A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy

Back to Search
Print
Bookmark Trial

NCT04281485

Last updated date
Back to Search
Print
Bookmark Trial
FOR MORE INFORMATION
Study Location
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Roma, , 00168, Italy
See other locations
Contact
1-800-718-1021
[email protected]
Related Links

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting the study website. Please see the references below:

By phone

Pfizer Clinical Trials Contact Center

1-800-718-1021

By email

Contact

[email protected]

Call Now

Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Duchenne Muscular Dystrophy
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
4-7 years

NEED INFO?

Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

Pfizer Clinical Trials Contact Center

1-800-718-1021

[email protected]

Call Now

TRY A NEW SEARCH

Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.

Advanced Search

Based on your search, you may also be interested in

Duchenne Muscular DystrophyA Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy
NCT02310763
  1. Los Angeles, California
  2. Los Angeles, California
  3. Los Angeles, California
  4. Sacramento, California
  5. Aurora, Colorado
  6. Tampa, Florida
  7. Chicago, Illinois
  8. Iowa City, Iowa
  9. Fairway, Kansas
  10. Fairway, Kansas
  11. Kansas City, Kansas
  12. Kansas City, Kansas
  13. Baltimore, Maryland
  14. Baltimore, Maryland
  15. Baltimore, Maryland
  16. Baltimore, Maryland
  17. Boston, Massachusetts
  18. Minneapolis, Minnesota
  19. Saint Louis, Missouri
  20. Durham, North Carolina
  21. Durham, North Carolina
  22. Cincinnati, Ohio
  23. Philadelphia, Pennsylvania
  24. Pittsburgh, Pennsylvania
  25. Salt Lake City, Utah
  26. Salt Lake City, Utah
  27. Salt Lake City, Utah
  28. Salt Lake City, Utah
  29. Salt Lake City, Utah
  30. Salt Lake City, Utah
  31. South Brisbane, Queensland
  32. Sofia,
  33. Sofia,
  34. Sofia,
  35. Sofia,
  36. Sofia,
  37. Calgary, Alberta
  38. Vancouver, British Columbia
  39. London, Ontario
  40. Montreal, Quebec
  41. Genova,
  42. Genova,
  43. Genova,
  44. Genova,
  45. Genova,
  46. Rome,
  47. Rome,
  48. Rome,
  49. Rome,
  50. Rome,
  51. Rome,
  52. Hyogo,
  53. Tokyo,
  54. Warszawa,
  55. Warszawa,
  56. Warszawa,
  57. Warszawa,
  58. Warszawa,
  59. Warszawa,
  60. Liverpool,
  61. Liverpool,
  62. London,
  63. London,
  64. Newcastle upon Tyne,
  65. Newcastle upon Tyne,
  66. Newcastle-upon-Tyne,
Male
6 Years+
years
MULTIPLE SITES
Duchenne Muscular DystrophyA Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
NCT03760029
  1. Chongqing, Chongqing
  2. Chongqing, Chongqing
  3. Fuzhou, Fujian
  4. Beijing,
  5. Beijing,
  6. Shanghai,
  7. Shanghai,
  8. Shanghai,
Male
0+
years
MULTIPLE SITES
Duchenne Muscular DystrophyA Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
NCT03362502
  1. Salt Lake City, Utah
  2. Los Angeles, California
  3. Los Angeles, California
  4. Los Angeles, California
  5. Los Angeles, California
  6. Los Angeles, California
  7. Los Angeles, California
  8. Los Angeles, California
  9. Durham, North Carolina
  10. Durham, North Carolina
  11. Durham, North Carolina
  12. Durham, North Carolina
  13. Durham, North Carolina
  14. Durham, North Carolina
  15. Salt Lake City, Utah
  16. Salt Lake City, Utah
  17. Salt Lake City, Utah
  18. Salt Lake City, Utah
  19. Salt Lake City, Utah
  20. Salt Lake City, Utah
  21. Salt Lake City, Utah
Male
4 Years+
years
MULTIPLE SITES
Duchenne Muscular DystrophyA Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
NCT04281485
  1. Roma,
  2. Jerusalem,
  3. Petach Tikvah,
  4. Esplugues de Llobregat, Barcelona
  5. Barcelona,
  6. Valencia,
Male
4 Years+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title  ICMJE A Phase 3 Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Official Title  ICMJE 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
Brief 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.
Detailed Description

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Parallel up to the measurement of the primary outcome at Week 52. At the beginning of study Year 2 participants who were originally assigned to placebo will have the opportunity to receive PF-06939926. All participants will be followed for 5 years following treatment with PF-06939926.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description:
The study will be quadruple blind.
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE
  • Genetic: PF-06939926
    PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.
  • Other: Placebo
    Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.
  • Other: Placebo
    Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.
  • Genetic: PF-06939926
    PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Study Arms  ICMJE
  • Cohort 1
    Approximately two thirds of participants will be randomized to Cohort 1.
    Interventions:
    • Genetic: PF-06939926
    • Other: Placebo
  • Cohort 2
    Approximately one third of participants will be randomized to Cohort 2.
    Interventions:
    • Other: Placebo
    • Genetic: PF-06939926
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 20, 2020)		99
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 29, 2027
Estimated Primary Completion Date September 29, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Gender Eligibility Description:Male
Ages  ICMJE 4 Years to 7 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Israel,   Italy,   Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04281485
Other Study ID Numbers  ICMJE C3391003
2019-002921-31 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description:Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d….
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d…
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date January 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP