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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE1

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-01-23

Study Completion Date

2025-07-28

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

NCT05429372

Muscular Dystrophy, Duchenne

TERMINATED PHASE2

A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

NCT05689164

Duchenne Muscular Dystrophy

TERMINATED PHASE3

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

NCT04281485

Duchenne Muscular Dystrophy

ACTIVE_NOT_RECRUITING PHASE3

An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

NCT02907619

Duchenne Muscular Dystrophy

TERMINATED PHASE2

A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy

NCT02310763

Duchenne Muscular Dystrophy

TERMINATED PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Duchenne Muscular Dystrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

PF-06939926

Group Type EXPERIMENTAL

PF-06939926

Intervention Type GENETIC

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type GENETIC

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Age as follows, based on ambulatory status:

* FOR AMBULATORY PARTICIPANTS, defined as the ability to walk at least 10 meters unassisted: Between 4 and 12 years, inclusive,
* FOR NON-AMBULATORY PARTICIPANTS, defined as the inability to walk at least 10 meters unassisted: No age restrictions so long as loss of ambulation occurs prior to the subject's 17th birthday;
* Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing;
* Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry;
* 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;
* Body weights as follows, based on ambulatory status:

* FOR AMBULATORY PARTICIPANTS: Between 15 kg and 50 kg,
* FOR NON-AMBULATORY PARTICIPANTS: Less than 75 kg, but which may be managed or adjusted to a lower limit, especially to ensure participant safety;
* Functional performance as follows, based on ambulatory status:

* FOR AMBULATORY PARTICIPANTS: Ability to rise from floor within seven (7) seconds,
* FOR NON-AMBULATORY PARTICIPANTS: Percent predicted forced vital capacity greater than 40% as part of pulmonary function tests, as well as adequate upper limb function.

Exclusion Criteria

* Receipt of live attenuated vaccination within 3 months prior to receiving PF-06939926 or exposure to an influenza (or other inactivated) vaccination or 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 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);
* Compromised cardiac function as indicated by left ventricular ejection fraction on cardiac MRI, as follows, based on ambulatory status:

* FOR AMBULATORY PARTICIPANTS: Less than 55%,
* FOR NON-AMBULATORY PARTICIPANTS: Less than 35%;
* Inadequate hepatic or renal function or risk factors for autoimmune disease on screening laboratory assessments.
* The following genetic abnormalities in the dystrophin gene as confirmed by the investigator based on the review of the DMD genetic testing:

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 30.

Sirolimus Cohort

* Hypersensitivity to sirolimus or intolerance to soy, including a history of angioedema
* Concomitant use with strong CYP3A4/P-gp inducers or inhibitors

Minimum Eligible Age

4 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

Explore where the study is taking place and check the recruitment status at each participating site.