Actively Recruiting
Registry Study of Children and Adults Patients With Relapsed, Refractory, or Progressive Sonic Hedgehog Medulloblastoma Harboring U1 Mutation
Led by Mohammad H. Abu Arja · Updated on 2026-04-13
300
Participants Needed
2
Research Sites
N/A
Total Duration
On this page
Sponsors
M
Mohammad H. Abu Arja
Lead Sponsor
B
Baylor College of Medicine
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting a multicenter observational study to create a biobank for children and young adults aged 3 to 50 years diagnosed with relapsed, refractory, or progressive Sonic Hedgehog (SHH) medulloblastoma, focusing on those with a U1 gene mutation. This mutation affects tumor DNA splicing and may influence tumor mutation burden and treatment responses. The study aims to better understand SHH medulloblastoma biology and guide future therapies, especially for relapsed cases, with no direct benefits guaranteed to participants. Participants include patients treated at registry institutions, those treated elsewhere, and their biological parents. The study collects clinical data, treatment histories, imaging, and biological samples including tumor tissue and optional buccal swabs from parents. Retrospective and prospective data will be gathered and stored in a secure biobank. Advanced tests like RNA sequencing, PCR, methylation profiling, and whole genome sequencing will be performed on samples. Imaging data will be securely stored and de-identified. Participants' involvement lasts about five years, during which their medical records, imaging, and tissue samples will be reviewed and analyzed. Data quality checks are performed regularly. The main outcome is detecting the U1 mutation using PCR or RNA sequencing. Secondary outcomes include comparing treatment outcomes by U1 mutation status. No safety assessments for adverse events are planned since this is an observational study.
CONDITIONS
Brief Title
Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 3 to 50 years at initial diagnosis
- Diagnosis of Sonic Hedgehog medulloblastoma by histologic or molecular criteria
- Disease status is recurrent, refractory, or progressive after therapy including radiotherapy and chemotherapy
- Available tumor tissue sample for U1 mutation testing
- Biological parents of eligible patients may participate
You will not qualify if you...
- Subjects not meeting the inclusion criteria will be excluded
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 2 years or until study completion
Participants who have relapsed, refractory, or progressive Sonic Hedgehog medulloblastoma are observed through collection of medical records, imaging, tissue samples, and genetic data to understand prior therapies and treatment courses.
Data collection through medical records and sample submissions with no additional in-person visits required
Trial Site Locations
Total: 2 locations
1
Baylor College of Medicine
Houston, Texas, United States, 77004
Actively Recruiting
2
MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
Research Team
M
Mohammad H Abu-Arja, MD, MSc
T
Ta Tara Rideau
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
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