A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.

Phase 1

• Conditions: Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A.; MedDRA version: 17.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

• Registration Number: EUCTR2011-004800-40-BE
• Lead Sponsor: Amicus Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-08-29
• Last Update Posted: 8 August 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

1.Subject with Fabry disease who completed treatment in a previous study of migalastat HCl given as monotherapy.

2. Male or female subjects 16 years of age or older.
Note: Subjects under the age of 18 will be enrolled only at sites with all required regulatory and ethics approvals to do so.

3. A female subject is eligible to participate if she is:
A. Of non-childbearing potential, or
B. Of childbearing potential and NOT pregnant or nursing, has a negative
urine pregnancy test at the Baseline Visit (Visit 1), and agrees to one of
the methods of avoiding pregnancy listed in Appendix 1 as per the study protocol from the time of
first dose of study medication until 30 days after study completion.
A female is considered Non-childbearing potential” if she is status-post
hysterectomy, status-post surgical removal of both ovaries, has current, documented tubal ligation, or is postmenopausal and >2 years without
menses. Female subjects who are post-menopausal <2 years must be confirmed menopausal by Follicle Stimulating Hormone (FSH) and estradiol levels.
A female is considered childbearingpotential” if she has functional ovaries, ducts, and uterus with no impairment that would cause sterility. This includes women with oligomenorrhea (even severe), and women who are
perimenopausal or who have just begun to menstruate.

4. Male subjects must agree to use one of the contraception methods listed in Appendix 1. This criterion must be followed from the time of the first dose of study medication until 30 days after study completion.

5. Subject is willing and able to provide written informed consent and authorization for use and disclosure of Personal Health Information (PHI) or has a legally authorized
representative who has given written informed consent.

6. French subjects: In France, a subject will be eligible for inclusion in this study only
if either affiliated to or a beneficiary of a social security category.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. The last available estimated glomerular filtration rate (eGFR) in the previous study
was <30 mL/min/1.73m2; unless there is measured GFR available within 3 months of Baseline Visit (Visit 1), which is >30 mL/min/1.73m2.

2. The subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis.

3. The subject is treated or has been treated with another investigational drug (except migalastat HCl) within 30 days of study start.

4. Subject is unable to comply with study requirements, or deemed otherwise unsuitable for study entry, in the opinion of the investigator.

5. Had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 months before Visit 1.

6. Has clinically significant unstable cardiac disease in the opinion of the investigator (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure).

7. Has a history of allergy or sensitivity to AT1001 (including excipients) or other iminosugars (e.g., miglustat, miglitol).

8. Requires treatment with Glyset® (miglitol) or Zavesca® (miglustat).

9. Has any intercurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator
that the potential subject may have an unacceptable risk by participating in this study.

10. Patients with severe or unsuitable concomitant medical condition (cardiovascular, neurological, hepatic, renal, metabolic, hematological, immunological, pulmonary, or gastrointestinal disorder). The medical monitor or designee must be contacted to discuss the stability of a subject's medical condition(s) and the potential impact of the
condition(s) on trial participation.

11. Patients with clinically significant abnormal laboratory value(s) and clinically significant electrocardiogram (ECG) findings at baseline. The medical monitor or designee must be contacted to discuss the stability of a subject's medical condition(s) and the potential impact of the condition(s) on trial participation.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified