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Study of ASP2957 in Male Patients with X-linked Myotubular Myopathy Who Need Ventilators
EU CTIS ID: 2025-523213-29-00
What this study is testing
To evaluate the safety and tolerability of ASP2957 and to determine the recommended dose level
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Participant is projected to be ≤ 36 months of age at dosing
- 2. Participant has molecular genetic report from a CAP-approved testing facility at screening that confirms a diagnosis of XLMTM and harbors a “pathogenic” or “likely pathogenic” variant in the MTM1 gene as classified using the ACMG standards and guidelines for interpretation of sequence variants
- 3. Participant is ventilator-dependent and meets the following criteria: a. Required respiratory support at birth b. Requires ≥ 20 hours per day of invasive ventilator support (confirmed during screening) c. Has a tracheostomy tube
- 4. Participant has no evidence of hepatic peliosis, increased echogenicity or any other clinically important abnormal finding on liver ultrasound as assessed by the investigator in consultation with the site hepatologist
- 5. Participant’s hepatobiliary laboratory measurements must meet the following criteria during screening and for the 2-month retrospective assessment of participant’s medical history: a. a. ALT and AST < 3 × ULN b. Direct bilirubin ≤ 1 × ULN c. Serum total bile acids < 2 × ULN, independent of fasting status
- 6. Participant’s parent(s) or LAR(s) must provide documentation of being current with recommended immunization schedule according to regional guidelines.
You likely can't join if
- 1. Participant born < 35 weeks gestation is still not term as per corrected age.
- 10. Participant tests positive for anti-MyoAAV3.8 TAb, as determined by central laboratory testing. a. Note: Since very young children may have passive antibodies transferred in utero from the mother, participants ≤ 6 months of age who initially test positive for anti- MyoAAV3.8 TAb may be rescreened for study eligibility.
- 2. Participant is nutritionally unstable with weight less than fifth percentile for age or has a vitamin A, E or K deficiency.
- 3. Participant requires supplemental oxygen on a routine or chronic basis. a. Note: The use of supplemental oxygen for acute, self-limited illnesses (for example, during hospitalization for pneumonia) shall not be exclusionary, provided the participant is neither acutely ill nor using supplemental oxygen at the time of screening.
- 4. Participant currently has a clinically important respiratory infection or other clinically important active infection of any kind.
- 5. Participant has an active viral or bacterial infection including, but not limited to, positive testing for: a. TB using the QuantiFERON-TB test b. Active HAV, HBV or HCV c. Prior HBV or HCV virus infection due to the risk of reactivation associated with immunosuppression d. HIV-1 and HIV-2 e. COVID-19 f. CMV, viral loads ≥ 500 IU/mL or attributable symptoms or evidence of end-organ disease due to CMV.
See the full eligibility criteria
Who can join
- 1. Participant is projected to be ≤ 36 months of age at dosing
- 2. Participant has molecular genetic report from a CAP-approved testing facility at screening that confirms a diagnosis of XLMTM and harbors a “pathogenic” or “likely pathogenic” variant in the MTM1 gene as classified using the ACMG standards and guidelines for interpretation of sequence variants
- 3. Participant is ventilator-dependent and meets the following criteria: a. Required respiratory support at birth b. Requires ≥ 20 hours per day of invasive ventilator support (confirmed during screening) c. Has a tracheostomy tube
- 4. Participant has no evidence of hepatic peliosis, increased echogenicity or any other clinically important abnormal finding on liver ultrasound as assessed by the investigator in consultation with the site hepatologist
- 5. Participant’s hepatobiliary laboratory measurements must meet the following criteria during screening and for the 2-month retrospective assessment of participant’s medical history: a. a. ALT and AST < 3 × ULN b. Direct bilirubin ≤ 1 × ULN c. Serum total bile acids < 2 × ULN, independent of fasting status
- 6. Participant’s parent(s) or LAR(s) must provide documentation of being current with recommended immunization schedule according to regional guidelines.
What rules you out
- 1. Participant born < 35 weeks gestation is still not term as per corrected age.
- 10. Participant tests positive for anti-MyoAAV3.8 TAb, as determined by central laboratory testing. a. Note: Since very young children may have passive antibodies transferred in utero from the mother, participants ≤ 6 months of age who initially test positive for anti- MyoAAV3.8 TAb may be rescreened for study eligibility.
- 2. Participant is nutritionally unstable with weight less than fifth percentile for age or has a vitamin A, E or K deficiency.
- 3. Participant requires supplemental oxygen on a routine or chronic basis. a. Note: The use of supplemental oxygen for acute, self-limited illnesses (for example, during hospitalization for pneumonia) shall not be exclusionary, provided the participant is neither acutely ill nor using supplemental oxygen at the time of screening.
- 4. Participant currently has a clinically important respiratory infection or other clinically important active infection of any kind.
- 5. Participant has an active viral or bacterial infection including, but not limited to, positive testing for: a. TB using the QuantiFERON-TB test b. Active HAV, HBV or HCV c. Prior HBV or HCV virus infection due to the risk of reactivation associated with immunosuppression d. HIV-1 and HIV-2 e. COVID-19 f. CMV, viral loads ≥ 500 IU/mL or attributable symptoms or evidence of end-organ disease due to CMV.
- 6. Participant has any history of cholestatic liver dysfunction and/or treatment for cholestasis. If the participant is taking prophylactic treatment for cholestasis (e.g., ursodiol, cholestyramine, rifampin or other therapies) which has not been prescribed for cholestatic liver dysfunction, treatment must be discontinued for at least 4 weeks prior to signing the ICF. a. Neonatal hyperbilirubinemia resolving within 4 weeks of birth in a full-term infant is not an exclusion.
- 7. Participant has prior history of abnormal transaminases (ALT or AST) and/or abnormal bilirubin metabolism associated with ascites, jaundice (aside from neonatal hyperbilirubinemia) or gastrointestinal bleeding.
- 8. Other than as required per protocol, participant has received or plans to receive systemic immunomodulating agents within 90 days before day 1 (use of inhaled corticosteroids to manage chronic respiratory conditions is allowed).
- 9. Participant received any treatment for cholestasis (e.g., ursodiol, cholestyramine, rifampin or other therapies) prior to signing the ICF.
The study team makes the final eligibility decision.
Where it's taking place
- Canada
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Canada; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.