Status:
TERMINATED
Study of Ataluren (PTC124) in Hemophilia A and B
Lead Sponsor:
PTC Therapeutics
Collaborating Sponsors:
Genzyme, a Sanofi Company
Conditions:
Hemophilia A
Hemophilia B
Eligibility:
MALE
18+ years
Phase:
PHASE2
Brief Summary
Hemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blo...
Detailed Description
In this study, participants with hemophilia A or hemophilia B due to a nonsense mutation were treated with an investigational drug called ataluren (PTC124). Evaluation procedures to determine if a par...
Eligibility Criteria
Inclusion
- Ability to provide written informed consent
- Age ≥18 years
- Presence of a nonsense mutation as the sole disease-causing mutation in the FVIII or FIX gene
- At least 20 prior treatments with FVIII or FIX concentrates
- Willingness and ability to comply with scheduled visits, drug administration plan, study restrictions, and study procedures
Exclusion
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Any history of prior anti-FVIII/FIX inhibitors
- Unable or unwilling to forego prophylactic FVIII/FIX concentrate use during the screening and on-study periods (Note: Participants were allowed use of FVIII/FIX concentrates for treatment of bleeding episodes while on study)
Key Trial Info
Start Date :
October 14 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 30 2011
Estimated Enrollment :
13 Patients enrolled
Trial Details
Trial ID
NCT00947193
Start Date
October 14 2009
End Date
August 30 2011
Last Update
June 16 2020
Active Locations (13)
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1
The Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States, 61614
2
St. Vincent Indianapolis Hospital
Indianapolis, Indiana, United States, 46260
3
New England Hemophilia Center
Worcester, Massachusetts, United States, 01605
4
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229