PF-06939926 Gene Therapy for Duchenne Muscular Dystrophy

Phase 1

Waitlist Available

Research Sponsored by Pfizer

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age as follows, based on ambulatory status: FOR AMBULATORY PARTICIPANTS: Between 4 and 12 years, FOR NON-AMBULATORY PARTICIPANTS: No age restrictions so long as loss of ambulation occurs prior to the subject's 17th birthday; Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing; Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry; Ability to tolerate magnetic resonance imaging (MRI) without sedation and with no contraindications; Ability to tolerate muscle biopsies under anesthesia with no contraindications; Body weights as follows, based on ambulatory status: FOR AMBULATORY PARTICIPANTS: Between 15 kg and 50 kg, FOR NON-AMBULATORY PARTICIPANTS: Less than 75 kg; Functional performance as follows, based on ambulatory status: FOR AMBULATORY PARTICIPANTS: Ability to rise from floor within seven (7) seconds, FOR NON-AMBULATORY PARTICIPANTS: Percent predicted forced vital capacity greater than 40% as part of pulmonary function tests, as well as adequate upper limb function; > 8 years of age for Sirolimus Cohort

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up to 1 year post dose of pf-06939926

Awards & highlights

No Placebo-Only Group

Summary

This trial is a safety and tolerability study of a new drug for Duchenne muscular dystrophy. The drug will be given to 22 people, some of whom will also take sirolimus. The trial will be conducted in two phases, with a review by an external data monitoring committee before progressing to the next phase.

Who is the study for?

This trial is for boys with Duchenne muscular dystrophy (DMD), both walking and non-walking, who can handle MRI scans without sedation. Participants must meet specific genetic criteria, have a certain level of muscle function, and be on stable glucocorticoids. Boys should weigh under 50 kg if walking or under 75 kg if not. Those in the Sirolimus Cohort must be over 8 years old.

What is being tested?

The study tests PF-06939926 gene therapy's safety in DMD patients by giving a single IV dose to about 22 participants. It includes staggered enrollment and reviews by an external committee before increasing doses. The trial also measures dystrophin levels, muscle strength, quality, and function.

What are the potential side effects?

Specific side effects are not listed but may include reactions related to immune response against the viral vector used for gene therapy or general risks associated with intravenous infusions.