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A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03362502
Recruitment Status : Not yet recruiting
First Posted : December 5, 2017
Last Update Posted : January 12, 2018
Information provided by (Responsible Party):

November 29, 2017
December 5, 2017
January 12, 2018
January 15, 2018
July 5, 2020   (Final data collection date for primary outcome measure)
Incidence of dose-limiting safety or intolerability, as measured by treatment-related adverse events [ Time Frame: through 1 year post-treatment ]
Same as current
Complete list of historical versions of study NCT03362502 on ClinicalTrials.gov Archive Site
  • Evidence of mini-dystrophin expression and distribution using upper limb muscle biopsies [ Time Frame: at baseline, 2 months and 1 year post-treatment ]
  • Incidence, severity and causal relationship of treatment-emergent adverse events and clinically significant safety findings [ Time Frame: through 5 years post-treatment ]
Same as current
Not Provided
Not Provided
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
A Phase 1b Multicenter, Open-label, Single Ascending Dose Study To Evaluate The Safety And Tolerability Of Pf-06939926 In Ambulatory Subjects With Duchenne Muscular Dystrophy

This is a first-in-human/first-in-patient, multi-center, open-label, non-randomized, ascending dose, safety and tolerability study of a single intravenous infusion of PF-06939926 in ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function.

Two dose cohorts are planned with up to 6 subjects for each. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between the two cohorts and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.

Not Provided
Phase 1
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Genetic: PF-06939926

Recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a human muscle specific promoter.

Subjects will receive a single intravenous infusion of one of 2 dose levels.

Experimental: PF-06939926
Intervention: Genetic: PF-06939926
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Not yet recruiting
July 7, 2024
July 5, 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing
  • Body weight between 15 and 50 kg
  • Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry
  • Ability to rise from floor within seven (7) seconds and ability to walk
  • Ability to tolerate magnetic resonance imaging (MRI) without sedation and with no contraindications to these procedures
  • Ability to tolerate muscle biopsies under anesthesia with no contraindications to these procedures

Exclusion Criteria:

  • Receipt of live attenuated vaccination within 3 months prior or exposure to a systemic antiviral and/or interferon therapy within 30 days prior to receipt of PF-06939926
  • Prior exposure to any gene therapy agent, including exon-skipping and missense agents
  • Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer
  • Neutralizing antibodies (NAb) against adeno-associated virus, serotype 9 (AAV9) or pre-existing anti-dystrophin T-cell response
  • Compromised cardiac function as indicated by a left ventricular ejection fraction of less than 55% on cardiac MRI
  • Inadequate hepatic or renal function or risk factors for autoimmune disease on screening laboratory assessments.
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
5 Years to 12 Years   (Child)
Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
United States
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Plan Description: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: http://www.pfizer.com/research/clinical_trials/trial_data_and_results/data_requests
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
January 2018

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