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Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

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ClinicalTrials.gov Identifier: NCT00925301
Recruitment Status : Completed
First Posted : June 22, 2009
Results First Posted : October 30, 2018
Last Update Posted : October 30, 2018
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double (Participant, Investigator);   Primary Purpose: Treatment
Condition Fabry Disease
Interventions Drug: migalastat hydrochloride
Drug: Placebo
Enrollment 67

Recruitment Details  
Pre-assignment Details During Stage 1 (0-6 Months), all participants were randomized to either Migalastat or Placebo. After Stage 1, all participants progressed to Stage 2 where they received open-label migalastat for 6 months (>6-12 Months). After Stage 2, participants were eligible to enter the optional, 12-month, open-label extension (OLE) (>12-24 Months).
Arm/Group Title Migalastat (0-6 Months) Placebo (0-6 Months) Migalastat (>6-12 Months) Migalastat (>12-24 Months)
Hide Arm/Group Description Migalastat hydrochloride (migalastat) 150-milligram (mg) capsule (equivalent to 123 mg of migalastat) given orally every other day (QOD) during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2). Includes all participants who progressed from both the migalastat (0-6 Months) and the placebo (0-6 Months) Stage 1 treatment groups. After Stage 2, participants were eligible to enter the optional, 12-month OLE. Migalastat 150-mg capsule given orally QOD for up to 12 months during the optional OLE. Includes all participants who decided to continue treatment after completing the 6-month, open-label Stage 2 treatment period.
Period Title: 6-Month Double-blind (Stage 1)
Started 34 33 0 [1] 0 [2]
Received at Least 1 Dose of Study Drug 34 33 0 0
Intent-to-Treat (ITT) [3] 34 33 0 0
Modified Intent-to-Treat (mITT) [4] 30 30 0 0
Completed 34 30 0 0
Not Completed 0 3 0 0
Reason Not Completed
Withdrawal by Subject             0             2             0             0
Pregnancy             0             1             0             0
[1]
Zero participants noted because participants did not start Stage 2 yet.
[2]
Zero participants noted because participants did not start the OLE yet.
[3]
All randomized participants regardless of their participation in the study beyond Randomization.
[4]
Participants with Baseline and Month 6 biopsy results.
Period Title: 6-Month Open-label (Stage 2)
Started 0 [1] 0 [1] 63 [2] 0 [3]
ITT-amenable [4] 0 0 47 0
Completed 0 0 60 0
Not Completed 0 0 3 0
Reason Not Completed
Withdrawal by Subject             0             0             1             0
Adverse Event             0             0             2             0
[1]
Zero participants noted because participants had completed Stage 1.
[2]
One participant (Migalastat [0-6 Months]) withdrew consent after completing Month 6.
[3]
Zero participants noted because participants did not start the OLE yet.
[4]
Amenable mutations based on Good Laboratory Practice (GLP) Human Embryonic Kidney (HEK) assay.
Period Title: 12-Month Open-label Extension (Optional)
Started 0 [1] 0 [1] 0 [2] 57 [3]
ITT-amenable [4] 0 0 0 42
Completed 0 0 0 54
Not Completed 0 0 0 3
Reason Not Completed
Withdrawal by Subject             0             0             0             1
Pregnancy             0             0             0             1
Lost to Follow-up             0             0             0             1
[1]
Zero participants noted because participants had completed Stage 1.
[2]
Zero participants noted because participants had completed Stage 2.
[3]
The OLE was optional for those who completed Stage 2. Reasons for not entering were not recorded.
[4]
Amenable mutations based on GLP HEK assay.
Arm/Group Title Migalastat-Migalastat Placebo-Migalastat Total
Hide Arm/Group Description Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1), during the 6-month open-label treatment period (Stage 2), and for up to 12 months during the OLE. Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2) and for up to 12 months during the OLE. Total of all reporting groups
Overall Number of Baseline Participants 34 33 67
Hide Baseline Analysis Population Description
Safety Population: All randomized participants who received at least 1 dose of study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 34 participants 33 participants 67 participants
40.0  (13.29) 44.5  (10.18) 42.2  (11.99)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 34 participants 33 participants 67 participants
Female
22
  64.7%
21
  63.6%
43
  64.2%
Male
12
  35.3%
12
  36.4%
24
  35.8%
1.Primary Outcome
Title Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide (GL-3) Inclusions
Hide Description Renal biopsies were taken at Baseline and Month 6 (Stage 1). The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. A responder was defined as a participant with a ≥50% reduction from Baseline to Month 6 in the average number of kidney IC GL-3 inclusions.
