New drug combo targets brain tumors with genetic flaw
NCT ID NCT04192981
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
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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21 people
The number who actually took part.
- Started
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Dec 2019
- Expected to finish
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Dec 2026
An estimate. End dates often move.
- Lead sponsor
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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
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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
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BAPTIST ALLIANCE - MCI (Data Collection Only)
Miami, Florida, 33143, United States
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Memorial Sloan Kettering Basking Ridge (Consent only)
Basking Ridge, New Jersey, 07920, United States
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Memorial Sloan Kettering Bergen (Consent only)
Montvale, New Jersey, 07645, United States
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Memorial Sloan Kettering Cancer Center (All protocol activities)
New York, New York, 10065, United States
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Memorial Sloan Kettering Commack (Consent only)
Commack, New York, 11725, United States
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Memorial Sloan Kettering Monmouth (All protocol activities)
Middletown, New Jersey, 07748, United States
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Memorial Sloan Kettering Nassau (Consent only)
Uniondale, New York, 11553, United States
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Memorial Sloan Kettering Westchester (All protocol activities)
Harrison, New York, 10604, United States
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University of Washington (Data Collection AND Data Analysis)
Seattle, Washington, 98109, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a single day of brain radiation match five days?
- Can doctors watch brain metastases instead of zapping them?
- Starving brain tumors of blood before surgery
- Tiny gadolinium particles aim to make brain radiation hit harder
- Radioactive nanoparticles aimed at cancer that spreads to the Brain's lining
- A sharper MRI could reveal radiation sensitivity of brain tumors