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PF-06939926 for Duchenne Muscular Dystrophy

The Children's Hospital of Philadelphia, Philadelphia, PA
Duchenne Muscular DystrophyPF-06939926 - Genetic
Eligibility
2 - 3
Male
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Study Summary

This trialwill study gene therapy to help boys with Duchenne Muscular Dystrophy (DMD) with safety and muscle strength. No randomization, open to all.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 2

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Study Objectives

0 Primary · 12 Secondary · Reporting Duration: Through 5 years

Week 52
Distribution of mini-dystrophin expression in muscle
Level of mini-dystrophin expression in muscle
Through 5 years
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Number of participants with abnormal and clinically relevant changes in body weight
Number of participants with abnormal and clinically relevant changes in neurological examinations
Number of participants with abnormal and clinically relevant changes in vital signs
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Number of participants with abnormal and clinically relevant changes on echocardiogram
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Number of participants with abnormal biochemistry test results
Number of participants with abnormal hematology test results
Number of participants with abnormal urine analysis

Trial Safety

Phase-Based Safety

2 of 3
This is further along than 68% of similar trials

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Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.

Trial Design

1 Treatment Group

PF-06939926
1 of 1

Experimental Treatment

10 Total Participants · 1 Treatment Group

Primary Treatment: PF-06939926 · No Placebo Group · Phase 2

PF-06939926
Genetic
Experimental Group · 1 Intervention: PF-06939926 · Intervention Types: Genetic

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: through 5 years

Who is running the clinical trial?

PfizerLead Sponsor
4,403 Previous Clinical Trials
25,776,464 Total Patients Enrolled
Pfizer CT.gov Call CenterStudy DirectorPfizer
3,344 Previous Clinical Trials
23,428,707 Total Patients Enrolled

Eligibility Criteria

Age 2 - 3 · Male Participants · 0 Total Inclusion Criteria

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References

Frequently Asked Questions

Is enrollment open to those over 20 years of age for this clinical experiment?

"This clinical trial's eligibility criteria necessitates that participants are aged between 2 and 3. Individuals who fall below the age of consent can take part in 59 separate studies, while those above 65 can enrol into 50 different trials." - Anonymous Online Contributor

Unverified Answer

Am I able to join this clinical investigation?

"Applicants aged 2 - 3 with a formally diagnosed case of becker muscular dystrophy are currently being sought for this study. Critically, those hoping to join must have prior genetic testing certifying their diagnosis." - Anonymous Online Contributor

Unverified Answer

Is this experiment actively accepting new participants?

"According to clinicaltrials.gov, the recruitment period for this medical trial is ongoing; it first went live on 8th August 2022 and was recently revised on 12th December 2022." - Anonymous Online Contributor

Unverified Answer

Has the FDA authorized PF-06939926 for general use?

"We at Power have determined that the safety of PF-06939926 can be rated a 2, as this is a Phase 2 clinical trial and while there exists some evidence demonstrating its security, no data has yet been provided suggesting efficacy." - Anonymous Online Contributor

Unverified Answer

How many participants have volunteered for this research experiment?

"Affirmative. Clinicaltrials.gov shows that this medical study, which was launched on August 8th 2022, is actively enrolling patients. At six different trial sites across the nation, 10 participants must be recruited for the clinical investigation to progress as planned." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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