Time Frame Baseline, Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT: All randomized participants regardless of their participation in the study beyond randomization. As per the statistical analysis plan, analysis excluded participants who were missing baseline kidney biopsy results.
Arm/Group Title Migalastat Placebo
Hide Arm/Group Description:
Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1).
Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1).
Overall Number of Participants Analyzed 32 32
Measure Type: Count of Participants
Unit of Measure: Participants
Responder
13
  40.6%
9
  28.1%
Non-Responder
19
  59.4%
23
  71.9%
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Migalastat, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2996
Comments [Not Specified]
Method Cochran-Mantel-Haenszel
Comments p-value from Cochran-Mantel-Haenszel test stratified by sex
Method of Estimation Estimation Parameter Difference
Estimated Value 12.5
Confidence Interval (2-Sided) 95%
-13.4 to 37.3
Estimation Comments The difference between the percentage of successes between migalastat and placebo treatment groups
2.Secondary Outcome
Title Percent Change In Kidney IC GL-3 Inclusions From Baseline To Month 6
Hide Description Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants' data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images.
Time Frame Baseline, Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT: All randomized participants regardless of their participation in the study beyond randomization.
Arm/Group Title Migalastat Placebo
Hide Arm/Group Description:
Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1).
Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1).
Overall Number of Participants Analyzed 34 33
Mean (Standard Deviation)
Unit of Measure: percent change
-7.948  (105.2736) 12.985  (90.5131)
3.Secondary Outcome
Title Change From Baseline Through Month 24 In Urine GL-3 Levels
Hide Description The effect of migalastat versus placebo on urine GL-3 levels was measured by liquid chromatography-mass spectrometry/mass spectrometry. The 24-hour urine samples were collected at Baseline, Month 6 (Stage 1), Month 12 (Stage 2), and Month 24 (OLE). Results are presented as changes in nanograms (ng)/mg creatinine from Baseline to the end of the 3 stages.
Time Frame Baseline, Months 6, 12, and 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Stage 1: ITT, all randomized participants regardless of participation in the study beyond randomization. Stage 2: participants who completed Stage 1 and entered Stage 2. OLE: participants who completed Stage 2 and entered the optional OLE. Once assay and sample issues were identified, later samples were not further analyzed; all data was listed.
Arm/Group Title Migalastat-Migalastat Placebo-Migalastat
Hide Arm/Group Description:
Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1), during the 6-month open-label treatment period (Stage 2), and for up to 12 months during the OLE.
Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2) and for up to 12 months during the OLE.
Overall Number of Participants Analyzed 33 30
Mean (Standard Deviation)
Unit of Measure: ng/mg creatinine
Stage 1 Number Analyzed 33 participants 30 participants
-234.80  (853.451) -186.24  (957.119)
Stage 2 Number Analyzed 31 participants 28 participants
-179.63  (699.157) -537.95  (1169.883)
OLE Number Analyzed 12 participants 9 participants
-62.37  (615.944) -177.42  (288.224)
4.Other Pre-specified Outcome
Title Change From Month 6 To Month 12 In Average Number Of Kidney IC GL-3 Inclusions
Hide Description Renal biopsies were taken at Month 6 and Month 12. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants’ data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Month 6 and Month 12. Assessments were made using digital images.
Time Frame Month 6, Month 12
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
mITT with amenable mutations: Randomized participants who switched from placebo to migalastat during Stage 2, received at least 1 dose of study drug, and underwent a renal biopsy at both Baseline and Month 6. Amenable mutations are mutant forms of α-galactosidase A (α Gal-A) amenable to migalastat. Amenable mutations based on the GLP HEK assay.
Arm/Group Title Placebo-Migalastat
Hide Arm/Group Description:
Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2).
Overall Number of Participants Analyzed 20
Least Squares Mean (95% Confidence Interval)
Unit of Measure: kidney IC GL-3 inclusions
-0.320
(-0.5719 to -0.0677)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo-Migalastat
Comments [Not Specified]
Type of Statistical Test Other
Comments Mixed effects model for repeated measures (MMRM) approach with fixed effects of treatment, time (Month 6 and Month 12), mutation type (amenable or non-amenable), time by treatment interaction, time by mutation type interaction, and the baseline value as a covariate. An unstructured covariance matrix to account for repeated measures within a participant was assumed.
