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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00283933
• Date of registration: 27/01/2006
• Prospective Registration: Yes
• Primary sponsor: Amicus Therapeutics
• Public title: A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
• Scientific title: A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease
• Date of first enrolment: May 9, 2006
• Target sample size: 5
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00283933
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Canada, France, United Kingdom

Contacts

• Name: Medical Monitor, Clinical Research
• Affiliation: Amicus Therapeutics

Key inclusion & exclusion criteria

Inclusion Criteria:

- Males between 18 and 65 years of age (inclusive)

- Hemizygous for Fabry disease

- Had a confirmed diagnosis of Fabry disease with a documented missense gene mutation
(individual or familial)

- Had enhanceable enzyme activity based on in vitro tests

- Had documented evidence of cardiac and/or renal dysfunction (for example, abnormal
electrocardiogram (ECG), left ventricular hypertrophy, renal insufficiency)

- Were previously untreated by enzyme replacement therapy (ERT) or substrate depletion
for Fabry disease, or if ERT or other specific treatment for Fabry disease was
administered, were able to stop ERT for at least 30 weeks.

- Were willing to undergo 2 kidney and 3 skin biopsies

- Agreed to be sexually abstinent or use a condom with spermicide when engaging in
sexual activity during the course of the study and for a period of 30 days following
completion of the study

- Were willing and able to sign an informed consent form

Exclusion Criteria:

- History of significant disease other than Fabry disease (for example, end-stage renal
disease; Class III or IV heart disease [per the New York Heart Association
classification]; current diagnosis of cancer, except for basal cell carcinoma of the
skin; diabetes [unless hemoglobin A1c =8]; or neurological disease that would have
impaired the participant's ability to participate in the study)

- History of organ transplant

- Serum creatinine >176 millimole per deciliter on Day -2

- Screening 12-lead ECG demonstrating corrected QT interval >450 milliseconds prior to
dosing

- Taking a medication prohibited by the protocol: Fabrazyme® (agalsidase beta),
Replagal™ (agalsidase alfa), Glyset® (miglitol), Zavesca® (miglustat), or any
experimental therapy for any indication

- Participated in a previous clinical trial in the last 30 days

- Any other condition, which, in the opinion of the investigator, would jeopardize the
safety of the participant or impact the validity of the study results



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: migalastat HCl

Primary Outcome(s)

Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) [Time Frame: Day 1 (after dosing) through Week 48]

Secondary Outcome(s)

a-Galactosidase A (a-Gal A) Activity In Peripheral Blood Mononuclear Cells (PBMC) At Baseline, Week 24, And Week 48 [Time Frame: Baseline, Week 24 (end of treatment period), Week 48 (end of extension period)]

Secondary ID(s)

FAB-CL-203 (AA1565522)

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 07/09/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00283933