Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study

Plain-language summary

This registry study follows patients with relapsed, refractory, or progressive Sonic Hedgehog (SHH) medulloblastoma, a type of brain tumor, and tests their tumors for the U1 mutation. It enrolls patients who were between 3 and 50 years old at first diagnosis and have tumor tissue available for U1 testing, as well as their biological parents. It is a registry study with no assigned phase and is currently recruiting participants.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Recruiting

Sponsor

Mohammad H. Abu Arja

Start date

9/30/2025

Estimated completion

12/30/2035

Countries

United States

Related genes

Locations

• Houston, United States
• Houston, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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.