration (Cmax) of GZ/SAR402671
Hide Description Maximum plasma concentration observed for study drug was reported.
Time Frame Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population which included all participants for whom the primary PK data were considered sufficient and interpretable. Here, 'number analyzed' = participants with available data at specified time-point.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 1 Number Analyzed 11 participants
24.7  (5.89)
Day 182 Number Analyzed 9 participants
192.0  (96.4)
12.Secondary Outcome
Title PK: Plasma Trough Concentration (Ctrough) of GZ/SAR402671
Hide Description Ctrough was defined as the plasma concentration of study drug observed just before treatment administration during repeated dosing.
Time Frame Predose on Days 14, 28, 56, 84, 126, and 182
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 14 Number Analyzed 11 participants
152.0  (68.9)
Day 28 Number Analyzed 11 participants
165.0  (66.1)
Day 56 Number Analyzed 10 participants
182.0  (90.3)
Day 84 Number Analyzed 10 participants
164.0  (124.0)
Day 126 Number Analyzed 9 participants
175.0  (94.7)
Day 182 Number Analyzed 9 participants
164.0  (89.4)
13.Secondary Outcome
Title PK: Time to Reach Maximum Plasma Drug Concentration (Tmax) of GZ/SAR402671
Hide Description Tmax was defined as time to reach maximum plasma concentration of study drug.
Time Frame Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 11
Median (Full Range)
Unit of Measure: hours
Day 1 Number Analyzed 11 participants
8.00
(1.07 to 24.00)
Day 182 Number Analyzed 9 participants
4.00
(0.00 to 8.00)
14.Secondary Outcome
Title PK: Area Under Plasma Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24) of GZ/SAR402671
Hide Description Area under the plasma concentration versus time curve of study drug from time 0 to 24 hours (AUC0-24) was calculated using the trapezoidal method over the dosing interval.
Time Frame Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: ng*hour/mL
Day 1 Number Analyzed 11 participants
476  (125)
Day 182 Number Analyzed 9 participants
4110  (2090)
15.Secondary Outcome
Title PK: Terminal Half-life (t1/2z) of GZ/SAR402671
Hide Description Plasma t1/2z was the time measured for the plasma concentration of drug to decrease by one half. The t1/2z was estimated based on 24-hour post-dose PK.
Time Frame Day 1 (predose and 1, 2, 4, 8, and 24 hours post-dose); Day 182 (predose and 1, 2, 4, 8, and 24 hours post-dose)
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'number analyzed' = participants with available data at specified time-point.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 11
Mean (Standard Deviation)
Unit of Measure: hours
Day 1 Number Analyzed 4 participants
86.8  (39.6)
Day 182 Number Analyzed 7 participants
128.0  (59.0)
16.Secondary Outcome
Title PK: Apparent Total Body Clearance of GZ/SAR402671 at Steady State (CLss/F)
Hide Description Apparent total body clearance at steady state was a quantitative measure of rate of clearance of drug from the blood following oral administration, and is described in terms of volume of fluid clear of drug per time unit (eg, mL/min).
Time Frame Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: mL/hour
7490  (10900)
17.Secondary Outcome
Title PK: Apparent Volume of Distribution of GZ/SAR402671 (Vss/F) at Steady State
Hide Description Volume of distribution at steady state was defined as the theoretical volume in which the total amount of drug would need to be uniformly distributed to produce the desired plasma concentration of drug.
Time Frame Predose and 1, 2, 4, 8, and 24 hours post-dose on Day 182
Hide Outcome Measure Data
Hide Analysis Population Description
Since the percent extrapolation of AUC for all participants was >30%, AUC could not be determined and hence, Vss/F could not be calculated.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
18.Secondary Outcome
Title PK: Cumulated Amount of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (Ae0-24)
Hide Description Ae0-24 was the cumulated amount of study drug excreted in urine during the time interval of 0 to 24 hours.
Time Frame 0-24 hours on Day 182
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: mcg
3210.0  (1640.0)
19.Secondary Outcome
Title PK: Percentage of Dose of GZ/SAR402671 Excreted in Urine From 0 to 24 Hours (fe0-24)
Hide Description fe0-24 was the fraction of dose excreted in urine during the time interval of 0 to 24 hours.
