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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):

Study Description
Brief Summary:

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Genetic: PF-06939926 Phase 1

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Anticipated Study Start Date : January 15, 2018
Estimated Primary Completion Date : July 5, 2020
Estimated Study Completion Date : July 7, 2024

Arms and Interventions

Arm Intervention/treatment
Experimental: PF-06939926 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.

Outcome Measures

Primary Outcome Measures :
  1. Incidence of dose-limiting safety or intolerability, as measured by treatment-related adverse events [ Time Frame: through 1 year post-treatment ]

Secondary Outcome Measures :
  1. Evidence of mini-dystrophin expression and distribution using upper limb muscle biopsies [ Time Frame: at baseline, 2 months and 1 year post-treatment ]
  2. Incidence, severity and causal relationship of treatment-emergent adverse events and clinically significant safety findings [ Time Frame: through 5 years post-treatment ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 12 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No

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.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03362502

Contact: Pfizer CT.gov Call Center 1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com

United States, North Carolina
Duke University Medical Center, Lenox Baker Children's Hospital Not yet recruiting
Durham, North Carolina, United States, 27705
Duke Children's Hospital & Health Center Not yet recruiting
Durham, North Carolina, United States, 27710
Duke Early Phase Clinical Research Unit Not yet recruiting
Durham, North Carolina, United States, 27710
Duke Hospital North (OR) Not yet recruiting
Durham, North Carolina, United States, 27710
Duke University Hospital Investigational Drug Services Not yet recruiting
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Study Director: Pfizer CT.gov Call Center Pfizer
More Information

Additional Information:
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03362502     History of Changes
Other Study ID Numbers: C3391001
First Posted: December 5, 2017    Key Record Dates
Last Update Posted: January 12, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
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

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Pfizer:
gene therapy, mini-dystrophin, AAV

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked