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New treatment option for X-Linked Myotubular Myopathy
Official title Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
ClinicalTrials.gov ID: NCT07052929
What this study is testing
What is ASP2957?
ASP2957 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for x-linked myotubular myopathy.
Also referred to as MyoAAV3.8 engineered capsid - myosin heavy chain kinase 7 - human myotubularin (MyoAAV3.8-MHCK7-hMTM1).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- X-linked myotubular myopathy (XLMTM) is a rare and serious condition present at birth where the muscles do not work properly. There are currently no approved therapies for XLMTM.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 1 year
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 36, men only
You may be able to join if
- Participant is projected to be ≤ 36 months of age at dosing.
- Participant has molecular genetic report from a CAP-approved testing facility at screening that confirms a diagnosis of XLMTM and harbors a...
- Participant is ventilator-dependent and meets the following criteria:
- Required respiratory support at birth
- Requires ≥ 20 hours per day of invasive ventilator support (confirmed during screening)
You likely can't join if
- Participant born \< 35 weeks gestation is still not term as per corrected age.
- Participant is nutritionally unstable with weight less than fifth percentile for age or has a vitamin A, E or K deficiency.
- Participant requires supplemental oxygen on a routine or chronic basis.
- Note: The use of supplemental oxygen for acute, self-limited illnesses (for example, during hospitalization for pneumonia) shall not be exclusionary...
- Participant currently has a clinically important respiratory infection or other clinically important active infection of any kind.
- Participant has an active viral or bacterial infection including, but not limited to, positive testing for the following:
See the full eligibility criteria
Who can join
- Participant is projected to be ≤ 36 months of age at dosing.
- 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...
- Participant is ventilator-dependent and meets the following criteria:
- Required respiratory support at birth
- Requires ≥ 20 hours per day of invasive ventilator support (confirmed during screening)
- Has a tracheostomy tube
- Participant has no evidence of hepatic peliosis, increased echogenicity or any other clinically important abnormal finding on liver ultrasound.
- Participant can receive immunosuppression per protocol.
- Participant's hepatobiliary laboratory measurements must meet the criteria during screening and for the 2-month retrospective assessment of participant's medical history from the time of signing the ICF:
- Participant's hematological laboratory measurements must meet the criteria during screening:
- Participant's parent(s) or legally authorized representative LAR(s) must provide documentation of being current with recommended immunization schedule according to regional guidelines.
- If any immunization has not been administered, the medical reasons must be documented by the investigator along with medical risk associated with ASP2957 and immunosuppression administration. The sponsor will review the...
- Immunizations requiring administration after inclusion in the study must be administered in accordance with regional guidelines for live, live attenuated and inactivated immunization prior to, during and after stopping...
- Immunization of household contacts can be considered based on regional standards of care of individuals receiving immunosuppression regimens.
- Participant and participant's parent(s) or LAR(s) are willing and able to comply with study visits and study procedures.
- Participant's parent(s) or LAR(s) agree that the participant will not participate in another treatment study from the time of signing the Informed Consent Form (ICF) through week 52.
- Participant's parent(s) or LAR(s) is willing to transition the participant to a separate long-term follow-up study after study completion.
What rules you out
- Participant born \< 35 weeks gestation is still not term as per corrected age.
- Participant is nutritionally unstable with weight less than fifth percentile for age or has a vitamin A, E or K deficiency.
- Participant requires supplemental oxygen on a routine or chronic basis.
- 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...
- Participant currently has a clinically important respiratory infection or other clinically important active infection of any kind.
- Participant has an active viral or bacterial infection including, but not limited to, positive testing for the following:
- tuberculosis (TB) using the QuantiFERON-TB test
- Active hepatitis A virus (HAV), hepatitis B virus (HBV) or hepatitis C virus (HCV)
- Prior HBV or HCV virus infection due to the risk of reactivation associated with immunosuppression
- human immunodeficiency virus type 1 (HIV-1) and human immunodeficiency virus type 2 (HIV-2)
- coronavirus disease 2019 (COVID-19)
- cytomegalovirus (CMV), viral loads ≥ 500 IU/mL or attributable symptoms or evidence of end-organ disease due to CMV.
- 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...
- Neonatal hyperbilirubinemia resolving within 4 weeks of birth in a full-term infant is not an exclusion.
- Participant has prior history of abnormal transaminases (alanine aminotransferase (ALT) or aspartate aminotransferase (AST)) and/or abnormal bilirubin metabolism associated with ascites, jaundice (aside from neonatal...
- Participant has a significant medical condition or life-threatening disease other than XLMTM that would interfere with adhering to protocol requirements or would increase the risk of immunosuppression and/or recombinant...
- Participant has musculoskeletal complications such as severe contractures and/or scoliosis that would limit the ability to observe improvements in neuromuscular function.
- In participants with scoliosis, the Cobb angle must be \< 40 degrees for study eligibility.
- 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...
- Participant has previously received monoclonal antibodies of any type.
- Exception: Monoclonal antibodies to prevent RSV are permitted, except for during the 4 weeks prior to the initiation of immunosuppression.
- Participant plans to have surgery within 12 weeks prior to day 1 through week 52 that may confound safety and how well it works data interpretation of the study intervention.
- Exception: Standard of care surgical interventions such as gastrostomy, jejunostomy and Nissen fundoplication procedures are allowed.
- Participant received any treatment for cholestasis (e.g., ursodiol, cholestyramine, rifampin or other therapies) prior to signing the ICF.
- Participant is participating in another treatment study or has received an adeno-associated virus (AAV)-based gene therapy.
- Participant tests positive for anti-MyoAAV3.8 TAb, as determined by central laboratory testing.
- 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 total antibody (TAb) may be rescreened for study...
- Participant has a known or suspected contraindication or hypersensitivity to methylprednisolone, prednisolone, sirolimus or any components of the ASP2957 formulation.
- Participant has a contraindication to general anesthesia, magnetic resonance imaging (MRI) or muscle biopsy procedures.
- Any other reason that would render the participant unsuitable for participation in the study, including risk of non-adherence to the study assessments and protocol.
The study team makes the final eligibility decision.
Where it's taking place
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- Portland, Oregon, United States
- Toronto, Ontario, Canada
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 study runs about 1 year per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling male, up to 36 months. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chicago, Illinois, United States; Boston, Massachusetts, United States; Portland, Oregon, United States; Toronto, Ontario, Canada. 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 ClinicalTrials.gov; the study team decides eligibility.