A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
NCT03362502
ABOUT THIS STUDY
FOR MORE INFORMATION
Contact a representative by phone, email, or visiting the study website. Please see the references below:
Pfizer Clinical Trials Contact Center
1-800-718-1021
- 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.
- 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.
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Descriptive Information | |||||
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Brief Title ICMJE | A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy | ||||
Official Title ICMJE | A PHASE 1B MULTICENTER, OPEN-LABEL, SINGLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND TOLERABILITY OF PF-06939926 IN AMBULATORY AND NON-AMBULATORY SUBJECTS WITH DUCHENNE MUSCULAR DYSTROPHY | ||||
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-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 30 subjects will receive PF-06939926, and these will include both ambulatory and non-ambulatory subjects. 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. | ||||
Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 | ||||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Sequential Assignment Masking: None (Open Label) Primary Purpose: Treatment | ||||
Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE | 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. | ||||
Study Arms ICMJE | Experimental: PF-06939926
Intervention: Genetic: PF-06939926 | ||||
Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. | |||||
Recruitment Information | |||||
Recruitment Status ICMJE | Enrolling by invitation | ||||
Estimated Enrollment ICMJE | 30 | ||||
Original Estimated Enrollment ICMJE | 12 | ||||
Estimated Study Completion Date ICMJE | July 28, 2026 | ||||
Estimated Primary Completion Date | July 28, 2022 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
| ||||
Ages ICMJE | 4 Years and older (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT03362502 | ||||
Other Study ID Numbers ICMJE | C3391001 | ||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
| ||||
Responsible Party | Pfizer | ||||
Study Sponsor ICMJE | Pfizer | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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PRS Account | Pfizer | ||||
Verification Date | December 2020 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |