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Gene Therapy
Gene Therapy for Duchenne Muscular Dystrophy
Phase 1 & 2
Waitlist Available
Led By Megan Waldrop, MD
Research Sponsored by Megan Waldrop
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Age greater than 6 months and less than 14 years
Confirmed duplication of exon 2 in the DMD gene using a clinically accepted technique that completely defines the mutation
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights
Study Summary
This trial is testing a new gene therapy for Duchenne muscular dystrophy, which is a progressive muscle-wasting disease. The therapy is given as a one-time injection into a vein in the arm or leg.
Who is the study for?
This trial is for boys aged 6 months to less than 14 years with Duchenne muscular dystrophy due to a specific genetic change (duplication of exon 2). Participants can be walking or not and must have been on stable corticosteroid therapy if over age 4. They cannot join if they have heart problems, certain infections like HIV or hepatitis, autoimmune diseases, abnormal blood counts, or high antibodies against the treatment vector.Check my eligibility
What is being tested?
The trial tests a single dose of gene therapy called scAAV9.U7.ACCA delivered through an injection into a vein in the arm. It's designed for boys with Duchenne muscular dystrophy who have duplication of exon 2 in their genes. The study is open-label, meaning everyone knows what treatment is being given.See study design
What are the potential side effects?
Potential side effects may include reactions at the injection site, immune responses to the AAV9 vector such as fever or chills, muscle pain from muscle testing procedures and possible risks associated with gene therapy that are currently unknown.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I am between 6 months and 14 years old.
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My test shows a specific genetic change in the DMD gene.
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I can walk 10 meters without help or I cannot walk yet.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Monitoring for the development of unacceptable toxicity.
Secondary outcome measures
Change in dystrophin expression from baseline following treatment with scAAV9.U7.ACCA.
Changes in exon 2 inclusion in the dystrophin mRNA transcript.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Cohort 1 (Minimal Efficacious Dose)Experimental Treatment1 Intervention
The Minimal Effective Dose (MED) will be delivered.
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Who is running the clinical trial?
Megan WaldropLead Sponsor
1 Previous Clinical Trials
10 Total Patients Enrolled
Audentes TherapeuticsIndustry Sponsor
7 Previous Clinical Trials
270 Total Patients Enrolled
Megan Waldrop, MDPrincipal InvestigatorNationwide Children's Hospital
2 Previous Clinical Trials
12 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am between 6 months and 14 years old.My test shows a specific genetic change in the DMD gene.I can walk 10 meters without help or I cannot walk yet.Your test results are outside the healthy range according to the Nationwide Children's Hospital Laboratory.You have high levels of AAV9 binding antibodies in your blood.Your white blood cell count is too low or too high, or your neutrophil count is too low.You have evidence of HIV, Hepatitis B, or Hepatitis C infection in your blood.I am a male.I experience symptoms like shortness of breath, swollen feet, or difficulty breathing when lying down.I've been on a stable dose of corticosteroid therapy for at least 12 weeks.I currently have an active viral infection.I have been diagnosed with or am being treated for an autoimmune disease.My heart's pumping ability is below normal.You have a long-term medical condition or need to take certain medications that the study doctors think could make the gene transfer treatment risky for you.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 1 (Minimal Efficacious Dose)
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Does this medical study accept elderly participants over the age of 55?
"This medical research is seeking participants aged between half a year and thirteen years."
Answered by AI
Am I eligible to volunteer for this research initiative?
"This gene transfer trial is recruiting 3 children who are 6 Months up to 13 years old with muscular dystrophy. Eligibility requirements include being male, having taken a stable dose of corticosteroids for 12 weeks prior to the study start date, and possessing pre-ambulatory or ambulatory capabilities (walking 10 meters unassisted)."
Answered by AI
Is this research study currently recruiting participants?
"Although it was uploaded on the 15th of January 2020, according to clinicaltrials.gov this medical trial is no longer recruiting patients. Luckily, there are 86 additional trials that individuals can consider joining at present."
Answered by AI
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