INCB7839 for Brain Cancer in Children

No longer recruiting at 18 trial locations

Overseen By Nina Butingan, MBS

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Pediatric Brain Tumor Consortium

Must not be taking: Anticancer, Investigational, Cannabinoids

Disqualifiers: Pregnancy, Breast-feeding, Hypertension, Malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 28 days per course

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called INCB7839, an experimental therapy for children with brain cancer, specifically high-grade gliomas like diffuse intrinsic pontine glioma (DIPG) that have recurred or worsened after initial treatment. The trial aims to determine the right dose and assess the treatment's safety and effectiveness. Children and teens with these tumors who have already tried other treatments might be suitable candidates for this trial. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Do I have to stop taking my current medications for the trial?

The trial does not specify if you need to stop taking your current medications, but you cannot be on any other anti-cancer, investigational, or alternative drug therapies. If you are on dexamethasone, the dose must be stable or decreasing for at least 7 days before joining the trial.

Is there any evidence suggesting that INCB7839 is likely to be safe for humans?

Research shows that INCB7839 is generally well-tolerated in children with brain cancer. Earlier studies reported mostly mild to moderate side effects. The most common issues included tiredness, nausea, loss of appetite, diarrhea, and vomiting. These side effects are typical for many treatments and, while uncomfortable, are usually manageable.

As this trial is in an early phase, researchers continue to gather safety information. The treatment's progression to this stage suggests some confidence in its safety. Always consult your doctor to determine if joining the trial is appropriate. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for brain cancer in children?

Unlike the standard treatments for brain cancer in children, which often include surgery, radiation, and chemotherapy, INCB7839 offers a different approach by targeting a specific enzyme involved in cancer cell growth. Researchers are excited about INCB7839 because it works by inhibiting ADAM10, an enzyme that plays a role in the progression of several cancers, potentially leading to more effective results with fewer side effects. This oral medication offers a novel mechanism of action, providing hope for improved outcomes in pediatric brain cancer treatments.

What evidence suggests that INCB7839 might be an effective treatment for brain cancer in children?

Research has shown that INCB7839, the investigational treatment in this trial, blocks certain proteins that help cancer cells grow. These proteins, known as ADAM10 and ADAM17, are inhibited by the drug, which can reach brain tissue and stop their activity. Other tests have demonstrated that INCB7839 can slow tumor growth when used alone. This finding offers hope for treating challenging brain tumors in children, particularly those that have returned or progressed after initial treatment. ^{1 2 3 4 5}

Who Is on the Research Team?

Michelle Monje, MD, PhD

Principal Investigator

Stanford University and Lucile Packard Children's Hospital

Are You a Good Fit for This Trial?

This trial is for children aged 3-21 with recurrent or progressive high-grade brain tumors who can swallow pills. They must have measurable disease, be recovered from prior treatments, and have stable health conditions. Those with controlled seizures or neurological deficits may qualify. Patients on birth control and HIV-positive individuals under certain criteria are also eligible.

Inclusion Criteria

I haven't taken any growth factor medications for over a week.

I have tried at least one cancer treatment other than surgery and it didn't work.

My brain tumor is aggressive and has come back or gotten worse.

Exclusion Criteria

My close family has a history of blood clots.

You are pregnant or breastfeeding.

I have not had significant new brain bleeding shown on an MRI in the last 2 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive INCB7839 for 28 consecutive days per course, with up to 26 courses (approximately 2 years) possible

28 days per course

Regular visits for dose administration and monitoring

Pharmacokinetic Studies

Pharmacokinetic studies performed after oral INCB7839 dose on days 1, 2, and 3 of Course 1

3 days

Daily visits for blood sampling

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 30 days post treatment

What Are the Treatments Tested in This Trial?

Interventions

• INCB7839

Trial Overview The trial tests INCB7839 in children with high-grade gliomas that came back or got worse after initial treatment. It's a phase 1 study to see how safe the drug is and how it affects these types of brain tumors.

How Is the Trial Designed?

Group I: Dose-finding Experimental Treatment 1 Intervention

INCB7839 dosing will begin at 120 mg/m2/dose BID which is equivalent to the adult RP2D (200 mg PO BID) based on a typical adult size of 1.67m2. The INCB7839 dose may be decreased to 80 mg/m2/dose BID if the staring dose is not tolerable. 28 consecutive days (4 weeks) will constitute one course. Patients may continue to receive INCB7839 for 26 courses (approximately 2 years).

Who Is Running the Clinical Trial?

Pediatric Brain Tumor Consortium

Lead Sponsor

Trials

38

Recruited

1,600+

American Lebanese Syrian Associated Charities (ALSAC)

Collaborator

Trials

9

Recruited

670+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

American Lebanese Syrian Associated Charities

Collaborator

Trials

9

Recruited

670+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT04295759

Study Details | NCT04295759 | INCB7839 in Treating ... In vivo testing also demonstrated that INCB7839 penetrates brain tissue sufficient to achieve its pharmacodynamic effect of ADAM10 inhibition. Further pre- ...

2. pbtc.org pbtc.org/public/Summaries/PBTC-056/PBTC-056%20v1.0%20abstract.pdf

A Phase I study of the ADAM-10 inhibitor, INCB7839 in ... In vivo testing also demonstrated that INCB7839 penetrates brain tissue sufficient to achieve its pharmacodynamic effect of ADAM10 inhibition. Further pre- ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11183952/

CNSC-02. PEDIATRIC BRAIN TUMOR CONSORTIUM (PBTC ... We report results of the clinical trial PBTC-056 (NCT04295759) evaluating safety and tolerability of INCB007839, an inhibitor of the ADAM 10 and 17 proteases.

4. withpower.com withpower.com/trial/phase-1-glioma-4-2020-dee98

INCB7839 for Brain Cancer in Children · Info for Participants This trial tests INCB7839, a drug that blocks proteins helping cancer cells grow, on children with tough-to-treat brain tumors that have returned or grown ...

5. researchgate.net researchgate.net/publication/313199385_A_multicenter_phase_Ib_study_of_the_safety_pharmacokinetics_biological_activity_and_clinical_efficacy_of_INCB7839_a_potent_and_selective_inhibitor_of_ADAM10_and_ADAM17

A multicenter phase Ib study of the safety, ... For example, potent non-selective inhibitors of ADAM17 and ADAM10, such as INCB3619 and INCB7839 (aderbasib), have shown promise in reducing tumour growth alone ...