Statistical Test of Hypothesis P-Value 0.014
Comments Significant at the 0.050 level
Method MMRM
Comments [Not Specified]
5.Post-Hoc Outcome
Title Change From Baseline To Month 6 In Average Number Of Kidney IC GL-3 Inclusions
Hide Description Renal biopsies were taken at Baseline and Month 6. The specimens were evaluated using virtual microscopy. The annotation, scoring, and adjudication of the kidney histology assessments were performed by the Clinical Pathology Endpoints Committee. The number of kidney IC GL-3 inclusions was assessed by 3 renal pathologists who were blinded to treatment assignments, participants’ data, and biopsy sequence. One pathologist served as annotator and identified the 300 capillaries on up to 8 slides per specimen to be scored. Two pathologists served as scorers; these pathologists completed the blinded paired assessments. Each pathologist served as annotator/adjudicator for 1/3 of the cases. Paired assessments were undertaken at Baseline and Month 6. Assessments were made using digital images. Treatment effect was estimated using the LS mean difference between treatments within the context of the ANCOVA model.
Time Frame Baseline, Month 6
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT-amenable: Randomized participants with amenable mutations who received study drug during Stage 1. Amenable mutations are mutant forms of α Gal-A amenable to migalastat. Amenable mutations based on the GLP HEK assay. Participants were analyzed according to their original randomized treatment group.
Arm/Group Title Migalastat
Hide Arm/Group Description:
Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1).
Overall Number of Participants Analyzed 25
Mean (Standard Deviation)
Unit of Measure: kidney IC GL-3 inclusions
-0.250  (0.5126)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Migalastat
Comments The change from Baseline in the average number of kidney ICs was analyzed using an ANCOVA model with covariate adjustment for the baseline value and factors for treatment group and the treatment by baseline interaction.
Type of Statistical Test Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0078
Comments Significant at the 0.010 level
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Difference LSMeans
Estimated Value -0.3
Confidence Interval (2-Sided) 95%
-0.6 to -0.1
Estimation Comments [Not Specified]
Time Frame Adverse events (AEs) were monitored up to 25 months (±7 days) after the first dose of study drug. AEs were reported from the first dose of study medication at Baseline (Visit 1) in Stage 1 to 1 month (±7 days) after the date of the last study visit, whether during Stage 1, Stage 2, or the optional OLE, or after premature discontinuation from the study.
Adverse Event Reporting Description Stage 1 AEs were any AEs that started between the first dose of Stage 1 and the first dose of Stage 2 (or last Stage 1 administration for participants that discontinued during Stage 1). Stage 2 AEs were any AEs that started between the first dose of Stage 2 and the first dose of the OLE (or the last Stage 2 administration for participants that discontinued during Stage 2). OLE AEs were any AEs that started after the first dose of the OLE.
 
Arm/Group Title Migalastat (0-6 Months) Placebo (0-6 Months) All Migalastat (>6-12 Months) All Migalastat (>12-24 Months)
Hide Arm/Group Description Migalastat 150-mg capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. Placebo capsule given orally QOD during the 6-month, double-blind, randomized placebo-controlled treatment period (Stage 1). After Stage 1, participants progressed to Stage 2 where they received open-label migalastat for 6 months. Migalastat 150-mg capsule given orally QOD during the 6-month open-label treatment period (Stage 2). Includes all participants who progressed from both the migalastat (0-6 Months) and the placebo (0-6 Months) Stage 1 treatment groups. After Stage 2, participants were eligible to enter the optional, 12-month OLE. Migalastat 150-mg capsule given orally QOD for up to 12 months during the optional OLE. Includes all participants who decided to continue treatment after completing the 6-month, open-label Stage 2 treatment period.
