MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED PHASE III STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF-06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Summary description of the study
This study collects information about whether Pfizer's gene therapy is safe and what effect it has on the muscles. The main idea (the theory) of gene therapy is to give back to the body something that is missing or not functioning properly. In the case of DMD, this is dystrophin. However, it is not yet known if this theory works. This study requires that participants visit the study team at the study center very frequently during the first month after receiving the investigational product or placebo, so that their health can be monitored for safety. After that, participants will visit the study center every 3–4 months until the end of the first year. This pattern repeats in the second year. For the remaining 3–4 years until the study is completed, participants will come to the study center twice a year. It is possible that the disease or health status may improve, worsen, or remain the same as a result of participating in this study. There is no guarantee that there will be any benefit.
(BASEC)
Intervention under investigation
Approximately 99 children will participate in this study worldwide. The study will be conducted at about 60 different study centers in approximately 15 countries. The design of this study is called "randomized, double-blind, placebo-controlled." There are two groups: one (with approximately 66 participants) receives the gene therapy PF-06939926 at the beginning of the first year and placebo at the beginning of the second year; the second group (with approximately 33 participants) receives placebo in the first year and gene therapy PF-06939926 in the second year. Participation in this study may last about 5 or 6 years.
(BASEC)
Disease under investigation
DUCHENNE MUSCULAR DYSTROPHY
(BASEC)
To participate in the study, the following conditions must be met: Male participants who are ≥4 and <8 years old at screening (Visit 1). Confirmed diagnosis of DMD by a prior genetic test showing the presence of a mutation in the dystrophin gene consistent with DMD at screening (Visit 1). Receiving a stable daily dose of glucocorticoids. Able to walk. (BASEC)
Exclusion criteria
Patients cannot participate in the study: If they have already received gene therapy. If another investigational product has been used within 30 days prior to the start (or possibly longer if the investigator believes the investigational product is still active in the body). (BASEC)
Trial sites
Bern, Zurich
(BASEC)
Sponsor
Pfizer AG, Schärenmoosstrasse 99, 8052 Zürich
(BASEC)
Contact
Contact Person Switzerland
Silvina Gallo MD, Lead Clinician
+1-800-718-1021
ClinicalTrials.gov_Inquiries@clutterpfizer.com66 Hudson Boulevard East New York, NY 10001 U.S.A
(BASEC)
General Information
Pfizer
(ICTRP)
Scientific Information
Pfizer
(ICTRP)
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Zurich
(BASEC)
Date of authorisation
22.12.2020
(BASEC)
ICTRP Trial ID
NCT04281485 (ICTRP)
Official title (approved by ethics committee)
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 (BASEC)
Academic 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 (ICTRP)
Public title
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy (ICTRP)
Disease under investigation
Duchenne Muscular Dystrophy (ICTRP)
Intervention under investigation
Genetic: PF-06939926Other: PlaceboOther: PlaceboGenetic: PF-06939926 (ICTRP)
Type of trial
Interventional (ICTRP)
Trial design
Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor). (ICTRP)
Inclusion/Exclusion criteria
Key inclusion criteria:
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
Key exclusion criteria:
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 30OR
3. A deletion that affects any exons between 56-71, inclusive. (ICTRP)
not available
Primary and secondary end points
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 (ICTRP)
Change From Baseline in Percent Normal Dystrophin Expression Level in Muscle Biopsies by Liquid Chromatography Mass Spectrometry (LC-MS) Based on LLQV Peptide at Week 52;Change From Baseline in Percent of Muscle Fibers Expressing Mini-Dystrophin in Muscle Biopsies by Immunofluorescence at Week 52;Change From Baseline in Serum Creatine Kinase (CK) Concentration at Week 52;Least Square Mean of Proportion of Skills Gained Based on the Individual Items of the NSAA at Week 52;Least Square Mean of Proportion of Skills Either Improved or Maintained Based on the Individual Items of the NSAA at Week 52;Change From Baseline in 10 Meter Run/Walk Velocity at Week 52;Change From Baseline in Rise From Floor Velocity at Week 52;Change From Baseline in Modified Pediatric Outcome Data Collection Instrument (PODCI)- Transfer and Basic Mobility Core Scale at Week 52;Change From Baseline in Modified PODCI- Sports and Physical Functioning Core Scale at Week 52 (ICTRP)
Registration date
not available
Incorporation of the first participant
not available
Secondary sponsors
not available
Additional contacts
Pfizer CT.gov Call Center, Pfizer (ICTRP)
Secondary trial IDs
2023-508510-42-00, C3391003 (ICTRP)
Results-Individual Participant Data (IPD)
not available
Further information on the trial
https://clinicaltrials.gov/ct2/show/NCT04281485 (ICTRP)
Results of the trial
Results summary
not available
Link to the results in the primary register
not available