Tocilizumab for Craniopharyngioma

Recruiting in Palo Alto (17 mi)

Overseen by Margaret E. Macy

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase < 1

Recruiting

Sponsor: University of Colorado, Denver

Disqualifiers: Pregnant, Active infection, Heart failure, others

No Placebo Group

Breakthrough Therapy

Approved in 6 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial tests if tocilizumab, an anti-inflammatory drug, can reach and affect brain tumors in patients with craniopharyngioma. If successful, patients will receive the drug regularly over several months to see if it helps control their tumors.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is tocilizumab generally safe for humans?

Tocilizumab, also known as Actemra or RoActemra, has been studied for various conditions and is generally considered safe, though it can cause side effects like infections, decreased white blood cells, and elevated liver enzymes. In clinical trials, common side effects included decreased neutrophils (a type of white blood cell), infections, and increased liver enzymes, but these were generally manageable. ^{1 2 3 4 5}

How does the drug tocilizumab differ from other treatments for craniopharyngioma?

Tocilizumab is unique because it is a monoclonal antibody that targets the interleukin-6 receptor, which is involved in immune system regulation and inflammation. This mechanism is different from traditional treatments for craniopharyngioma, which typically involve surgery or radiation. ^{1 2 6 7 8}

Research Team

Margaret E. Macy

Principal Investigator

Children's Hospital Colorado

Eligibility Criteria

This trial is for children and young adults (2 to <21 years old) with a type of brain tumor called craniopharyngioma. It's open to those with new, recurrent, or progressive tumors that haven't been fully removed by surgery or have come back after treatment. Participants need functioning major organs and acceptable blood counts, must not be pregnant or breastfeeding, and should agree to use birth control.

Inclusion Criteria

My kidney function, based on my age and gender, meets the required levels.

My kidney function is normal.

Subjects of childbearing or child fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study.

Exclusion Criteria

Pregnant or breastfeeding

I have an irregular heartbeat.

psychiatric illness/social situations that would limit compliance with study requirements are not eligible.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Phase 0

Patients receive one dose of tocilizumab before SOC surgery to assess drug penetration in the tumor

1 day

1 visit (in-person)

Feasibility Phase

Patients receive tocilizumab every two weeks for up to 13 cycles (approximately 1 year)

1 year

26 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

5 years

Treatment Details

Interventions

• Tocilizumab (Monoclonal Antibodies)

Trial Overview The study tests Tocilizumab in two phases: Phase 0 checks if the drug reaches the tumor when given before standard surgery. If successful, Phase 1 gives Tocilizumab every two weeks for up to a year to see how well it works as a treatment. Patients can move from Phase 0 to Phase 1 and will be monitored for five years.

Participant Groups

Group I: Tocilizumab Administration: Phase 0 Experimental Treatment 1 Intervention

In Phase 0, patients will receive one dose of tocilizumab prior to surgery.

Group II: Tocilizumab Administration: Feasibility Phase Experimental Treatment 1 Intervention

During the Feasibility Phase, patients will receive tocilizumab every 2 weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years.

Tocilizumab is already approved in Canada, Japan for the following indications:

Approved in Canada as Actemra for:

• Rheumatoid Arthritis
• Systemic Juvenile Idiopathic Arthritis
• Polyarticular Juvenile Idiopathic Arthritis
• Giant Cell Arteritis
• Cytokine Release Syndrome
• COVID-19

Approved in Japan as Actemra for:

• Rheumatoid Arthritis
• Systemic Juvenile Idiopathic Arthritis
• Polyarticular Juvenile Idiopathic Arthritis
• Giant Cell Arteritis
• Cytokine Release Syndrome
• COVID-19

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Children's Hospital Colorado Aurora, CO

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials

1842

Patients Recruited

3,028,000+

National Cancer Institute (NCI)

Collaborator

Trials

14080

Patients Recruited

41,180,000+

Findings from Research

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab. Venkiteshwaran, A.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tocilizumab: a review of its use in the treatment of juvenile idiopathic arthritis. Frampton, JE.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Phase I Clinical Study Comparing the Tolerance, Immunogenicity, and Pharmacokinetics of Proposed Biosimilar BAT1806 and Reference Tocilizumab in Healthy Chinese Men. Zhang, H., Wang, H., Wei, H., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and safety of candidate tocilizumab biosimilar CT-P47 versus reference tocilizumab: a randomized, double-blind, single-dose phase I study. Yu, KS., Kim, B., Shin, D., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A phase I study comparing the pharmacokinetics and safety of HS628 (tocilizumab biosimilar) and reference tocilizumab in healthy male subjects. Qian, H., Cheng, J., Gui, Y., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Persistent aphthous mouth ulcers associated with tocilizumab: two cases]. Samimi, M., Lauferon, F., Hüttenberger, B., et al.[2016]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Intravenous tocilizumab: a review of its use in adults with rheumatoid arthritis. Dhillon, S.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Off-label use of tocilizumab to treat non-juvenile idiopathic arthritis in pediatric rheumatic patients: a literature review. Jung, JY., Kim, MY., Suh, CH., et al.[2020]

References

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

Tocilizumab. [2021]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Tocilizumab: a review of its use in the treatment of juvenile idiopathic arthritis. [2021]

3. Switzerland pubmed.ncbi.nlm.nih.gov

A Phase I Clinical Study Comparing the Tolerance, Immunogenicity, and Pharmacokinetics of Proposed Biosimilar BAT1806 and Reference Tocilizumab in Healthy Chinese Men. [2021]

4. England pubmed.ncbi.nlm.nih.gov

Pharmacokinetics and safety of candidate tocilizumab biosimilar CT-P47 versus reference tocilizumab: a randomized, double-blind, single-dose phase I study. [2023]

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

A phase I study comparing the pharmacokinetics and safety of HS628 (tocilizumab biosimilar) and reference tocilizumab in healthy male subjects. [2023]

6. France pubmed.ncbi.nlm.nih.gov

[Persistent aphthous mouth ulcers associated with tocilizumab: two cases]. [2016]

7. New Zealand pubmed.ncbi.nlm.nih.gov

Intravenous tocilizumab: a review of its use in adults with rheumatoid arthritis. [2022]

8. England pubmed.ncbi.nlm.nih.gov

Off-label use of tocilizumab to treat non-juvenile idiopathic arthritis in pediatric rheumatic patients: a literature review. [2020]