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Recurrent Atypical Teratoid/Rhabdoid TumorPhase 2NCT07447076

Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)

A Phase 2 study testing Lumber Puncture, Blood Specimen Collection and Magnetic resonance imaging (MRI) in people with Recurrent Atypical Teratoid/Rhabdoid Tumor and Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS, run by Sabine Mueller, MD, PhD at 3 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This is a multi-treatment arm study that will be conducted through the Pacific Pediatric Neuro-oncology Consortium (PNOC).The study will assess the safety and efficacy of novel therapies and combinatorial strategies for participants with recurrent or progressive ATRT. Read more about this study

Who is it for?
Ages 1 year to 39 years
What is being tested?
Lumber Puncture, Blood Specimen Collection and Magnetic resonance imaging (MRI)
Could I receive a placebo?
No, everyone receives an active treatment
How many people
29 participants
Where
3 sitesUnited States
Study dates
17 Jun 2026to 31 Dec 2036
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 39 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • In addition to the below, investigators are to refer to arm-specific inclusion criteria in the appendix.
  • Participants must have a pathologic diagnosis of central nervous system (CNS) ATRT, with confirmation of SWI/SNF-related, matrix-associated, actin-dependent regulator of chromatin, subfamily b, member 1 (SMARCB1) (INI1) loss by immunohistochemistry (IHC) and/or biallelic loss of function of SMARCB1 by molecular report. Loss of SWI/SNF Related, Matrix Associated, Actin Dependent Regulator of Chromatin, Subfamily A, Member 4 (SMARCA4) as confirmed by IHC or molecular report is also acceptable but requires study chair approval.
  • Participants must have confirmation of methylation report, co-enrollment on PNOC-030 or sufficient tumor tissue available for methylation-based subgrouping
  • Participants must have recurrent or progressive ATRT.
  • Participants age must be ≥1 and ≤ 39 years at the time of study enrollment. Please refer to arm specific inclusion criteria for potential variations in lower age eligibility limit.
  • Prior Therapy: Participants must have fully recovered from the acute effects of prior anti-cancer therapy, and the following wash-out periods need to be observed prior to enrollment:
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  • Systemic myelosuppressive therapy: ≥ 21 days after the last dose (42 days for nitrosoureas or mitomycin C).
  • Intrathecal/intraventricular chemotherapy: > 7 days after the last dose.
  • Small molecule/targeted/biologic agent: ≥ 7 days after the last dose.
  • Monoclonal antibodies: ≥ 21 days after the last dose. Other non-myelosuppressive anti-cancer agents: ≥ 3 drug half-lives after the last dose.
  • CAR-T cell therapy (systemic or intraventricular): > 21 days.
  • Previous radiotherapy. Participants will be eligible following radiotherapy, if they meet the following criteria:
  • Previous craniospinal or total body radiotherapy: Participants must have received their last fraction ≥ 12 weeks prior to enrollment and have evidence of progressive/recurrent evaluable disease post radiation.
  • Previous focal radiotherapy to target lesions: Participants must have received their last fraction to target lesions ≥12 weeks prior to enrollment and have evidence of progressive/recurrent evaluable disease post radiation; investigators are reminded to review potentially eligible cases to avoid confusion with pseudo-progression.
  • Focal radiotherapy to non-target lesions: Participants may have received radiotherapy to nontarget lesions as long as the last fraction was > 14 days prior to enrollment. Participants must have at least one non-irradiated lesion that is evaluable for response.
  • Performance Score: Karnofsky ≥ 50 for participants > 16 years of age and Lansky ≥ 50 for participants ≤ 16 years of age. Participants who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score.
  • Corticosteroids: Participants who are receiving corticosteroids must be on a stable or decreasing dose for at least 1 week prior to enrollment. Please also refer to arm-specific inclusion criteria for potential variations in steroid limitations.
  • Organ Function Requirements.
  • Adequate Bone Marrow Function Defined as:
  • Peripheral absolute neutrophil count (ANC) ≥ 750/mm3
  • Platelet count ≥ 75,000/cubic millimeters (mm3) (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment).
  • Adequate Renal Function Defined as:
  • Serum creatinine ≤ 1.5 Upper Limit Normal (ULN) based on age and gender
  • Adequate Liver Function Defined as:
  • Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age; in presence of Gilbert's syndrome, total bilirubin < 3 x ULN or direct bilirubin < 1.5 x ULN
  • Alanine aminotransferase (ALT) ≤ 3 x ULN
  • Aspartate aminotransferase (AST) ≤ 3 x ULN
  • Adequate Neurologic Function Defined as:
  • Participants with seizure disorder may be enrolled if well controlled. See arm-specific recommendations for potential interactions between anticonvulsant agent(s) with study drug.
  • Effect on the developing human fetus Recommendations on the potential effect of interventional agents on the developing human fetus will be specified in each study arm's details of therapeutic agents. Unless otherwise specified, the effects of study interventions should be considered potentially teratogenic. Thus, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study treatment and four months after its completion. Should a woman become pregnant or suspect she is pregnant while she is participating in this study, or should a male participant's partners become pregnant during study participation, they should inform the treating physician immediately.
  • A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate.
  • Participants must enroll on Pediatric Neuro-oncology Consortium (PNOC) COMP if PNOC COMP is open to accrual at the enrolling institution.
  • Subjects must meet all inclusion criteria for the overall study.
  • Patients must be evaluable per Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria for medulloblastoma and other leptomeningeal seeding tumors to be evaluated for the primary endpoint (Warren et al. 2018); patients with evaluable but non-measurable disease, including leptomeningeal disease or positive CSF cytology only are eligible.
  • Patients with recurrent or progressive ATRT who receive surgery only for their disease progression and do not have evaluable disease may be eligible for study treatment but would not be included towards the primary efficacy endpoint (to be discussed with study chairs).
  • Subjects must be able to swallow intact capsules.
  • Adequate Metabolic Function Defined as:
  • Non-fasting glucose ≤ 140 milligrams per deciliter (mg/dL) without the use of antihyperglycemic agents.
  • If non-fasting glucose > 140 mg/dL, a fasting glucose should be done. If fasting glucose ≤ 125 mg/dL without the use of antihyperglycemic agents, participant will meet adequate metabolic function criteria.
  • Triglycerides of < 300 mg/dl and total cholesterol of < 300 mg/dl - can be on lipid lowering medications as needed to achieve.
  • Adequate Cardiac Function Defined as:
  • Electrocardiogram (ECG) must be obtained to verify the Corrected QT Interval (QTC). If an abnormal reading is obtained, the ECG should be repeated in triplicate.
  • QTC < 470 millisecond (msec)
  • Arm A

