APX005M for Pediatric Brain Cancer

Not currently recruiting at 11 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and ideal dosage of APX005M, a treatment for certain pediatric brain cancers. It focuses on children with brain tumors that are worsening or have returned, and those newly diagnosed with a type of brainstem tumor called diffuse intrinsic pontine glioma (DIPG). APX005M is designed to activate specific immune cells and directly attack tumor cells. Children with a worsening brain tumor or a new diagnosis of DIPG, who have completed radiation therapy, might be a good fit for this study. As a Phase 1 trial, this research seeks to understand how APX005M works in people, offering participants the opportunity to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that APX005M is likely to be safe for humans?

In a previous study, researchers examined the safety of APX005M for young patients with brain tumors. They found that the treatment was generally well-tolerated, with most side effects being mild to moderate. These side effects included tiredness, fever, and changes in blood cell counts, which are common with many cancer treatments. It is important to note that this study remains in its early stages, so more research is needed to fully understand all possible side effects.

APX005M is being tested because it can activate immune cells, like B cells and dendritic cells, to directly fight tumor cells. While this is promising, it also means the treatment might cause reactions as the immune system activates. So far, serious side effects have been rare. However, as with all new treatments, researchers will closely monitor participants for any unexpected reactions. ^{1 2 3 4 5}

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

Most treatments for pediatric brain cancer, like chemotherapy and radiation, aim to kill cancer cells directly but can affect healthy brain tissue too. APX005M stands out because it works by activating the body's immune system to specifically target and destroy cancer cells, potentially reducing harm to healthy tissue. Researchers are excited about APX005M because it represents a novel approach with the possibility of more precise treatment and fewer side effects compared to traditional therapies. This targeted immune response could lead to better outcomes for children with difficult-to-treat brain tumors.

What evidence suggests that APX005M might be an effective treatment for pediatric brain cancer?

Research has shown that APX005M can help the immune system fight cancer by activating certain cells within the body's defense system. This activation releases proteins called cytokines, which signal the immune system to attack. APX005M may also directly kill cancer cells with a specific marker called CD40. Although limited information exists on its effectiveness for brain tumors in children, these actions suggest it could enhance the body's natural defenses against cancer. Participants in this trial will receive APX005M, with Stratum 1 focusing on recurrent, progressive, or refractory primary malignant non-brainstem CNS tumors, and Stratum 2 focusing on newly diagnosed diffuse intrinsic pontine gliomas (DIPGs). ^{1 2 6 7 8}

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

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

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. clinicaltrials.gov clinicaltrials.gov/study/NCT03389802?term=APX005M&

Phase I Study of APX005M in Pediatric Central Nervous ... 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 ...

2. med.stanford.edu med.stanford.edu/mosslab/research/_jcr_content/main/clinicaltrials.html?ctid=NCT03389802&conditionId=&serviceLineId=&condition=&alpha=p

Eligibility - Stanford Medicine 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 ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6639749/

Role of Radiation Therapy in the Management of Diffuse ... Diffuse intrinsic pontine glioma (DIPG) is the most aggressive primary pediatric brain tumor, with <10% of children surviving 2 years.

4. fda.gov fda.gov/media/105924/download

APX005M - Apexigen, Inc. In general, pediatric patients with CNS tumors do not share the favorable prognosis of those with other common pediatric malignancies. Patients with newly ...

5. georgiacancerinfo.org georgiacancerinfo.org/clinical-trials/detail/4672

CD40 Agonistic Monoclonal Antibody APX005M in ... This phase I trial studies the side effects and best dose of CD40 agonistic monoclonal antibody APX005M in treating pediatric patients with brain tumors that ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11106442/

Current immunotherapeutic approaches to diffuse intrinsic ... Diffuse intrinsic pontine glioma (DIPG) is an aggressive brain tumour that occurs in the pons of the brainstem and accounts for over 80% of all ...

7. pbtc.org pbtc.org/public/PBTC-051%20Protocol%20Abstract%20online.pdf

NCI Protocol # PBTC-051 1 Phase I Study to Evaluate the ... The primary objectives of the study are to (1) evaluate the safety of APX005M administered intravenously every 3 weeks to children with central nervous system ...

8. sciencedirect.com sciencedirect.com/science/article/pii/S016836592400302X

Current status and advances to improving drug delivery in ... Among pediatric cancers, brain tumors are the leading cause of morbidity and mortality, representing approximately 40% of all cancer-related deaths [2,3].