Tocilizumab for Craniopharyngioma

Great Ormond Street Hospital, London, United Kingdom
Craniopharyngioma+1 More ConditionsTocilizumab - Drug
1 - 25
All Sexes

Study Summary

This trial will test the use of ACTEMRA to treat kids with a rare brain tumor who have already had surgery and/or radiation.

Eligible Conditions
  • Adamantinomatous Craniopharyngioma
  • Recurrent Adamantinomatous Craniopharyngioma

Video Summary

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 2

Study Objectives

2 Primary · 4 Secondary · Reporting Duration: 12 months

12 months
PFS of ACP patients treated with tocilizumab after radiation
PFS of ACP patients treated with tocilizumab who have not received radiation
Day 30
Biological effects of tocilizumab on ACP tumor tissue and cyst fluid.
Sustained objective response rate of patients with measurable ACP who have undergone surgery but have not been previously treated with radiation to treatment with systemic tocilizumab
Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with systemic tocilizumab
Toxicities associated with tocilizumab in children with ACP

Trial Safety

Phase-Based Safety

2 of 3
This is further along than 68% of similar trials

Side Effects for

11%Low ANC
5%Inpatient admission
This histogram enumerates side effects from a completed 2017 Phase 4 trial (NCT02034474) in the Tocilizumab ARM group. Side effects include: Low ANC with 11%, fatigue with 5%, Inpatient admission with 5%, nausea with 5%, agranulocytosis with 5%.

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.

Trial Design

1 Treatment Group

Stratum 1 and Stratum 2
1 of 1

Experimental Treatment

38 Total Participants · 1 Treatment Group

Primary Treatment: Tocilizumab · No Placebo Group · Phase 2

Stratum 1 and Stratum 2
Experimental Group · 1 Intervention: Tocilizumab · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
Completed Phase 4

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months

Who is running the clinical trial?

Nationwide Children's HospitalLead Sponsor
319 Previous Clinical Trials
5,217,558 Total Patients Enrolled
1 Trials studying Craniopharyngioma
38 Patients Enrolled for Craniopharyngioma
Children's Hospital ColoradoOTHER
106 Previous Clinical Trials
5,127,000 Total Patients Enrolled
1 Trials studying Craniopharyngioma
38 Patients Enrolled for Craniopharyngioma
Maryam Fouladi, MDPrincipal InvestigatorNationwide Children's Hospital
8 Previous Clinical Trials
2,337 Total Patients Enrolled
Todd C Hankinson, MDStudy ChairChildren's Hospital Colorado
Kathleen H Dorris, MDStudy ChairChildren's Hospital Colorado

Eligibility Criteria

Age 1 - 25 · All Participants · 3 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Your neurological condition should be stable for at least a week before enrolling in the study. If you have a seizure disorder, you can participate if your seizures are well-controlled with medication.
Stratum 1: You have a type of brain tumor called ACP that has come back or gotten worse at least 6 months after radiation therapy, and it has both cystic and solid components. Stratum 2: You have measurable ACP and you have had surgery but haven't received radiation therapy yet. It's okay if your tumor is getting worse, but it's not required.
You received radiation treatment more than 6 months ago for a specific area, but not for your entire head and spine.

Who else is applying?

What state do they live in?
What portion of applicants met pre-screening criteria?
Met criteria100.0%

Frequently Asked Questions

How many sites are participating in this clinical trial?

"Presently, this trial is open to patients at Duke University Health System, Cincinnati Children's Hospital Medical Center, Seattle Children's Hospital, and a total of 12 other hospitals." - Anonymous Online Contributor

Unverified Answer

Can young adults between the ages of 18 and 29 participate in this research?

"For this particular clinical trial, only individuals aged 1 year to 25 are eligible. Out of the 12 total clinical trials, 9 are for patients under 18 years old and 3 recruit adults over 65." - Anonymous Online Contributor

Unverified Answer

Are new participants still being accepted for this trial?

"This particular clinical trial, which was created on November 30th 2022, is not presently recruiting patients. That being said, there are 11 other trials that potential participants could look into." - Anonymous Online Contributor

Unverified Answer

What are the goals of this research?

"The primary goal of this 24-month study is to observe the efficacy of tocilizumab in patients with radiation-resistant ACP. Secondary objectives include observing one-year progression-free survival rates for (1) patients that have not received radiation and (2) those that have, as well as measuring the concentration of certain biomarkers in tumor tissue, cyst fluid, or blood." - Anonymous Online Contributor

Unverified Answer

Does Tocilizumab have a good safety profile for human use?

"While there is some evidence to support the safety of Tocilizumab, it only received a score of 2 because this is a Phase 2 trial and no efficacy data has been collected yet." - Anonymous Online Contributor

Unverified Answer

How can I sign up to participate in this research?

"Eligible patients for this clinical trial must have adamantinous craniopharyngioma and be between 1 year old to 25 years old. In total, the study will allow 38 participants." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.