Who cannot join

  • Evidence of synchronous tumors or other extra-CNS malignancy
  • Participants who are receiving any other investigational agents
  • Participants who are currently receiving other anti-cancer agents
  • Participants with uncontrolled infection or other uncontrolled systemic illness
  • Female participants of childbearing potential who are pregnant or breast-feeding. Female participants of childbearing potential must have a negative serum or urine pregnancy test prior to the start of therapy and throughout study treatment.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition as the intended treatment regimen, as detailed in that arm's treatment description.
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  • Arm A
  • In addition to not meeting any of the exclusion criteria of the overall study, participation on arm A will also require that subjects do not meet any of the following:
  • Previous exposure to gemcitabine or paxalisib
  • Concomitant use of an antihyperglycemic agent (e.g. metformin)
  • Chronic diarrhea greater than Grade 2

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

PNOC Operations Office contact

415-502-1600

PNOC035@ucsf.edu

United States3 sites, 3 recruiting

  • University of California, San Francisco

    San Francisco, California, 94143

    PNOC Operations Office, 415-502-1600, PNOC035@ucsf.edu

    cancertrials@ucsf.edu

    Not yet recruiting

  • University of California, San Francisco

    San Francisco, California, 94143

    Sabine Mueller, MD, PhD, MAS, 877-827-3222, cancertrials@ucsf.edu

    Sabine Mueller, MD, PhD, MAS

    Recruiting

  • Seattle Children's Hospital University of Washington, Seattle

    Seattle, Washington, 98105

    Erin Crotty, MD, 206-987-2106, erin.crotty@seattlechildrens.org

    Erin Crotty, MD

    Recruiting

Other trials in United States

About this study

Official title: A Platform Study of Novel Therapies for Children, Adolescents and Young Adults With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT)

This is a multi-treatment arm study that will be conducted through the Pacific Pediatric Neuro-oncology Consortium (PNOC).The study will assess the safety and efficacy of novel therapies and combinatorial strategies for participants with recurrent or progressive ATRT.

