Phase 2
N=9
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Fabry Disease
Bottom Line
View on ClinicalTrials.gov: NCT00214500 ↗Enrolled (actual)
9
Serious AEs
0.0%
Results posted
Sep 2018
Primary outcome: Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- migalastat HCl (Drug)
- Age
- Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Amicus Therapeutics
- Primary completion
- Jan 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) |
2 | — |
| SECONDARY PK: Area Under The Concentration Versus Time Curve (AUC) After Administration Of Migalastat |
1052.96; 4217.95; 10880.66; 1360.69; 5643.50; 12244.47 | — |
| SECONDARY α-Galactosidase A (α-Gal A) Activity In Leukocytes At Baseline, Week 12, And Week 96 |
5.2; 17.4; 20.3; 4.7; 24.6; 22.8 | — |
Summary
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
Eligibility Criteria
Inclusion Criteria
- Males between 18 and 55 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
- In the judgment of the investigator, were either able to safely suspend ERT throughout the study, or be ERT naive
- 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 >2 mg per deciliter on Day -2
- Screening 12-lead electrocardiogram 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
Data sourced from ClinicalTrials.gov (NCT00214500). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.