Summary

Eligibility
for males ages 4-7 (full criteria)
Location
at UCLA
Dates
study started
completion around
Principal Investigator
by Perry Shieh (ucla)

Description

Summary

The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study with two thirds of participants assigned to gene therapy. The one third of participants who are randomized to the placebo arm will have an opportunity for treatment with gene therapy at the beginning of the second year.

Official Title

A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

Details

The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in Cohort 1 and receive gene therapy at the start of the study; approximately one third will be in Cohort 2 and receive placebo at the start of the study and receive gene therapy after one year, as long as it remains safe to do so. The treatment (PF-06939926 gene therapy or placebo) will be given as an intravenous infusion lasting up to 2 hours.

The study includes boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.

The primary outcome of the study will be assessed at 52 weeks. All participants will be followed in the study for 5 years after treatment with gene therapy.

The study medication, all medical tests associated with the study, and the visits to the study sites are free of charge. Participants will also be supported for travel costs associated with study visits.

Keywords

Duchenne Muscular Dystrophy, Clinical trial, Gene therapy, fordadistrogene movaparvovec, Muscular Dystrophies

Eligibility

You can join if…

Open to males ages 4-7

  1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing
  2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening
  3. Ambulatory, as assessed by protocol-specified criteria

You CAN'T join if...

  1. Positive test performed by Pfizer for neutralizing antibodies to AAV9
  2. Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™)
  3. Any prior treatment with gene therapy
  4. Any non-healed injury that may impact functional testing (eg NSAA)
  5. Abnormality in specified laboratory tests, including blood counts, liver and kidney function
  6. Any of the following genetic abnormalities in the dystrophin gene:
    1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR
    2. A deletion that affects both exon 29 and exon 30;OR
    3. A deletion that affects any exons between 56-71, inclusive.

Locations

  • MRI Research Center
    Los Angeles California 90095 United States
  • Reed Neurological Research Center
    Los Angeles California 90095 United States
  • Ronald Reagan UCLA Medical Center (Investigational Drug Section)
    Los Angeles California 90095 United States
  • Ronald Reagan UCLA Medical Center - Drug Information Center
    Los Angeles California 90095 United States
  • UCLA (David Geffen School of Medicine)
    Los Angeles California 90095 United States
  • UCLA Children's Heart Center
    Los Angeles California 90095 United States
  • UCLA Clinical Lab Services
    Los Angeles California 90095 United States
  • UCLA Mattel Children's Hospital
    Los Angeles California 90095 United States
  • UCLA Medical Center
    Los Angeles California 90095 United States
  • UCLA Outpatient Surgery Center
    Los Angeles California 90095 United States

Lead Scientist at University of California Health

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Pfizer
Links
To obtain contact information for a study center near you, click here.
ID
NCT04281485
Phase
Phase 3 Duchenne Muscular Dystrophy Research Study
Study Type
Interventional
Participants
About 122 people participating
Last Updated