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New hope for young brain tumor patients: drug combo targets IDH mutations

NCT ID NCT03749187

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tests a new drug (BGB-290) combined with chemotherapy (temozolomide) in teens and young adults with a type of brain tumor called glioma that has an IDH mutation. The goal is to find the safest dose and see how well the combination works for both newly diagnosed and recurrent tumors. About 78 participants will be enrolled.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 78 people

The number the study aims to enrol. It can still change while the study runs.

Started

Apr 2019

Expected to finish

Jul 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

13 to 39 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Arm A Only: Participants must have histologically confirmed World Health Organization (WHO) grade III-IV newly diagnosed IDH1/2-mutant glioma. * Arm B Only: WHO grades I-IV recurrent IDH1/2 mutant glioma. Participants in Arm B must have magnetic resonance imaging (MRI) confirming progressive disease; re-biopsy is encouraged, but not required at the time of recurrence for confirmation. * Participants with a primary spinal tumor, secondary glioma, or multifocal disease in the brain, but without evidence of diffuse leptomeningeal spread, are eligible. In cases where there are questions about multifocality versus diffuse leptomeningeal spread, the study chair or co-chair must be contacted to make a final decision on eligibility. * Participants must have IDH1 or IDH2 mutation associated with neomorphic activity of the encoded proteins. * Participants must be willing to provide archival formalin-fixed embedded (FFPE) and frozen tissue specimens for biomarker studies if available. * Participants in Arm A must have been treated with maximal safe resection of primary tumor followed by adjuvant radiation therapy (RT). Treatment with TMZ during radiation is allowed but not required. * Participants in Arm B must have been treated with maximal safe resection of tumor. * Lower grade glioma (LGG) participants who progressed after initial surgery alone are eligible. Any number of prior therapies are allowed. * High grade glioma (HGG) participants enrolled on Arm B must have been treated with a minimum of maximal safe resection of primary tumor followed by adjuvant RT prior to recurrence. Any number of prior therapies are allowed. * Participants must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study. * Myelosuppressive chemotherapy: participants must have received their last dose of known myelosuppressive anticancer chemotherapy at least three weeks prior to study registration or at least six weeks if nitrosourea. * Biologic agent: participants must have recovered from any toxicity related to biologic agents and received their last dose \>= 7 days prior to study registration. * For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval should be discussed with the study chair. * For biologic agents that have a prolonged half-life, the appropriate interval since last treatment should be discussed with the study chair prior to registration. * Monoclonal antibody treatment: at least three half-lives must have elapsed prior to registration, and participants on bevacizumab must have received their last dose \>= 32 days prior to study registration. * Participants in Arm A should begin therapy with TMZ and BGB-290 after completion of radiation therapy and when all other eligibility criteria are met. * For participants in Arm B, patients must not have received radiation therapy within 4 weeks prior to the initiation of study treatment. Post-RT, the diagnosis of true progression versus pseudo-progression can be challenging when imaging modalities are exclusively used, and thus an additional resection is encouraged if clinically indicated. * Peripheral absolute neutrophil count (ANC) \>= 1000/mm\^3. * Platelet count \>= 100,000/mm\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment). * Hemoglobin \>= 9 g/dL. * Serum creatinine =\< 1.5 x upper limit of normal (ULN) or estimated creatinine clearance \>= 50 mL/min (calculated using the institutional standard method). * Total serum bilirubin (sum of conjugated + unconjugated) =\< 1.5 x upper limit of normal (ULN). * Aspartate and alanine aminotransferase (AST and ALT) =\< 3 x ULN. * Serum albumin \>= 2 g/dL. * Participants with seizure disorder may be enrolled if on non-enzyme inducing anticonvulsants and well controlled. * Participants who have neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration. * Corticosteroids: Participants who are receiving dexamethasone must be on a stable or decreasing dose for at least 1 week prior to registration. * The effects of BGB-290 on the developing human fetus are unknown. For this reason and because alkylating agents (such as TMZ) are known to be teratogenic, 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 participation and 4 months after completion of BGB-290 or TMZ administration. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. * Participants must be able to swallow capsules. * Participants must have the ability to undergo serial MRI scans (computerized tomography \[CT\] cannot substitute for MRI). * A legal parent/guardian or patient must be able to understand, and willing to sign, a written informed consent and assent document, as appropriate. * 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. Exclusion Criteria: * Participants who are receiving any other investigational agents at any time may not be enrolled. * Participants who have received a PARP inhibitor previously. * Participants with active infection requiring antibiotics at time of therapy start. * Participants with other diagnosis of malignancy. * Participants with clinically significant active bleeding disorder, hemoptysis, or melena =\< 6 months prior to day 1. * Participants on therapeutic anti-coagulation with heparin, warfarin, or other anticoagulants: * Use of low-dose aspirin and/or non-steroidal anti-inflammatory agents are allowed. * Use of thrombolytic to establish patency of indwelling venous catheters is allowed. * Prophylactic anticoagulation for venous access devices is allowed as long as institutional normalized ratio (INR) is =\< 1.5 and partial thromboplastin time (aPTT) =\< 1.5 x institutional ULN. * Use of low-molecular weight heparin is allowed. * Participants with known disseminated leptomeningeal disease. * Participants with diffuse intrinsic pontine glioma (DIPG) are not eligible for this study. * Unresolved acute effects of any prior therapy of grade \>= 2, except for adverse events (AEs) not constituting a safety risk by investigator judgement. * Use =\< 10 days (or =\< 5 half-lives, whichever is shorter) prior to day 1 or anticipated need for food or drugs known to be strong or moderate Cytochrome P450, family 3, subfamily A (CYP3A) inhibitors or strong CYP3A inducers. * History of allergic reactions attributed to compounds of similar chemical or biologic composition to TMZ or pamiparib (BGB-290). * Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. * Female participants of childbearing potential must not be pregnant or breast-feeding. Female participants of childbearing potential must have a negative serum or urine pregnancy test within 7 days of first dose. * Human immunodeficiency virus (HIV)-positive participants on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with pamiparib (BGB-290) and TMZ. In addition, these participants are at increased risk of lethal infections when treated with marrow-suppressive therapy. * Participants with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Children's Hospital Los Angeles

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Johns Hopkins University/Sidney Kimmel Cancer Center

    Baltimore, Maryland, 21287, United States

  • Joseph M. Sanzari Children's Hospital at Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • University of California, San Francisco

    San Francisco, California, 94143, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

  • Yale University

    New Haven, Connecticut, 06520, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.