AAV9 U7snRNA Gene Therapy to Treat Boys With DMD Exon 2 Duplications.

Launched by MEGAN WALDROP · Jan 22, 2020

Keywords

ClinConnect Summary

This clinical trial is studying a new gene therapy called AAV9 U7snRNA to treat boys with Duchenne Muscular Dystrophy (DMD) who have a specific genetic change known as a duplication of exon 2. DMD is a serious condition that affects muscle strength and function. In this trial, boys aged between 6 months and 14 years who have this genetic mutation and are either not yet walking or can walk a short distance without help may be eligible to participate.

Participants will receive a single dose of the therapy through an injection in their arm or leg vein. Before joining the study, participants will need to meet certain health criteria, such as being able to cooperate with muscle testing and having stable treatment with corticosteroids for at least 12 weeks before the therapy. The study is currently active but not recruiting new participants. It’s important for families to know that there are specific health conditions that could prevent a boy from joining, such as serious infections or heart problems. Overall, the goal of this trial is to explore how well this new therapy can help improve muscle function in boys with this specific type of DMD.

Gender

MALE

Eligibility criteria

• Inclusion Criteria:
• • 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
• • Pre-ambulant (not yet walking) or ambulant (as defined by the ability to walk 10 meters without assistance)
• • Males of any ethnic group will be eligible
• • Ability to cooperate with muscle testing
• • In subjects age 4 and above, stable dose and regimen of corticosteroid therapy (prednisone, deflazacort, or their generic forms) for at least 12 weeks prior to gene transfer.
• Exclusion Criteria:
• • Active viral infection based on clinical observations
• * Symptoms or signs of cardiomyopathy, including:
• • 1. Dyspnea on exertion, pedal edema, shortness of breath upon lying flat, or rales at the base of the lungs
• • 2. Echocardiogram with ejection fraction below 40%
• • Serological evidence of HIV infection, or Hepatitis B or C infection
• • Diagnosis of (or ongoing treatment for) an autoimmune disease
• • Persistent leukopenia or leukocytosis (WBC ≤ 3.5 K/µL or ≥ 20.0 K/µL) or an absolute neutrophil count \< 1.5K/µL
• • Concomitant illness or requirement for chronic drug treatment that in the opinion of the SI creates unnecessary risks for gene transfer
• • AAV9 binding antibody titers ≥ 1:400 as determined by ELISA immunoassay
• • Abnormal laboratory values in the clinically significant range as listed in Table 7, based upon normal values in the Nationwide Children's Hospital Laboratory.

Trial Officials