In short
Clinical trials are investigating ASP2957 in male patients with X-linked myotubular myopathy who need ventilator support. These studies aim to assess safety, tolerability, and the dose level to use in future research. The trial data focus on treatment effects over time and detailed safety checks.
Key points
- Clinical trials of ASP2957 are studying male patients with X-linked myotubular myopathy who need ventilator support. The available trial is an early Phase 1/2 study and is authorised. Its main goals are to assess safety, tolerability, and the dose level that may be recommended for future research. The study plans to enroll 9 participants. Researchers will also track side effects and safety changes through week 52 using lab tests, heart tests, muscle MRI, muscle tissue checks, and physical exams.
Trial overview
The available study is an interventional trial, which means researchers give ASP2957 to participants and then measure what happens. It is studying male patients with X-linked myotubular myopathy, a rare muscle disease that can cause serious weakness and breathing problems.
The brief goal of the study is to evaluate the safety and tolerability of ASP2957 and to find the recommended dose level for future research.
Who can join the study
This study is for male patients with X-linked myotubular myopathy who need ventilator support, meaning they need a machine to help them breathe. The source data do not give more detailed entry rules, so the main known target group is narrow and specific.
- Male patients only: the trial title states that the study is for male patients.
- Patients with X-linked myotubular myopathy: this is the condition being studied.
- Patients who need ventilators: the trial focuses on people with breathing support needs.
What is being measured
The main outcome is the incidence and severity of TEAEs and AESIs, which means how often unwanted medical events happen and how serious they are. TEAEs are treatment-emergent adverse events, and AESIs are adverse events of special interest that researchers watch closely.
The study also measures change from baseline, which means it compares each patient’s results after treatment with their starting results. The safety checks include clinical laboratory tests, cardiac findings from ECG and ECHO, muscle findings from muscle MRI and histopathology, and physical examinations.
- Clinical laboratory tests: blood or other lab tests that help show how the body is responding.
- ECG and ECHO: heart tests used to look at heart rhythm and heart function.
- Muscle MRI: a scan that shows detailed pictures of muscle tissue.
- Histopathology: examination of tissue under a microscope to look for changes in the muscle.
- Physical examinations: doctor-led checks of the body and general health.
Study phase and size
The trial is a Phase 1/2 study, which means it is in an early stage of clinical research. Early-phase studies usually focus on safety first, while also beginning to look for signs that help guide later research.
The planned enrollment is 9 participants, so this is a small study. Small studies like this are common when researchers are first learning how a treatment performs in a specific patient group.
Safety follow-up through week 52
The safety assessments are tracked through week 52, which means the study follows patients for up to one year after starting treatment. This long follow-up helps researchers see whether problems appear early or later over time.
Because the trial checks labs, heart tests, muscle scans, tissue findings, and physical exams, it is looking at safety from several angles rather than relying on just one test.
Trial status
The study status is Authorised, which means the trial has been approved to proceed according to the source data. The trial is identified as NCT07052929.