ong-term safety study of AT1001 in people with Fabry disease
- Conditions
- Fabry DiseaseMedDRA version: 14.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2007-001838-13-GB
- Lead Sponsor
- Amicus Therapeutics, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 0
1.Subject completed the main treatment period of another Phase 2 trial of AT1001 in Fabry disease
2.Women of childbearing potential must have a negative result on their pregnancy test
3.Male and female subjects agree to use reliable methods of contraception during study treatment and for 4 weeks after study treatment termination
4.Subject is willing and able to provide written informed consent
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
1. Subject had a major protocol violation in the preceding AT1001 trial and was discontinued.
2.Subject has undergone, or is scheduled to undergo kidney transplantation or is currently on dialysis
3.Subject is treated or has been treated with another investigational drug (except AT1001) within 30 days of study start
4.Subject has been treated with Fabrazyme (agalsidase beta), Replagal (agalsidase alfa), Glyset (miglitol) or Zavesca(miglustat) within 2 weeks prior to enrollment
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term safety and tolerability of oral AT1001 in patients with Fabry disease;Secondary Objective: To gain information about the pharmacodynamics and pharmacokinetics of orally administered AT1001 in patients with Fabry disease;Timepoint(s) of evaluation of this end point: End of study visit;<br> Primary end point(s): •Treatment emergent marked laboratory abnormalities up to end of study (EOS) visit<br> •Treatment emergent adverse events up to 48 hours after study medication discontinuation <br> •Serious adverse events up to 28 days after study medication discontinuation <br> •Change in concomitant treatments<br>
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): Levels of a-Gal A in leukocytes<br> Levels of GL-3 in plasma and<br> Levels of AT1001 in plasma<br> ;Timepoint(s) of evaluation of this end point: Monitored throughout study