PTC Therapeutics has initiated a long-term clinical trial with the investigational drug ataluren (formerly PTC124®) in patients with advanced nonsense mutation Duchenne and Becker muscular dystrophy (nmDBMD) who have lost the ability to walk independently.

The main goal of the multi-center, open-label trial is to obtain additional information about the long-term safety of ataluren. The study will also evaluate the use of several outcome measures of physical, heart, and lung function in nonambulatory patients.

The study will enroll about 30 boys and young men with advanced nmDBMD at five trial sites in the United States and one site in the United Kingdom. Enrollment will be divided to ensure evaluation of about 15 participants who are receiving corticosteroid therapy every day and of about 15 participants who are not receiving any corticosteroid therapy. Participants will receive ataluren for 48 weeks. During the first 12 weeks of the study, participants will need to visit the clinic every 6 weeks. Thereafter, clinic visits are required every 12 weeks. Additional required laboratory tests may be collected at the research site, at a local laboratory, or at home via a visiting nursing service.

Important Requirements for Taking Part in this Study
To be eligible for this trial, patients must have had a genetic test (a DNA test, usually of a blood sample), to evaluate the dystrophin gene and determine that a nonsense mutation is present. Patients must also meet the following criteria:

• Be at least 7 years of age
• Have been unable to walk independently for at least one year before the start of study
• Have adequate shoulder and arm mobility to perform trial procedures requiring the arms and hands, such as placing a large peg in a hole
• Have the ability to provide evaluable measurements of heart and lung function
• Have normal or near normal blood tests for liver, adrenal gland, and kidney function
• Not require daytime ventilator assistance
• Not be taking warfarin (Coumadin) or phenytoin (Dilantin)

Other eligibility requirements will be explained at the trial site.

Other Resources
• Trial Sites
• Frequently Asked Questions
• Clinicaltrials.gov Overview
• Ataluren Overview
• Genetic Testing

Additional details about this ataluren clinical trial can be found at www.clinicaltrials.gov or by contacting PTC Therapeutics Patient and Professional Advocacy at (866) 282-5873 (toll free in the United States), (908) 912-9256, or PatientInfo@ptcbio.com.