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

NCT03362502

Last updated date
Study Location
Duke Biospecimen Repository & Processing Core - BPRC
Durham, North Carolina, 27710, United States
Contact
1-800-718-1021

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1-800-718-1021

By email

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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
4-12 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.

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Advanced Information
Descriptive Information
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 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 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.

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 human 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 Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 3, 2019)
15
Original Estimated Enrollment  ICMJE
 (submitted: November 29, 2017)
12
Estimated Study Completion Date  ICMJE August 26, 2025
Estimated Primary Completion Date August 26, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study:Male
Gender Based Eligibility:Yes
Ages  ICMJE 4 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer CT.gov Call Center1-800-718-1021[email protected]
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
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:No
Plan Description:Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d….
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP