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

Last updated on June 29, 2018

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.
Show details

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

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A Study to Evaluate the Safety and Tolerability of PF-06939926 Gene Therapy in Duchenne Muscular Dystrophy
A Phase 1b Multicenter, Open-label, Single Ascending Dose Study To Evaluate The Safety And Tolerability Of Pf-06939926 In Ambulatory Subjects With Duchenne Muscular Dystrophy

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

Two dose cohorts are planned with up to 6 subjects for each. In order to mitigate unanticipated risks to subject safety, enrollment will be staggered within and between the two cohorts and will include a formal review by an external data monitoring committee (E-DMC) prior to dose progression.

Not Provided
Interventional
Phase 1
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Genetic: PF-06939926

Recombinant adeno-associated virus, serotype 9 (AAV9) carrying a truncated human dystrophin gene (mini-dystrophin) under the control of a human muscle specific promoter.

Subjects will receive a single intravenous infusion of one of 2 dose levels.

Experimental: PF-06939926
Intervention: Genetic: PF-06939926
Not Provided


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
12
July 7, 2024
July 5, 2020   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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:

  • 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.
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
5 Years to 12 Years   (Child)
No

Contact: Pfizer CT.gov Call Center 1-800-718-1021 [email protected]
United States
 
 
NCT03362502
C3391001
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Plan Description: Information relating to our policy on data sharing and the process for requesting data can be found at the following link: http://www.pfizer.com/research/clinical_trials/trial_data_and_results/da...
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
March 2018

ICMJE     Data element required by the

International Committee of Medical Journal Editors
and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

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