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A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

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ClinicalTrials.gov Identifier: NCT00283959
Recruitment Status : Completed
First Posted : January 31, 2006
Results First Posted : September 7, 2018
Last Update Posted : October 31, 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 4
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Migalastat
Hide Arm/Group Description Participants received migalastat 150 milligrams (mg) orally every other day (QOD) during the 12-week treatment period and the optional 36-week extension period.
Period Title: Treatment Period
Started 4
Safety Population [1] 4
Pharmacodynamics (PD) Population [2] 4
Completed 4
Not Completed 0
[1]
All enrolled participants who received at least 1 dose of study drug
[2]
Received study drug and had a non-missing baseline and postbaseline PD parameter recorded
Period Title: Extension Period
Started 4
Completed 3
Not Completed 1
Reason Not Completed
Low Compliance             1
Arm/Group Title Migalastat
Hide Arm/Group Description Participants received migalastat 150 mg orally QOD during the 12-week treatment period and the optional 36-week extension period.
Overall Number of Baseline Participants 4
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 4 participants
33.3  (21.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants
Female
0
   0.0%
Male
4
 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 a 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 (end of extension period)
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:
Participants received migalastat 150 mg orally QOD during the 12-week treatment period and the optional 36-week extension period.
Overall Number of Participants Analyzed 4
Measure Type: Count of Participants
Unit of Measure: Participants
1
  25.0%
2.Secondary Outcome
Title α-Galactosidase A (α-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 12, And Week 48
Hide Description

PBMCs were isolated from whole blood and lysed, and α-Gal A activity was measured by 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 PBMCs are presented by individual participants. Values of "0" presented below represent α-Gal A activity levels that were below the lower limit of quantification.

Time Frame Baseline, Week 12 (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. Participants who discontinued prior to the specified time point were not analyzed because no data were available.
Arm/Group Title Migalastat
Hide Arm/Group Description:
Participants received migalastat 150 mg orally QOD during the 12-week treatment period and the optional 36-week extension period.
Overall Number of Participants Analyzed 4
Measure Type: Number
Unit of Measure: nmol 4-MU/hr/mg protein
Participant 1 Baseline Number Analyzed 1 participants
0.14
Participant 1 Week 12 Number Analyzed 1 participants
NA [1] 
Participant 1 Week 48 Number Analyzed 1 participants
0.12
Participant 2 Baseline Number Analyzed 1 participants
0.24
Participant 2 Week 12 Number Analyzed 1 participants
2.3
Participant 2 Week 48 Number Analyzed 0 participants
Participant 3 Baseline Number Analyzed 1 participants
0.21
Participant 3 Week 12 Number Analyzed 1 participants
2.43
Participant 3 Week 48 Number Analyzed 0 participants
Participant 4 Baseline Number Analyzed 1 participants
0.3
Participant 4 Week 12 Number Analyzed 1 participants
7.36
Participant 4 Week 48 Number Analyzed 1 participants
6.06
[1]
Data for this participant were below the limit of quantification.
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 Participants received migalastat 150 mg orally QOD during the 12-week treatment period and the optional 36-week extension period.
All-Cause Mortality
Migalastat
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Migalastat
Affected / at Risk (%)
Total   1/4 (25.00%) 
Cardiac disorders   
Atrioventricular block  1  1/4 (25.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 8.0
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   3/4 (75.00%) 
Cardiac disorders   
Atrial Fibrillation  1  1/4 (25.00%) 
Bifascicular block  1  1/4 (25.00%) 
Bundle branch block left  1  1/4 (25.00%) 
Gastrointestinal disorders   
Abdominal Pain Upper  1  2/4 (50.00%) 
Diarrhoea  1  1/4 (25.00%) 
Nausea  1  1/4 (25.00%) 
Abdominal discomfort  1  1/4 (25.00%) 
Abdominal distension  1  1/4 (25.00%) 
Dyspepsia  1  1/4 (25.00%) 
General disorders   
Oedema Peripheral  1  1/4 (25.00%) 
Pyrexia  1  1/4 (25.00%) 
Fatigue  1  1/4 (25.00%) 
Pain  1  1/4 (25.00%) 
Infections and infestations   
Nasopharyngitis  1  1/4 (25.00%) 
Investigations   
Blood thyroid stimulating hormone increased  1  1/4 (25.00%) 
QRS axis abnormal  1  1/4 (25.00%) 
Venous pressure jugular increased  1  1/4 (25.00%) 
Musculoskeletal and connective tissue disorders   
Musculoskeletal stiffness  1  1/4 (25.00%) 
Pain in extremity  1  1/4 (25.00%) 
Psychiatric disorders   
Nervousness  1  1/4 (25.00%) 
Renal and urinary disorders   
Proteinuria  1  1/4 (25.00%) 
Renal pain  1  1/4 (25.00%) 
Skin and subcutaneous tissue disorders   
Nail discolouration  1  1/4 (25.00%) 
Vascular disorders   
Poor peripheral circulation  1  1/4 (25.00%) 
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: NCT00283959     History of Changes
Other Study ID Numbers: FAB-CL-202 (AA1565521)
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: October 31, 2018