Table of Contents
- Trial overview
- Who can join the study
- What is being measured
- Study phase and size
- Safety follow-up through week 52
- Trial status
Trial overview
The available study is an interventional trial, which means researchers give ASP2957 to participants and then measure what happens.[1] It is studying male patients with X-linked myotubular myopathy, a rare muscle disease that can cause serious weakness and breathing problems.[1]
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.[1]
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.[1] The source data do not give more detailed entry rules, so the main known target group is narrow and specific.[1]
- Male patients only: the trial title states that the study is for male patients.[1]
- Patients with X-linked myotubular myopathy: this is the condition being studied.[1]
- Patients who need ventilators: the trial focuses on people with breathing support needs.[1]
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.[1] TEAEs are treatment-emergent adverse events, and AESIs are adverse events of special interest that researchers watch closely.[1]
The study also measures change from baseline, which means it compares each patient’s results after treatment with their starting results.[1] The safety checks include clinical laboratory tests, cardiac findings from ECG and ECHO, muscle findings from muscle MRI and histopathology, and physical examinations.[1]
- Clinical laboratory tests: blood or other lab tests that help show how the body is responding.[1]
- ECG and ECHO: heart tests used to look at heart rhythm and heart function.[1]
- Muscle MRI: a scan that shows detailed pictures of muscle tissue.[1]
- Histopathology: examination of tissue under a microscope to look for changes in the muscle.[1]
- Physical examinations: doctor-led checks of the body and general health.[1]
Study phase and size
The trial is a Phase 1/2 study, which means it is in an early stage of clinical research.[1] Early-phase studies usually focus on safety first, while also beginning to look for signs that help guide later research.[1]
The planned enrollment is 9 participants, so this is a small study.[1] Small studies like this are common when researchers are first learning how a treatment performs in a specific patient group.[1]
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.[1] This long follow-up helps researchers see whether problems appear early or later over time.[1]
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.[1]
Trial status
The study status is Authorised, which means the trial has been approved to proceed according to the source data.[1] The trial is identified as NCT07052929.[1]
