Recruiting

Sonic Hedgehog Medulloblastoma

Sponsor:

Mohammad H. Abu Arja

Code:

NCT07242963

Conditions

Medulloblastoma Recurrent

Medulloblastoma, Childhood, Recurrent

Medulloblastoma, SHH-activated and TP53 Mutant

Medulloblastoma, SHH-activated and TP53 Wildtype

Eligibility Criteria

Sex: All

Age: 3 - 50

Healthy Volunteers: Not accepted

Study Details

Brief summary:

The purpose of this study is to create a biobank for patients diagnosed with Sonic Hedgehog Medulloblastoma at Baylor College of Medicine/Texas Children's Cancer Center. A biobank is a facility that stores and manages biological samples (such as blood, tissue, or DNA) from individuals, along with detailed health information, for use in medical research to study diseases and develop new treatments. The investigators are requesting participants' permission to add their information and samples to this biobank.

Being in this research study is voluntary; it is the participant's choice. If the participant joins this study, they can still stop at any time.

If the participant decides to participate, the investigators will review the participant's clinical medical records, demographics, treatment history, family history, and imaging. The investigators will also collect biological samples from the participant and the biological parents' buccal swabs (optional).

The participation in this biobank will last about 5 years from the decision to participate.

Why am I being asked to participate? The participant or their child is invited to participate in this study if the participant or their tumor may have a U1 mutation. U1 mutation is associated with an error in the gene that splices the tumor DNA, leading to random splicing that may increase the tumor mutation burden and generate novel tumor neoantigens (targets). Studying the U1 mutation will enable the investigator to design more effective therapies and guide future treatments for patients with relapsed or refractory sonic hedgehog medulloblastoma, thereby improving their outcomes and quality of life. Moreover, the investigators aim to determine whether germline mutations inherited from parents may increase the risk of medulloblastoma in their offspring. The participant will receive no direct benefit from their participation in this study. However, participation in this study may help the investigators better understand SHH Medulloblastomas and benefit other patients in the future.

Conditions

Medulloblastoma Recurrent

Medulloblastoma, Childhood, Recurrent

Medulloblastoma, SHH-activated and TP53 Mutant

Medulloblastoma, SHH-activated and TP53 Wildtype

Study ID

NCT07242963

Start date

Sep 30, 2025

Status verified date

Aug, 2026

Completion date

Dec 30, 2035

Anticipated

Primary completion date

Dec 30, 2035

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 3 - 50

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

300 participants

Anticipated

Interventions and Outcome Measures

Arms

Group 1

Patients treated at registry institutions

Group 2

Patients not treated at registry institutions

Group 3

Biological parents of a subject participating in Group 1 or 2

Primary outcome measure

  • To describe the incidence of the U1 mutation in SHH medulloblastoma subtypes and verify the feasibility of U1 testing. The primary outcome will be detecting the U1 mutation by polymerase chain reaction (PCR) testing or RNA sequencing (RNAseq). [ Time Frame: Through study completion, an average of 2 years ]

Central Contacts and Locations

Central contacts

Mohammad H Abu-Arja, MD, MSc

832-824-0592mohammad.abuarja@bcm.edu

Ta Tara Rideau

TLRIDEAU@texaschildrens.org

Locations

Baylor College of Medicine

Recruiting

Houston, Texas, United States, 77004

Contacts

Mohammad H Abu-Arja, MD, MSc

832-824-0592mohammad.abuarja@bcm.edu

Ta-Tara Rideau

TLRIDEAU@texaschildrens.org

MD Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Gregory K Friedman, MD

713-792-6620 , 713-792-2121GKFriedman@mdanderson.org

Shahzeen Ali

832-468-8955Sali18@mdanderson.org

More Information

Sponsor

Mohammad H. Abu Arja

Last update posted

Sep 1, 2026

Last verified

Aug, 2026

Keywords

  • SHH medulloblastoma
  • medulloblastoma
  • medulloblastoma Recurrent

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Mohammad H. Abu Arja on 2026-09-01.