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

Last updated on March 14, 2019

FOR MORE INFORMATION
Study Location
Ronald Reagan UCLA Medical Center Drug Information Center
Los Angeles, California, 90095 United States
Contact
1-800-718-1021
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Duchenne Muscular Dystrophy
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
5-12 years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
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- 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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

- 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.

NCT03362502
Pfizer
Enrolling by invitation
A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy

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