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

NCT03362502 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 23

Last updated 2025-09-15

Study results available
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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 and non-ambulatory subjects with Duchenne muscular dystrophy (DMD). Other objectives include measurement of dystrophin expression and distribution, and assessments of muscle strength, quality, and function.

A total of approximately 22 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. Up to 13 subjects may be included in a cohort that includes the concomitant medication, sirolimus. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between two planned dose-levels and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.

Conditions

Interventions

GENETIC

PF-06939926

Recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a muscle-specific promoter. Subjects will receive a single intravenous infusion of one of 2 dose levels.

Sponsors & Collaborators

Principal Investigators

  • Pfizer CT.gov Call Center · Pfizer

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
4 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-01-23
Primary Completion
2022-03-28
Completion
2025-07-28
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03362502 on ClinicalTrials.gov