Gene Therapy for Charcot-Marie-Tooth Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new gene therapy, scAAV1.tMCK.NTF3, for individuals with Charcot-Marie-Tooth Disease Type 1A (CMT1A), which affects leg nerves and causes muscle weakness. The treatment involves a one-time injection into the leg muscles to improve muscle strength and nerve function. Ideal participants have CMT1A, experience ankle weakness, but can still stand on their heels for at least three seconds. As a Phase 1, Phase 2 trial, this research aims to understand how the treatment works in people and measures its effectiveness in an initial, smaller group.
Do I need to stop my current medications for the trial?
The trial does not specify if you need to stop your current medications, but you cannot participate if you are on ongoing immunosuppressive therapy or have taken it within 6 months before the trial. It's best to discuss your current medications with the trial team.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
Research has shown that the gene therapy under study, scAAV1.tMCK.NTF3, appears safe for humans. Studies have found that patients who received this treatment still had detectable levels in their blood six months later. This indicates that the treatment remains active in the body over time without causing immediate harmful effects.
In earlier studies, those who received the treatment showed significant improvement compared to those who did not, starting about ten weeks after administration. This suggests that the therapy might not only be effective but also generally safe.
While detailed safety data is not provided here, the treatment's progression to this stage indicates it has demonstrated an acceptable level of safety so far. However, as with any new treatment, monitoring for side effects remains important.12345Why do researchers think this study treatment might be promising?
Unlike other treatments for Charcot-Marie-Tooth disease, which typically focus on symptom management, the investigational treatment scAAV1.tMCK.NTF3 offers a genetic approach. This therapy uses a viral vector to deliver the human NTF3 gene directly into muscle tissue, aiming to promote nerve health and function. Researchers are excited because this gene therapy could potentially address the underlying cause of the disease rather than just alleviating symptoms. The delivery method via intramuscular injection allows targeted treatment, which might lead to more effective and long-lasting results compared to conventional therapies.
What evidence suggests that this gene therapy might be an effective treatment for Charcot-Marie-Tooth Disease?
Research has shown that a new gene therapy, scAAV1.tMCK.NTF3, may help treat Charcot-Marie-Tooth Disease (CMT1A). In earlier studies, patients had measurable levels of the beneficial protein NT-3 in their blood six months after treatment. This therapy improved muscle function and nerve signals in animal studies by enlarging muscle fibers and aiding nerve healing. It also activates mTOR, a process that enhances cell growth and function. These early findings suggest potential benefits for people with CMT1A, but further research is necessary.13456
Who Is on the Research Team?
Zarife Sahenk, MD., PhD.
Principal Investigator
Nationwide Children's Hospital
Are You a Good Fit for This Trial?
This trial is for individuals aged 18 to 35 with Charcot-Marie-Tooth Disease type 1A (CMT1A) who have a specific gene duplication. Participants should have ankle muscle weakness but can still move against gravity and stand on their heels. They must be able to undergo clinical evaluations and nerve studies, and if sexually active, agree to use contraception.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a one-time intramuscular injection of scAAV1.tMCK.NTF3 into muscles in both legs
Follow-up
Participants are monitored for safety and effectiveness after treatment with follow-up visits on days 7, 14, 30, 60, 90, 120, and months 6, 9, 12, 15, 18, and 24
What Are the Treatments Tested in This Trial?
Interventions
- scAAV1.tMCK.NTF3
Find a Clinic Near You
Who Is Running the Clinical Trial?
Nationwide Children's Hospital
Lead Sponsor