PHASE 3, RANDOMIZED, DOUBLE BLIND, PLACEBO VS PF-06939926 STUDY IN DMD
Research type
Research Study
Full 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
IRAS ID
275551
Contact name
Michela Guglieri
Contact email
Sponsor organisation
Pfizer Inc 235 East 42nd Street, New York, NY 10017
Eudract number
2019-002921-31
Clinicaltrials.gov Identifier
Duration of Study in the UK
6 years, 7 months, 30 days
Research summary
Duchenne Muscular Dystrophy (DMD) is a severe disease of the muscles caused by a mutation in the gene responsible for producing a protein called dystrophin. This leads to muscle degeneration and premature death.
Current glucocorticoid treatment aims to keep the patient independent for as long as possible and prevent complications, however this has many side effects. The 3 approved therapies available to restore dystrophin levels are only applicable to approximately 30% of the DMD population and therefore a therapy for the entire DMD population is needed.
The study drug PF-06939926 is a gene replacement therapy anticipated to restore dystrophin production in the muscles and heart.
This study will assess the safety and efficacy of PF-06939926 compared with placebo (an inactive substance which resembles the study drug but has no medical value) in male participants between ≥4 and <8 years with DMD who are taking glucocorticoids daily.
Approximately 99 participants will be enrolled in the study. Participants will be randomly assigned to one of 2 treatment groups. Cohort 1 will receive a single dose of PF-06939926 on Day 1 and a single dose of placebo at Day 390. Cohort 2 will receive placebo on Day 1 and PF-06939926 at Day 390. Neither the doctor or patient will know what treatment group they have been assigned to.
Participants will be asked to attend clinic approximately 41 times over 5 years for Cohort 1 and 43 times over 6 years for Cohort 2. All participants will be followed by the centre for 5 years after dose of PF-0639926. Study procedures include physical and neurological examinations, vital signs, ECG, blood, urine and saliva samples, ECHO, questionnaires and assessments.
Pfizer is the Sponsor.REC name
North East - York Research Ethics Committee
REC reference
20/NE/0172
Date of REC Opinion
12 Aug 2020
REC opinion
Further Information Favourable Opinion