Actively Recruiting

Age: 3Years - 50Years
All Genders
NCT07242963

Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study

Led by Mohammad H. Abu Arja · Updated on 2026-04-13

300

Participants Needed

2

Research Sites

534 weeks

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

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

Official Title

Relapsed and Progressive Sonic Hedgehog Medulloblastoma With U1 Mutation Registry Study

Who Can Participate

Age: 3Years - 50Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients aged 3 to 50 years at initial diagnosis
  • Diagnosis of SHH medulloblastoma by histologic or molecular criteria
  • Disease status is recurrent, refractory, or progressive after therapy including radiotherapy and chemotherapy
  • Available tumor tissue samples for U1 mutation testing
  • Biological parents of enrolled subjects are eligible to participate
Not Eligible

You will not qualify if you...

  • Subjects not meeting all of the inclusion criteria will be excluded

AI-Screening

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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

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Research Team

M

Mohammad H Abu-Arja, MD, MSc

CONTACT

T

Ta Tara Rideau

CONTACT

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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