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Duchenne Muscular Dystrophy-Phase 1b Multicenter - Healing Genes
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Duchenne Muscular Dystrophy-Phase 1b Multicenter

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A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

A Phase 1b Multicenter, Open-label, Single Ascending Dose Study To Evaluate The Safety And Tolerability Of Pf-06939926 In Ambulatory Subjects With Duchenne Muscular Dystrophy


Phase 1 / 2

DESCRIPTION:
Doctors at 20 research centers in the U.S. seek patients with Duchenne Muscular Dystrophy (DMD) to trial an investigatory gene therapy, a virus engineered to transfer a functional micro-dystrophin gene to the patient, so that their bodies can produce normal dystrophin and normal muscle fibers.

Participants will undergo a single IV infusion of the investigatory gene therapy at one of 2 dose levels,, then be assessed at treatment time, baseline, 2 months and 1 year post-treatment, with a total follow-up period of 5 years.


PATIENT MUST:

  • Be male, 5 to 12 years of age
  • Have a clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype
  • Have body weight between 15 and 50 kg
  • Not have received live attenuated vaccination within 3 months prior or exposure to a systemic antiviral and/or interferon therapy within 30 days prior to receipt of PF-06939926

THE STUDY INVOLVES:

  1. Screening to confirm eligibility.
  2. Participants receive a single infusion of the investigatory gene therapy.
  3. Lab and biopsy assessments will continue for 12 months, with long-term follow up for 5 years.

LOCATIONS AND CONTACTS:

There are 20 study locations in the United States. Find the closest to you here.

Contacts:
Pfizer CT.gov Call Center  |  1-800-718-1021  |  [email protected]

SPONSOR INFORMATION:
Pfizer

Or go online:
https://clinicaltrials.gov/ct2/show/NCT03362502

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