ASP2957 for Myotubular Myopathy
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to test a new gene therapy, ASP2957, for boys with X-linked myotubular myopathy (XLMTM), a serious condition affecting muscle function. The trial will introduce a healthy MTM1 gene into the body to improve muscle development and breathing. Initially, the study will test different doses to find the safest and most effective amount, then monitor participants for up to a year. Boys who are ventilator-dependent and have a confirmed diagnosis of XLMTM may be suitable candidates for this trial. As a Phase 1, Phase 2 trial, participants will be among the first to receive this new treatment and help determine its effectiveness in improving muscle function.
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are taking treatments for cholestasis, you must stop them at least 4 weeks before joining the study. Also, you should not have received systemic immunomodulating agents within 90 days before the trial starts.
Is there any evidence suggesting that ASP2957 is likely to be safe for humans?
Research shows that ASP2957 is undergoing its first safety tests in humans. This early-phase trial primarily examines treatment tolerance, with all potential side effects and reactions closely monitored. Young boys receive a single dose of ASP2957, along with medications to prevent immune reactions.
As an early-stage trial, limited information exists on treatment responses. However, the study is carefully designed to identify any medical issues, especially since the treatment uses a modified virus to deliver a gene. The goal is to determine a dose that is both effective and safe. Participants will be monitored for up to a year to assess any long-term effects, ensuring the treatment's safety before advancing to larger studies.12345Why do researchers think this study treatment might be promising?
Unlike the standard treatments for myotubular myopathy, which mainly focus on managing symptoms and providing supportive care, ASP2957 offers a new approach by potentially addressing the underlying cause of the disease. Researchers are excited because ASP2957 is designed to work at the genetic level, aiming to correct the faulty gene responsible for the condition. This method could lead to more substantial and lasting improvements in muscle function compared to existing treatments. Additionally, the use of immunosuppression prophylaxis alongside ASP2957 helps ensure the treatment is safely tolerated, which is a crucial step toward making this innovative therapy viable for patients.
What evidence suggests that ASP2957 might be an effective treatment for myotubular myopathy?
Research has shown that ASP2957 aims to help with X-linked myotubular myopathy (XLMTM) by providing a healthy version of the MTM1 gene. This gene is crucial because it produces myotubularin, a protein essential for proper muscle function. The goal is for the body to produce this protein correctly, potentially improving muscle strength and breathing in affected children. Early results from similar gene therapies have been promising in treating genetic muscle disorders. Although ASP2957 is being tested in humans for the first time, other studies have successfully replaced faulty genes. Participants in this trial will receive ASP2957 in two parts: Part 1 involves dose escalation, and Part 2 involves dose expansion, both with immunosuppression prophylaxis.12345
Who Is on the Research Team?
Medical Director
Principal Investigator
Astellas Gene Therapies
Are You a Good Fit for This Trial?
This trial is for boys ≤ 3 years old with X-linked myotubular myopathy (XLMTM) who need ventilators over 20 hours a day and have a tracheostomy tube. They must have a confirmed genetic diagnosis of XLMTM, no significant liver issues, be able to receive immunosuppression, and be up-to-date on vaccinations.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Dose Escalation
Participants receive sequential dose levels of a single infusion of ASP2957 and immunosuppression prophylaxis
Dose Expansion
Participants receive a single infusion of ASP2957 at the dose selected in Part 1 with immunosuppression prophylaxis
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of monitoring in another study for long-term follow-up
What Are the Treatments Tested in This Trial?
Interventions
- ASP2957
Trial Overview
The study tests ASP2957, which carries a healthy MTM1 gene into the body using an empty virus to improve muscle function in XLMTM. It includes two phases: Phase 1 finds the right dose by giving different amounts to small groups; Phase 2 uses that dose in another group.
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Participants will receive a single infusion of ASP2957 (dose selected in Part 1) and immunosuppression prophylaxis including methylprednisolone, prednisolone and sirolimus.
Participants will receive sequential dose levels of a single infusion of ASP2957 and immunosuppression prophylaxis including methylprednisolone, prednisolone and sirolimus.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Astellas Gene Therapies
Lead Sponsor
Citations
Study of ASP2957 in Male Participants With X-linked ...
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly.
74PDesign of a phase 1/2 study assessing the safety and ...
74PDesign of a phase 1/2 study assessing the safety and preliminary efficacy of ASP2957 gene therapy for X-linked myotubular myopathy (XLMTM). Author links ...
Study of ASP2957 in male participants with X-linked ...
X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly.
Clinical Trials
The first myotubular myopathy patient was dosed in September 2017 and preliminary results published in January 2018 and October 2019 were very encouraging.
ASP2957 for Myotubular Myopathy
This Phase 1 & 2 medical study run by Astellas Gene Therapies is evaluating whether ASP2957 will have tolerable side effects & efficacy for patients with ...
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