Status:
TERMINATED
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
Lead Sponsor:
Pfizer
Conditions:
Duchenne Muscular Dystrophy
Eligibility:
MALE
4+ years
Phase:
PHASE1
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 and non...
Eligibility Criteria
Inclusion
- 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
- 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:
- Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR
- A deletion that affects both exon 29 and exon 30.
- Sirolimus Cohort
- Inclusion Criteria
- \> 8 years of age Exclusion Criteria
- Hypersensitivity to sirolimus or intolerance to soy, including a history of angioedema
- Concomitant use with strong CYP3A4/P-gp inducers or inhibitors
Key Trial Info
Start Date :
January 23 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 28 2025
Estimated Enrollment :
23 Patients enrolled
Trial Details
Trial ID
NCT03362502
Start Date
January 23 2018
End Date
July 28 2025
Last Update
September 15 2025
Active Locations (22)
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1
MRI Research Center
Los Angeles, California, United States, 90095
2
Reed Neurological Research Center
Los Angeles, California, United States, 90095
3
Ronald Reagan UCLA Medical Center (Investigational Drug Section)
Los Angeles, California, United States, 90095
4
Ronald Reagan UCLA Medical Center - Interventional Radiology
Los Angeles, California, United States, 90095