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New drug combo targets brain tumors with genetic flaw

NCT ID NCT04192981

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage study tests a drug called GDC-0084 given alongside radiation therapy for people whose cancer has spread to the brain and has a specific genetic mutation (PI3K). The main goal is to find the safest dose of the drug and see if the combination helps shrink tumors. Only 21 participants are enrolled, so results are very preliminary.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
GDC-0084 (a drug that targets PI3K pathway mutations) combined with whole brain radiation therapy
What this could lead to
If it works, this could point toward a new treatment option for people with brain metastases that have specific genetic mutations.
What could go wrong
This is a very early phase 1 trial with only 21 participants, so it is primarily testing safety and dosing. The combination may not prove effective against the cancer, and side effects could be significant.

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

21 people

The number who actually took part.

Started

Dec 2019

Expected to finish

Dec 2026

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

18 years and older

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: * Histologically confirmed solid tumor malignancies harboring PIK3CA mutations which include mutations in any of the following genes: PIK3CA, PIK3CB, PIK3CD, PIK3CG, PIK3R1, PIK3R2, PIK3R3, PIK3C2G, PIK3C3, INPP4A, INPP4B, INPPL1, INPP5D, PTEN, AKT1, AKT2, AKT3, and MTOR. * Brain metastases and/or leptomeningeal metastases involving the brain demonstrated by MR imaging of the brain. For patients with brain metastases, measurable lesion by RANO-BM is required28. Patients with spine leptomeningeal metastases are eligible for the study if they have leptomeningeal metastases of the brain demonstrated by MRI imaging of the brain. * KPS ≥ 70 * Age ≥ 18 years * Able to provide informed consent. * If a patient is on corticosteroid, he/she must be on a stable daily dose of \< 4mg dexamethasone or equivalent. Patient does not need to be given corticosteroid as prophylaxis if not clinically indicated. * No limit to prior therapies with the last systemic therapy ≥ 1 week from initiation of protocol therapy. Systemic therapy can resume after completion of protocol DLT assessment period. * Patients with prior SRS are eligible, provided that there are new, non-irradiated brain lesions or leptomeningeal metastases. Patients must be ≥ 3 months post prior cranial radiation therapy. * Patients with seizure history related to brain metastases or leptomeningeal metastases controlled on antiepileptic medications are eligible. * Patient at reproductive potential must agree to practice an effective contraceptive method * Patient must be able to swallow and retain oral medication * Adequate organ function as assessed by laboratory tests. * Adequate bone marrow function * Hemoglobin ≥ 8g/dL * Absolute neutrophil count ≥1,000/mm\^3 * Platelet count ≥ 100,000/mm\^3 * Adequate liver function * Bilirubin ≤1.5 times upper limit normal (ULN) * AST and ALT ≤ 2.5 times ULN * Alkaline phosphatase ≤ 2 times ULN * Adequate renal function ° BUN and Creatinine ≤ 1.5 times ULN Exclusion Criteria: * Previous radiotherapy to the intended treatment site that precludes developing a treatment plan that respects tissue tolerances * Patients with brain metastases eligible for single fraction stereotactic radiation therapy * Serious medical co-morbidities precluding radiotherapy * Insulin-treated diabetes; subjects with diabetes or impaired glucose tolerance that is not treated with insulin may be enrolled * QT interval ≥ 450 msec on EKG * Cardiac dysfunction defined as: myocardial infarction within 6 months of study entry, NYHA Class II/III/IV heart failure, unstable angina or unstable cardiac arrhythmias * Known hypersensitivity or intolerance to GDC-0084 or to any other inhibitor of the PI3K/ Akt/ mTOR pathway * Past medical history of interstitial lung disease, drug-induced ILD, radiation pneumonitis that required steroid treatment, or any evidence of clinically active interstitial lung disease * Subject receiving any medications or substances that are moderate and/or potent enzyme inducers or inhibitors which may have an effect on the metabolism of GDC-0084. * Pregnant or lactating women.

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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

  • BAPTIST ALLIANCE - MCI (Data Collection Only)

    Miami, Florida, 33143, United States

  • Memorial Sloan Kettering Basking Ridge (Consent only)

    Basking Ridge, New Jersey, 07920, United States

  • Memorial Sloan Kettering Bergen (Consent only)

    Montvale, New Jersey, 07645, United States

  • Memorial Sloan Kettering Cancer Center (All protocol activities)

    New York, New York, 10065, United States

  • Memorial Sloan Kettering Commack (Consent only)

    Commack, New York, 11725, United States

  • Memorial Sloan Kettering Monmouth (All protocol activities)

    Middletown, New Jersey, 07748, United States

  • Memorial Sloan Kettering Nassau (Consent only)

    Uniondale, New York, 11553, United States

  • Memorial Sloan Kettering Westchester (All protocol activities)

    Harrison, New York, 10604, United States

  • University of Washington (Data Collection AND Data Analysis)

    Seattle, Washington, 98109, United States

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