All-Cause Mortality
Migalastat (0-6 Months) Placebo (0-6 Months) All Migalastat (>6-12 Months) All Migalastat (>12-24 Months)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Migalastat (0-6 Months) Placebo (0-6 Months) All Migalastat (>6-12 Months) All Migalastat (>12-24 Months)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/34 (5.88%)   4/33 (12.12%)   5/63 (7.94%)   11/57 (19.30%) 
Cardiac disorders         
Palpitations  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Ventricular Tachycardia  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Gastrointestinal disorders         
Abdominal Pain Lower  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Constipation  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
General disorders         
Fatigue  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Malaise  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Non-cardiac chest pain  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Infections and infestations         
Bacterial Infection  1  0/34 (0.00%)  1/33 (3.03%)  0/63 (0.00%)  0/57 (0.00%) 
Helicobacter Gastritis  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Meningitis Viral  1  0/34 (0.00%)  1/33 (3.03%)  0/63 (0.00%)  0/57 (0.00%) 
Injury, poisoning and procedural complications         
Multiple Fractures  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Post Procedural Haematoma  1  1/34 (2.94%)  0/33 (0.00%)  0/63 (0.00%)  0/57 (0.00%) 
Post Procedural Haemorrhage  1  0/34 (0.00%)  1/33 (3.03%)  0/63 (0.00%)  0/57 (0.00%) 
Musculoskeletal and connective tissue disorders         
Bone Cyst  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)         
Anaplastic Large Cell Lymphoma T - and Null - Cell Types  1  0/34 (0.00%)  1/33 (3.03%)  0/63 (0.00%)  0/57 (0.00%) 
Nervous system disorders         
Amyotrophic Lateral Sclerosis  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Cerebral Haemorrhage  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Syncope  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Paraesthesia  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Transient Ischaemic Attack  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Psychiatric disorders         
Bulimia Nervosa  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Renal and urinary disorders         
Hydronephrosis  1  1/34 (2.94%)  0/33 (0.00%)  0/63 (0.00%)  0/57 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Pneumothorax  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  1/57 (1.75%) 
Pulmonary Embolism  1  0/34 (0.00%)  0/33 (0.00%)  2/63 (3.17%)  0/57 (0.00%) 
Vascular disorders         
Deep Vein Thrombosis  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Migalastat (0-6 Months) Placebo (0-6 Months) All Migalastat (>6-12 Months) All Migalastat (>12-24 Months)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   31/34 (91.18%)   30/33 (90.91%)   50/63 (79.37%)   46/57 (80.70%) 
Cardiac disorders         
Atrial Fibrillation  1  2/34 (5.88%)  0/33 (0.00%)  1/63 (1.59%)  3/57 (5.26%) 
Ear and labyrinth disorders         
Vertigo  1  2/34 (5.88%)  3/33 (9.09%)  3/63 (4.76%)  0/57 (0.00%) 
Gastrointestinal disorders         
Abdominal Pain Upper  1  2/34 (5.88%)  0/33 (0.00%)  2/63 (3.17%)  2/57 (3.51%) 
Constipation  1  2/34 (5.88%)  2/33 (6.06%)  1/63 (1.59%)  2/57 (3.51%) 
Dry Mouth  1  2/34 (5.88%)  2/33 (6.06%)  0/63 (0.00%)  0/57 (0.00%) 
Diarrhoea  1  3/34 (8.82%)  1/33 (3.03%)  5/63 (7.94%)  2/57 (3.51%) 
Nausea  1  4/34 (11.76%)  2/33 (6.06%)  2/63 (3.17%)  3/57 (5.26%) 
Vomiting  1  1/34 (2.94%)  2/33 (6.06%)  1/63 (1.59%)  3/57 (5.26%) 
Abdominal Distension  1  2/34 (5.88%)  1/33 (3.03%)  2/63 (3.17%)  0/57 (0.00%) 
General disorders         
Fatigue  1  4/34 (11.76%)  4/33 (12.12%)  2/63 (3.17%)  3/57 (5.26%) 
Pyrexia  1  4/34 (11.76%)  1/33 (3.03%)  1/63 (1.59%)  0/57 (0.00%) 
