APX005M for Pediatric Brain Cancer

Recruiting at 10 trial locations

Overseen By Nina Butingan, MBS

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Pediatric Brain Tumor Consortium

Must not be taking: Corticosteroids, Immunosuppressives

Disqualifiers: Systemic illness, Bulky tumor, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

32 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This phase I trial studies the side effects and best dose of APX005M in treating younger patients with primary malignant central nervous system tumor that is growing, spreading, or getting worse (progressive), or newly diagnosed diffuse intrinsic pontine glioma. APX005M can trigger activation of B cells, monocytes, and dendritic cells and stimulate cytokine release from lymphocytes and monocytes. APX005M can mediate a direct cytotoxic effect on CD40+ tumor cells.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but it does exclude patients who are on certain anticancer or investigational drugs, and those requiring systemic corticosteroids or immunosuppressive medications within 14 days of the study drug. It's best to discuss your specific medications with the trial team.

How does the drug APX005M differ from other treatments for pediatric brain cancer?

APX005M is unique because it targets specific molecular alterations in pediatric brain tumors, such as the BRAF pathway, which is involved in cell growth and survival. This targeted approach may offer a more precise treatment option compared to traditional chemotherapy and radiation, which are less specific and can have more side effects. ^{1 2 3 4 5}

Who Is on the Research Team?

Ira Dunkel

Principal Investigator

Memorial Sloan Kettering Cancer Center

Are You a Good Fit for This Trial?

This trial is for children and young adults aged 1 to 21 with primary malignant non-brainstem CNS tumors that are recurrent, progressive, or refractory, or those newly diagnosed with DIPG. Participants must have stable neurological deficits, adequate organ function, not be pregnant or breastfeeding, use effective contraception methods if applicable, and meet specific recovery criteria from previous treatments.

Inclusion Criteria

I have a newly diagnosed brainstem tumor without needing a biopsy.

My cancer in the central nervous system is returning, worsening, or not responding to treatment.

I have recovered from side effects of my previous cancer treatments.

Exclusion Criteria

I have had a severe allergic reaction to a monoclonal antibody treatment.

I do not have any serious illnesses or infections that could affect my participation.

I've been cancer-free for 5 years, except for a secondary brain tumor.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive APX005M to determine the safety, tolerability, and optimal dosing in pediatric patients with CNS tumors

6 weeks

Multiple visits for dose administration and monitoring

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 weeks

Regular visits for pharmacokinetic studies and safety assessments

Long-term Follow-up

Participants are monitored for overall survival and progression-free survival

Up to 2 years

What Are the Treatments Tested in This Trial?

Interventions

• APX005M

Trial Overview The trial tests APX005M's safety and optimal dosage in treating aggressive brain tumors in the young. It activates immune cells and may directly kill tumor cells expressing CD40. The study includes patients with new diagnoses of diffuse intrinsic pontine glioma (DIPG) as well as those with recurring or resistant CNS tumors.

How Is the Trial Designed?

Group I: Stratum 2 Experimental Treatment 1 Intervention

The newly diagnosed diffuse intrinsic pontine gliomas (DIPGs) patients will be treated with APX005M.

Group II: Stratum 1 Experimental Treatment 1 Intervention

The recurrent, progressive, or refractory primary malignant non-brainstem CNS tumor patients will be treated with APX005M.

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+

Pyxis Oncology, Inc

Industry Sponsor

Trials

4

Recruited

400+

National Cancer Institute (NCI)

Collaborator

Trials

14,080

Recruited

41,180,000+

Apexigen America, Inc.

Industry Sponsor

Trials

12

Recruited

630+

American Lebanese Syrian Associated Charities

Collaborator

Trials

9

Recruited

670+

Ty Louis Campbell Foundation

Collaborator

Trials

1

Recruited

30+

A Kids' Brain Tumor Cure Foundation

Collaborator

Trials

1

Recruited

30+

Apexigen, Inc.

Industry Sponsor

Trials

12

Recruited

630+

Solving Kids' Cancer

Collaborator

Trials

11

Recruited

200+

Citations

1. England pubmed.ncbi.nlm.nih.gov

Human induced pluripotent stem cell engineering establishes a humanized mouse platform for pediatric low-grade glioma modeling. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Gliomas in children. [2021]

3. United States pubmed.ncbi.nlm.nih.gov

The therapy of infantile malignant brain tumors: current status? [2018]

4. United States pubmed.ncbi.nlm.nih.gov

Targeting BRAF in pediatric brain tumors. [2018]

5. France pubmed.ncbi.nlm.nih.gov

[Determination of target volume in pediatric radiotherapy: application to brain tumors]. [2019]