Atypical teratoid rhabdoid tumor

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ORPHA:99966OMIM:609322C49.9
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14Active trials42Specialists8Treatment centers

Where are you in your journey?

ℹ
UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Also known as:

ATRT

Clinical phenotype terms— hover any for plain English:

Malignant neoplasm of the central nervous systemHP:0100836
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Jul 2026B7-H3.CD28Z.CART in CNS Neoplasms

Robbie Majzner — PHASE1

TrialNOT YET RECRUITING
May 2026Gallium Maltolate for the Treatment of Pediatric Patients With Relapsed or Refractory Pediatric High-Grade Glioma and Atypical Teratoid Rhabdoid Tumor

Sarah Rumler — PHASE1

TrialNOT YET RECRUITING
May 2026Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)

Sabine Mueller, MD, PhD — PHASE2

TrialNOT YET RECRUITING
Mar 2026Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas

Milton S. Hershey Medical Center — PHASE1, PHASE2

TrialRECRUITING
Jul 2025A Phase 0/1 Study of cDNA for TP53, Checkpoint Inhibition and Radiation in Children With Recurrent, Progressive or Refractory CNS Malignancies.

Children's National Research Institute — EARLY_PHASE1

TrialNOT YET RECRUITING
Nov 2024Study to ONO-4538 in Patients With Rhabdoid Tumor

Ono Pharmaceutical Co. Ltd — PHASE2

TrialRECRUITING
Sep 2024Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)

Children's National Research Institute — PHASE1

TrialRECRUITING
Aug 2023Tazemetostat+Nivo/Ipi in INI1-Neg/SMARCA4-Def Tumors

Susan Chi, MD — PHASE1, PHASE2

TrialRECRUITING
Apr 2023Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors

St. Jude Children's Research Hospital — PHASE1

TrialRECRUITING
Nov 2022Tiragolumab and Atezolizumab for the Treatment of Relapsed or Refractory SMARCB1 or SMARCA4 Deficient Tumors

National Cancer Institute (NCI) — PHASE1, PHASE2

TrialACTIVE NOT RECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Atypical teratoid rhabdoid tumor.

14 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

14 recruitingView all trials with filters →
Phase 26 trials
Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors
Phase 2
Active
PI: Pfizer CT.gov Call Center (Pfizer) · Sites: Birmingham, Alabama; Phoenix, Arizona +99 more · Age: 2–20 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 2 Study of Alisertib Therapy for Rhabdoid Tumors
Phase 2
Active
PI: Amar Gajjar, MD (St. Jude Children's Research Hospital) · Sites: Palo Alto, California; San Diego, California +7 more · Age: 0–21 yrs
View on ClinicalTrials.gov ↗I'm interested →
Feasibility of Using Bortezomib With or Without Chemotherapy in Patients With Atypical Teratoid/Rhabdoid Tumors
Phase 2
Active
· Sites: Taipei, Taipei · Age: 1–20 yrs
View on ClinicalTrials.gov ↗I'm interested →
Study of Nivolumab and Ipilimumab in Children and Young Adults With INI1-Negative Cancers
Phase 2
Active
PI: Suzanne Forrest, MD (Dana-Farber Cancer Institute) · Sites: San Francisco, California; Atlanta, Georgia +6 more · Age: 0–40 yrs
View on ClinicalTrials.gov ↗I'm interested →
Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
Phase 2
Actively Recruiting
PI: Andreas Peyrl, MD (Medical University of Vienna) · Sites: Chicago, Illinois; Boston, Massachusetts +20 more · Age: 0–19 yrs
View on ClinicalTrials.gov ↗I'm interested →
Study to ONO-4538 in Patients With Rhabdoid Tumor
Phase 2
Actively Recruiting
PI: Project Leader (Ono Pharmaceutical Co. Ltd) · Sites: Nagoya, Aichi-ken; Sapporo, Hokkaido +3 more · Age: 1–99 yrs
View on ClinicalTrials.gov ↗I'm interested →
Phase 15 trials
HER2-specific CAR T Cell Locoregional Immunotherapy for HER2-positive Recurrent/Refractory Pediatric CNS Tumors
Phase 1
Active
PI: Rebecca Ronsley, MD (Seattle Children's Hospital) · Sites: Seattle, Washington · Age: 1–26 yrs
View on ClinicalTrials.gov ↗I'm interested →
Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)
Phase 1
Actively Recruiting
PI: Brian Rood, MD (Children's National Research Institute) · Sites: Washington D.C., District of Columbia · Age: 1–30 yrs
View on ClinicalTrials.gov ↗I'm interested →
B7H3 CAR T Cell Immunotherapy for Recurrent/Refractory Solid Tumors in Children and Young Adults
Phase 1
Active
PI: Catherine Albert, MD (Seattle Children's Hospital) · Sites: Seattle, Washington · Age: 0–26 yrs
View on ClinicalTrials.gov ↗I'm interested →
Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors
Phase 1
Actively Recruiting
PI: Christopher DeRenzo, MD (St. Jude Children's Research Hospital) · Sites: Memphis, Tennessee · Age: 0–21 yrs
View on ClinicalTrials.gov ↗I'm interested →
Study of B7-H3-Specific CAR T Cell Locoregional Immunotherapy for Diffuse Intrinsic Pontine Glioma/Diffuse Midline Glioma and Recurrent or Refractory Pediatric Central Nervous System Tumors
Phase 1
Actively Recruiting
PI: Rebecca Ronsley, MD (Seattle Children's Hospital) · Sites: Seattle, Washington · Age: 1–26 yrs
View on ClinicalTrials.gov ↗I'm interested →