Read the detailed description

PRIMARY OBJECTIVES:

  1. To assess efficacy of each treatment arm based on arm-specific endpoints. II. To evaluate the safety and tolerability of each treatment arm for patients with recurrent/progressive ATRT.

EXPLORATORY OBJECTIVES:

  1. To collect additional treatment arm-specific safety and tolerability information as applicable.

II. To determine additional treatment arm-specific time-to-event endpoints. III. To assess the correlations between methylation-based subgroups and objective response and outcome measures.

IV. To collect biologic samples (tumor, blood, CSF) for future biomarker discovery.

  1. Additional treatment arm-specific exploratory objectives may be added in each interventional arm.

OUTLINE: Participants will enroll onto a treatment arm as treatment arms open for enrollment. Participants who are taken off treatment on one trial arm for progression or toxicity may enroll on a new treatment arm, if arm-specific eligibility criteria are met. If the study is opened at the treating institution, participants will be followed under the Pediatric Neuro-oncology Consortium (PNOC) COMP protocol until death or withdrawal from study.

Being tested
Lumber Puncture, Blood Specimen Collection, Magnetic resonance imaging (MRI), Gemcitabine, Paxalisib
Keywords
Novel therapies
Medical subject headings
Rhabdoid Tumor

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Lumber PunctureProcedure or surgery

    Cerebral Spinal Fluid (CSF) will be collected for research

  • Blood Specimen CollectionProcedure or surgery

    Perform blood draw

  • Magnetic resonance imaging (MRI)Procedure or surgery

    Undergo imaging procedure

  • GemcitabineDrug

    Given IV

  • PaxalisibDrug

    Given orally (PO)

  • Tumor BiopsyProcedure or surgery

    Undergo biopsy

Study groups in NCT07447076
Study groupRoleWhat they receive
Treatment Arm A: Phase IExperimentalParticipants will receive a combination of gemcitabine and paxalisib with starting dose, 1,200mg/m2 gemcitabine and 15mg/m2 paxalisib (phase I). If this dose level is tolerated, it will be confirmed as the RP2D. However, if the dose limiting toxicity (DLT) is not tolerated, the RP2D will be set at the highest dose level tolerated by the previous group.
Receives: Procedure: Lumber Puncture; Procedure: Blood Specimen Collection; Procedure: Magnetic resonance imaging (MRI); Drug: Gemcitabine; Drug: Paxalisib; Procedure: Tumor Biopsy
Treatment Arm A: Efficacy (Phase II)ExperimentalAll participants will receive a combination of gemcitabine and paxalisib over a 28-day cycle, for up to 24 cycles or until they meet criteria to stop treatment, whichever comes first. Gemcitabine will be administered via infusion on days 1, 8, and 15 of each cycle, while paxalisib will be taken daily throughout each cycle.
Receives: Procedure: Lumber Puncture; Procedure: Blood Specimen Collection; Procedure: Magnetic resonance imaging (MRI); Drug: Gemcitabine; Drug: Paxalisib; Procedure: Tumor Biopsy

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT)

    Tolerability is defined as the proportion of participants receiving at least one dose of combination gemcitabine and paxalisib with a reported dose-limiting toxicity (DLT) during cycle 1 for all participants in Phase I.

    Measured: up to 28 days

  2. Arm A (Phase I): Recommended Phase 2 Dose (RP2D) (Phase I)

    The confirmed RP2D of combination gemcitabine and paxalisib implemented for participants enrolled in Phase II will be reported.

    Measured: up to 28 days

  3. Arm A (Phase II): Rate of Clinical Benefit

    Assess the efficacy of combination gemcitabine and paxalisib for participants in Phase II. Clinical Benefit rate (CBR) is defined as complete response (CR) + partial response (PR) + stable disease (SD), where SD is sustained over 4 months.

    Measured: up to 2 years (24 cycles)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Mar 2026
  2. Study start17 Jun 2026
  3. Primary completion31 Dec 2035 (estimated)
  4. Study completion31 Dec 2036 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
29 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07447076
Sponsor's protocol ID
25083
Status last verified
Oct 2026
First submitted
25 Feb 2026
Last updated
27 Oct 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Lumber Puncture, Blood Specimen Collection and Magnetic resonance imaging (MRI) in people with Recurrent Atypical Teratoid/Rhabdoid Tumor and Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS, run by Sabine Mueller, MD, PhD at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 44 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact PNOC Operations Office (415-502-1600, PNOC035@ucsf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT) (time frame: up to 28 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07447076.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.

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