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A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

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ClinicalTrials.gov Identifier: NCT00283933
Recruitment Status : Completed
First Posted : January 31, 2006
Results First Posted : September 7, 2018
Last Update Posted : September 7, 2018
Sponsor:
Information provided by (Responsible Party):
Amicus Therapeutics

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Fabry Disease
Intervention Drug: migalastat HCl
Enrollment 5
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Migalastat
Hide Arm/Group Description Migalastat 150 milligrams (mg) was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
Period Title: Treatment Period
Started 5
Safety Population [1] 5
Pharmacodynamic (PD) Population [2] 5
Completed 5
Not Completed 0
[1]
Received at least 1 dose of study drug
[2]
Received study drug and had at least 1 non-missing postbaseline PD parameter recorded
Period Title: Extension Period
Started 5
Completed 5
Not Completed 0
Arm/Group Title Migalastat
Hide Arm/Group Description Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
Overall Number of Baseline Participants 5
Hide Baseline Analysis Population Description
Safety Population: All participants who received at least 1 dose of study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 5 participants
41.6  (9.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 5 participants
Female
0
   0.0%
Male
5
 100.0%
1.Primary Outcome
Title Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
Hide Description TEAEs were defined as any adverse event with start date on or after administration of study drug or pre-existing conditions that worsened on or after the start of the first study drug administration (on Day 1). A severe adverse event was defined as an adverse event that was incapacitating and required medical intervention. The number of participants who experienced one or more severe TEAEs after dosing on Day 1 through Week 48 is presented. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Time Frame Day 1 (after dosing) through Week 48
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: All participants who received at least 1 dose of study drug.
Arm/Group Title Migalastat
Hide Arm/Group Description:
Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
Overall Number of Participants Analyzed 5
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
2.Secondary Outcome
Title α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48
Hide Description PBMC were isolated from whole blood and lysed, and α-Gal A activity was measured using a validated fluorometric assay, with catalysis to fluorescent 4-methylumbelliferone (4-MU) as the activity measure. The activity values obtained were normalized to protein (measured using a colorimetric assay) and reported as enzyme activity (nanomole [nmol] 4-MU/hour [hr]) per mg of protein. On Day 1 of the first visit and at every visit thereafter, the samples were collected prior to dosing with migalastat. α-Gal A activity in leukocytes are presented by individual participants.
Time Frame Baseline, Week 24 (end of treatment period), Week 48 (end of extension period)
Hide Outcome Measure Data
Hide Analysis Population Description
PD Population: All participants who received at least 1 dose of study drug and had at least 1 non-missing postbaseline PD parameter recorded.
Arm/Group Title Migalastat
Hide Arm/Group Description:
Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
Overall Number of Participants Analyzed 5
Measure Type: Number
Unit of Measure: nmol 4-MU/hr/mg protein
Participant 1: Baseline Number Analyzed 1 participants
0.05
Participant 1: Week 24 Number Analyzed 1 participants
0.36
Participant 1: Week 48 Number Analyzed 1 participants
0.1
Participant 2: Baseline Number Analyzed 1 participants
0.06
Participant 2: Week 24 Number Analyzed 1 participants
0.13
Participant 2: Week 48 Number Analyzed 1 participants
0.1
Participant 3: Baseline Number Analyzed 1 participants
0.14
Participant 3: Week 24 Number Analyzed 1 participants
0.3
Participant 3: Week 48 Number Analyzed 1 participants
0.33
Participant 4: Baseline Number Analyzed 1 participants
3.4
Participant 4: Week 24 Number Analyzed 1 participants
10.9
Participant 4: Week 48 Number Analyzed 1 participants
7.4
Participant 5: Baseline Number Analyzed 1 participants
0.18
Participant 5: Week 24 Number Analyzed 1 participants
1.32
Participant 5: Week 48 Number Analyzed 1 participants
0.13
Time Frame Day 1 after dosing through Week 48 (end of extension period)
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Migalastat
Hide Arm/Group Description Migalastat 150 mg was administered orally QOD during the 24-week treatment period and then during the optional 24-week extension period.
All-Cause Mortality
Migalastat
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Migalastat
Affected / at Risk (%)
Total   0/5 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Migalastat
Affected / at Risk (%)
Total   5/5 (100.00%) 
Cardiac disorders   
Ventricular tachycardia  1  1/5 (20.00%) 
Gastrointestinal disorders   
Diarrhoea  1  1/5 (20.00%) 
Infections and infestations   
Gastroenteritis  1  1/5 (20.00%) 
Nasopharyngitis  1  1/5 (20.00%) 
Injury, poisoning and procedural complications   
Contusion  1  1/5 (20.00%) 
Muscle strain  1  1/5 (20.00%) 
Investigations   
Gamma-glutamyltransferase increased  1  1/5 (20.00%) 
Metabolism and nutrition disorders   
Gout  1  1/5 (20.00%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/5 (20.00%) 
Bursitis  1  1/5 (20.00%) 
Joint swelling  1  1/5 (20.00%) 
Nervous system disorders   
Dizziness  1  1/5 (20.00%) 
Headache  1  2/5 (40.00%) 
Renal and urinary disorders   
Haematuria  1  1/5 (20.00%) 
Proteinuria  1  2/5 (40.00%) 
Reproductive system and breast disorders   
Scrotal mass  1  1/5 (20.00%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/5 (20.00%) 
Skin and subcutaneous tissue disorders   
Pruritis  1  1/5 (20.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.0
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 only publish the results from this trial provided they supply the sponsor (or authorized entity) a copy of any proposed publication for review. If requested, 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
Layout table for Results Point of Contact information
Name/Title: Amicus Therapeutics
Organization: Medical Affairs
Phone: +1-877-426-4287 (877-4-AMICUS)
EMail: MedInfoUSA@amicusrx.com
Layout table for additonal information
Responsible Party: Amicus Therapeutics
ClinicalTrials.gov Identifier: NCT00283933     History of Changes
Other Study ID Numbers: FAB-CL-203 (AA1565522)
First Submitted: January 27, 2006
First Posted: January 31, 2006
Results First Submitted: August 10, 2018
Results First Posted: September 7, 2018
Last Update Posted: September 7, 2018