Specialists

Showing 25 of 42View all specialists →
AM
Amar Gajjar, MD
MEMPHIS, TN
Specialist
PI on 8 active trials
MM
Maryam Fouladi, MD
COLUMBUS, OH
Specialist
PI on 10 active trials
PL
Project Leader
Specialist
PI on 11 active trials
SP
Sabine Mueller, MD, PhD
Birmingham, Alabama
Specialist

Rare Disease Specialist

PI on 9 active trials
DP
Donald W Parsons
Specialist
PI on 1 active trial17 Atypical teratoid rhabdoid tumor publications
SP
Stanislaw R. Burzynski, MD, PhD
HOUSTON, TX
Specialist
PI on 29 active trials
DM
David Steffin, MD
HOUSTON, TX
Specialist
PI on 3 active trials
PM
Premal Lulla, MD
HOUSTON, TX
Specialist
PI on 4 active trials
SM
Suzanne Forrest, MD
Specialist
PI on 1 active trial
AF
Andrea T Franson
ANN ARBOR, MI
Specialist
PI on 1 active trial
MI
Meredith S Irwin
CINCINNATI, OH
Specialist
PI on 1 active trial
TM
Thomas Cash, MD
BIRMINGHAM, AL
Specialist
PI on 3 active trials
SM
Sabine Mueller, MD, PhD, MAS
SAN FRANCISCO, CA
Specialist
PI on 8 active trials
RM
Rajen Mody
ANN ARBOR, MI
Specialist
PI on 2 active trials
KM
Kim E. Nichols, MD
Memphis, Tennessee
Specialist

Rare Disease Specialist

PI on 2 active trials
JM
Jeffrey Dome, MD
WASHINGTON, DC
Specialist
PI on 1 active trial
CM
Chris DeRenzo, MD
MEMPHIS, TN
Specialist
PI on 1 active trial
CM
Corinne Summers, MD
SEATTLE, WA
Specialist
PI on 1 active trial
CM
Colleen Annesley, MD
SEATTLE, WA
Specialist
PI on 5 active trials
CM
Catherine Albert, MD
Specialist
PI on 3 active trials
JM
Jordan Milner, MD
GAINESVILLE, FL
Specialist
PI on 5 active trials
MM
Muna Qayed, MD
ATLANTA, GA
Specialist
PI on 2 active trials
IM
Ipsen Medical
Specialist
PI on 1 active trial
MM
Mariko D. DeWire-Schottmiller, MD
CINCINNATI, OH
Specialist
PI on 1 active trial
SM
Scot C. Remick, MD
SCARBOROUGH, ME
Specialist
PI on 9 active trials

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center ↗

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center ↗

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program ↗

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site ↗

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site ↗

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site ↗

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine ↗

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program ↗

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Atypical teratoid rhabdoid tumor.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Atypical teratoid rhabdoid tumor

Disease timeline:

New recruiting trial: Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Study to ONO-4538 in Patients With Rhabdoid Tumor

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Tazemetostat+Nivo/Ipi in INI1-Neg/SMARCA4-Def Tumors

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: C7R-GD2.CAR T Cells for Patients With GD2-expressing Brain Tumors (GAIL-B)

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Eflornithine (DFMO) and AMXT 1501 for Neuroblastoma, CNS Tumors, and Sarcomas

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor Expressed in T Cells for Pediatric Solid Tumors

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Immunotherapy for Malignant Pediatric Brain Tumors Employing Adoptive Cellular Therapy (IMPACT)

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

New recruiting trial: Loc3CAR: Locoregional Delivery of B7-H3-CAR T Cells for Pediatric Patients With Primary CNS Tumors

A new clinical trial is recruiting patients for Atypical teratoid rhabdoid tumor

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Atypical teratoid rhabdoid tumor

Are there clinical trials for Atypical teratoid rhabdoid tumor?

Yes — 14 recruiting clinical trials are currently listed for Atypical teratoid rhabdoid tumor on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Atypical teratoid rhabdoid tumor?

25 specialists and care centers treating Atypical teratoid rhabdoid tumor are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.