Asthenia  1  2/34 (5.88%)  1/33 (3.03%)  0/63 (0.00%)  1/57 (1.75%) 
Infections and infestations         
Bronchitis  1  1/34 (2.94%)  0/33 (0.00%)  1/63 (1.59%)  6/57 (10.53%) 
Influenza  1  0/34 (0.00%)  3/33 (9.09%)  0/63 (0.00%)  2/57 (3.51%) 
Nasopharyngitis  1  6/34 (17.65%)  2/33 (6.06%)  5/63 (7.94%)  3/57 (5.26%) 
Upper Respiratory Tract Infection  1  2/34 (5.88%)  3/33 (9.09%)  5/63 (7.94%)  3/57 (5.26%) 
Urinary Tract Infection  1  2/34 (5.88%)  0/33 (0.00%)  3/63 (4.76%)  5/57 (8.77%) 
Cystitis  1  2/34 (5.88%)  0/33 (0.00%)  0/63 (0.00%)  0/57 (0.00%) 
Injury, poisoning and procedural complications         
Overdose  1  2/34 (5.88%)  1/33 (3.03%)  0/63 (0.00%)  0/57 (0.00%) 
Procedural Pain  1  2/34 (5.88%)  1/33 (3.03%)  7/63 (11.11%)  0/57 (0.00%) 
Post Procedural Complication  1  2/34 (5.88%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Investigations         
Weight Increased  1  2/34 (5.88%)  1/33 (3.03%)  0/63 (0.00%)  0/57 (0.00%) 
Metabolism and nutrition disorders         
Vitamin D Deficiency  1  1/34 (2.94%)  1/33 (3.03%)  1/63 (1.59%)  3/57 (5.26%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/34 (0.00%)  2/33 (6.06%)  4/63 (6.35%)  5/57 (8.77%) 
Muscle Spasms  1  1/34 (2.94%)  3/33 (9.09%)  2/63 (3.17%)  1/57 (1.75%) 
Back Pain  1  3/34 (8.82%)  0/33 (0.00%)  2/63 (3.17%)  3/57 (5.26%) 
Myalgia  1  2/34 (5.88%)  1/33 (3.03%)  1/63 (1.59%)  2/57 (3.51%) 
Pain In Extremity  1  0/34 (0.00%)  4/33 (12.12%)  2/63 (3.17%)  2/57 (3.51%) 
Torticollis  1  2/34 (5.88%)  0/33 (0.00%)  0/63 (0.00%)  0/57 (0.00%) 
Nervous system disorders         
Dizziness  1  2/34 (5.88%)  1/33 (3.03%)  2/63 (3.17%)  1/57 (1.75%) 
Paraesthesia  1  4/34 (11.76%)  4/33 (12.12%)  4/63 (6.35%)  2/57 (3.51%) 
Headache  1  12/34 (35.29%)  7/33 (21.21%)  9/63 (14.29%)  6/57 (10.53%) 
Hypoaesthesia  1  2/34 (5.88%)  0/33 (0.00%)  2/63 (3.17%)  0/57 (0.00%) 
Psychiatric disorders         
Depression  1  1/34 (2.94%)  0/33 (0.00%)  3/63 (4.76%)  4/57 (7.02%) 
Insomnia  1  3/34 (8.82%)  2/33 (6.06%)  0/63 (0.00%)  0/57 (0.00%) 
Renal and urinary disorders         
Proteinuria  1  0/34 (0.00%)  0/33 (0.00%)  1/63 (1.59%)  9/57 (15.79%) 
Haematuria  1  3/34 (8.82%)  0/33 (0.00%)  1/63 (1.59%)  0/57 (0.00%) 
Microalbuminuria  1  0/34 (0.00%)  0/33 (0.00%)  0/63 (0.00%)  3/57 (5.26%) 
Respiratory, thoracic and mediastinal disorders         
Epistaxis  1  3/34 (8.82%)  1/33 (3.03%)  1/63 (1.59%)  0/57 (0.00%) 
Cough  1  3/34 (8.82%)  0/33 (0.00%)  1/63 (1.59%)  2/57 (3.51%) 
Oropharyngeal Pain  1  3/34 (8.82%)  2/33 (6.06%)  0/63 (0.00%)  2/57 (3.51%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 15.0
During Phase 3, prior to unblinding, the assay used for enrollment was validated (GLP HEK assay). Mutant forms of α-Gal A that met assay criteria were categorized as amenable; additional analyses were subsequently performed on this target population.
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 investigator can publish only the results from this trial, provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review prior to submission for publication. If requested and prior to publication, the investigator will remove information deemed confidential or proprietary by the sponsor and will withhold publication for an additional period of time to allow the sponsor to take appropriate measures to establish and preserve its proprietary rights.
Results Point of Contact
Name/Title: Medical Affairs
Organization: Amicus Therapeutics
Phone: +1-877-426-4287 (877-4-AMICUS)
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT00925301     History of Changes
Other Study ID Numbers: AT1001-011
FACETS ( Other Identifier: Amicus Therapeutics )
2009-013459-31 ( EudraCT Number )
First Submitted: June 19, 2009
First Posted: June 22, 2009
Results First Submitted: August 10, 2018
Results First Posted: October 30, 2018
Last Update Posted: October 30, 2018