Time Frame 0-24 hours on Day 182
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: percentage of dose
21.4  (10.9)
20.Secondary Outcome
Title PK: Renal Clearance (CLR) of GZ/SAR402671 From 0 to 24 Hours
Hide Description CLR was calculated by dividing the cumulative amount of drug excreted in urine during the dosing interval of 0-24 hours by area under the plasma drug concentration time-curve during the dosing interval of 0-24 hours.
Time Frame 0-24 hours on Day 182
Hide Outcome Measure Data
Hide Analysis Population Description
Analysis was performed on PK population. Here, 'overall number of participants analyzed' = participants with available data for this outcome measure.
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description:
GZ/SAR402671 15 mg once daily, orally for 26 weeks.
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: mL/hour
925  (407)
Time Frame Baseline up to 212 days
Adverse Event Reporting Description Reported AEs are TEAEs that is AEs developed/worsened during "on treatment period" (from first administration through last administration of study drug + 30 days or end of study participation for participant, whichever occurs first). Analysis was performed on safety population that included all enrolled participants who received at least 1 dose of study drug.
 
Arm/Group Title GZ/SAR402671
Hide Arm/Group Description GZ/SAR402671 15 mg once daily, orally for 26 weeks.
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%) 
Musculoskeletal and connective tissue disorders   
Floppy infant  1  1/11 (9.09%) 
Psychiatric disorders   
Depressed mood  1  1/11 (9.09%) 
1
Term from vocabulary, MedDRA 19.0
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%) 
Ear and labyrinth disorders   
Tinnitus  1  2/11 (18.18%) 
Eye disorders   
Blepharitis  1  1/11 (9.09%) 
Chalazion  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  1/11 (9.09%) 
Vision blurred  1  1/11 (9.09%) 
Gastrointestinal disorders   
Constipation  1  1/11 (9.09%) 
Dry mouth  1  1/11 (9.09%) 
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%) 
Pyrexia  1  2/11 (18.18%) 
Infections and infestations   
Ear infection  1  1/11 (9.09%) 
Nasopharyngitis  1  1/11 (9.09%) 
Rhinitis  1  2/11 (18.18%) 
Upper respiratory tract infection  1  2/11 (18.18%) 
Injury, poisoning and procedural complications   
Muscle strain  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%) 
Nuclear magnetic resonance imaging brain abnormal  1  1/11 (9.09%) 
Vibration test abnormal  1  1/11 (9.09%) 
Musculoskeletal and connective tissue disorders   
Muscle twitching  1  1/11 (9.09%) 
Musculoskeletal chest pain  1  1/11 (9.09%) 
Myalgia  1  1/11 (9.09%) 
Neck pain  1  1/11 (9.09%) 
Pain in extremity  1  1/11 (9.09%) 
Nervous system disorders   
Amnesia  1  1/11 (9.09%) 
Dizziness  1  2/11 (18.18%) 
Headache  1  4/11 (36.36%) 
Hypoaesthesia  1  1/11 (9.09%) 
Migraine  1  1/11 (9.09%) 
Paraesthesia  1  1/11 (9.09%) 
Sinus headache  1  1/11 (9.09%) 
White matter lesion  1  1/11 (9.09%) 
Psychiatric disorders   
Anxiety  1  1/11 (9.09%) 
Depressed mood  1  1/11 (9.09%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  2/11 (18.18%) 
Dyspnoea  1  2/11 (18.18%) 
Oropharyngeal pain  1  1/11 (9.09%) 
Skin and subcutaneous tissue disorders   
Acne  1  1/11 (9.09%) 
Angiokeratoma  1  2/11 (18.18%) 
Hyperhidrosis  1  1/11 (9.09%) 
1
Term from vocabulary, MedDRA 19.0
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
Layout table for additonal information
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT02228460    
Other Study ID Numbers: ACT13739
2013-005324-41
U1111-1152-1456 ( Other Identifier: UTN )
First Submitted: August 27, 2014
First Posted: August 29, 2014
Results First Submitted: November 26, 2019
Results First Posted: December 17, 2019
Last Update Posted: December 17, 2019