Registry for relapsed or progressive SHH medulloblastoma with U1 mutation
Registry Study of Children and Adults Patients With Relapsed, Refractory, or Progressive Sonic Hedgehog Medulloblastoma Harboring U1 Mutation
This registry collects medical records and biological samples from people aged 3–50 with relapsed or progressive SHH medulloblastoma to test for U1 mutations and create a biobank for research.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 300 (estimated) |
| Ages | 3 Years to 50 Years |
| Sex | All |
| Sponsor | Baylor College of Medicine Academic / other |
| Drugs / interventions | chemotherapy |
| Locations | 2 sites (Houston, Texas and 1 other locations) |
| Trial ID | NCT07242963 on ClinicalTrials.gov |
What this trial studies
This multicenter observational registry collects both retrospective and prospective clinical data and tumor samples from patients diagnosed with relapsed, refractory, or progressive SHH medulloblastoma. Participants provide tumor tissue for U1 mutation testing and may provide blood and optional buccal swabs from biological parents for genetic studies. Clinical charts, treatment history, imaging (uploaded or sent on CD), and demographics will be centralized and linked to the biobank. The registry aims to enable molecular characterization and future translational research by making annotated samples and data available to investigators.
Who should consider this trial
Good fit: Children and young adults diagnosed at age 3–50 with relapsed, refractory, or progressive SHH medulloblastoma who have available tumor tissue for U1 testing, with biological parents eligible to provide optional samples.
Not a fit: Patients without available tumor tissue, those with non-SHH medulloblastoma subtypes, or those seeking an immediate therapeutic intervention are unlikely to receive direct benefit from participation.
Why it matters
Potential benefit: If successful, the biobank could help researchers better understand U1-mutated SHH medulloblastoma and support development of targeted treatments in the future.
How similar studies have performed: Biobanks and molecular registries for medulloblastoma subtypes have aided research, but focused data on U1-mutated SHH medulloblastoma remain relatively novel and limited.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: For Groups 1 and 2, subjects are eligible to be included in the study only if all of the following criteria are met: * Age Patients must be ≥ 3 and ≤ 50 years of age at the time of initial diagnosis. * Diagnosis Participants must have a diagnosis of SHH medulloblastoma by histologic or molecular criteria at the time of original diagnosis or relapse. * Disease status The disease must be recurrent, refractory, or progressive following therapy, including radiotherapy and chemotherapy. * Available tumor tissue sample for U1 testing Participants must have available tumor tissue samples to be tested for the U1 mutation. For Group 3, biological parent(s) of a subject participating in Group 1 or 2 are eligible. Exclusion Criteria: Subjects not meeting the inclusion criteria will be excluded.
Where this trial is running
Houston, Texas and 1 other locations
- Baylor College of Medicine — Houston, Texas, United States (Recruiting)
- MD Anderson Cancer Center — Houston, Texas, United States (Recruiting)
Study contacts
- Principal investigator: Mohammad H Abu-Arja, MD, MSc — Baylor College of Medicine
- Study coordinator: Mohammad H Abu-Arja, MD, MSc
- Email: mohammad.abuarja@bcm.edu
- Phone: 832-824-0592
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.