Study of Ataluren (PTC124®) in Hemophilia A and B
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00947193|
Recruitment Status : Terminated
First Posted : July 27, 2009
Last Update Posted : May 7, 2019
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia A Hemophilia B||Drug: Ataluren||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 2a Study of Ataluren (PTC124) as an Oral Treatment for Nonsense-Mutation-Mediated Hemophilia A and B|
|Actual Study Start Date :||October 14, 2009|
|Actual Primary Completion Date :||August 30, 2011|
|Actual Study Completion Date :||August 30, 2011|
The dose level of Ataluren will be 10 mg/kg in the morning,10 mg/kg at midday, and 20 mg/kg in the evening for 14 days.
Ataluren (PTC124) will be provided as a vanilla-flavored powder to be mixed with water or milk. Ataluren (PTC124) will be taken 3 times per day, with dosing based on the participant's body weight. The dose level for ataluren (PTC124) will be 10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for 14 days.
Other Name: PTC124
- Pharmacological effect as measured by plasma FVIII/FIX activity [ Time Frame: 1.5 months ]The proportion of participants with a plasma FVIII/FIX activity response; a plasma FVIII/FIX activity response is defined as an end-of-treatment activity of ≥1% as determined by functional assay.
- Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs) by Severity and Relationship to Study Drug [ Time Frame: 1.5 months ]The relationship of TEAEs and SAEs to the study drugs will be assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0, as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life-threatening), or Grade 5 (fatal). A summary of serious and all other non-serious adverse events, regardless of causality, will be located in the Reported Adverse Events module.
- Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory Parameter [ Time Frame: 1.5 months ]Number of participants with an abnormal clinical laboratory parameter considered clinically meaningful by the Investigator will be reported. Clinical laboratory tests included hematology, biochemistry, and urinalysis. A summary of serious and all other non-serious adverse events, regardless of causality, will be located in the Reported Adverse Events module.
- Compliance with ataluren (PTC124) administration [ Time Frame: 1.5 Months ]Ataluren compliance as assessed by quantification of used and unused drug.
- Ataluren (PTC124) plasma exposure [ Time Frame: 1.5 months ]Steady-state ataluren plasma concentrations before and 2 hours after the first morning dose at end of treatment
- Occurrence of bleeding episodes [ Time Frame: 1.5 months ]Frequency, timing, anatomic location, and severity of any bleeding episodes
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00947193
|United States, Illinois|
|The Bleeding and Clotting Disorders Institute|
|Peoria, Illinois, United States, 61614|
|United States, Indiana|
|St. Vincent Indianapolis Hospital|
|Indianapolis, Indiana, United States, 46260|
|United States, Massachusetts|
|New England Hemophilia Center|
|Worcester, Massachusetts, United States, 01605|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229|
|United States, Pennsylvania|
|Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|United States, Tennessee|
|Vanderbilt Hemostatis and Thrombosis Clinic|
|Nashville, Tennessee, United States, 37232|
|United States, Washington|
|Puget Sound Blood Center|
|Seattle, Washington, United States, 98104|
|Canada, British Columbia|
|St. Paul's Hospital|
|Vancouver, British Columbia, Canada, V6Z 1Y6|
|Lille Cedex, France|
|Hôpital Edouard Herriot|
|Lyon Cedex, France|
|Hôpital Necker Enfants Malades|
|Azienda Ospedaliero-Universitaria Careggi Viale G.B. Morgagni|
|A.Bianchi Bonomi Hemophilia and Thrombosis Center|
|Principal Investigator:||Jay Barth, MD||PTC Therapeutics|