PTC124 Clinical Trials

Nonsense-Mutation Duchenne and Becker Muscular Dystrophy (DMD and BMD)

NOW RECRUITING: Phase 2b DMD/BMD Trial
PTC has initiated a Phase 2b clinical study of PTC124 in DMD/BMD.   The trial is a multi-center, randomized, double-blind, placebo-controlled study to determine whether PTC124 can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.

To be eligible for the trial you must:

• Have DMD or BMD due to a nonsense mutation. A DNA blood test known as a sequence analysis or full-length gene sequencing can confirm presence of a nonsense mutation.
• Be able to walk at least 75 meters (80 yards) unassisted in a 6-minute time period.
• Be at least 5 years of age.
• Meet additional eligibility criteria (full eligibility criteria is available at www.clinicaltrials.gov).

Please see the Frequently Asked Questions document for further information about the trial. Each center participating in the study is responsible for evaluating patients to determine whether they meet the eligibility criteria.

The trial will be conducted at more than 35 centers on 4 continents, as shown on this map. Once a center is ready to accept patients, the center's contact information will be posted on the map and on www.clinicaltrials.gov (search on PTC124). Please contact the centers directly to inquire about trial participation.

Information regarding the trial is also available in the following languages:

• Dutch
• French
• German
• Hebrew
• Italian
• Spanish
• Swedish

DMD/BMD Phase 2b Trial Information for Physicians
Physicians who treat DMD/BMD patients and are interested in referring patients to the study should complete the Referring Physician Survey or contact a participating center directly.

Nonsense-Mutation Cystic Fibrosis

NOT YET RECRUITING: Proposed Phase 2b CF Trial
PTC is planning to initiate a longer-term, Phase 2b clinical study of PTC124 in patients with nonsense-mutation CF within the next six months. The main goal of this study will be to understand whether PTC124 can improve how patients with CF due to a nonsense mutation feel and function and whether the drug can safely be given over a long period. The trial will be a multi-center, randomized, double-blind, placebo-controlled study. Additional details will be posted as they become available.

PTC sends updates via email about our trials and other news about PTC. To join the mailing list, visit the "Contact Us" portion of our website and be sure to check "receive PTC updates."