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
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
This is further along than 68% of similar trials
Side Effects for
Tocilizumab
11%Low ANC
5%fatigue
5%Inpatient admission
5%nausea
5%agranulocytosis
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
Drug
Experimental Group · 1 Intervention: Tocilizumab · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Tocilizumab
2012
Completed Phase 4
~1840
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?
| Iowa | 100.0% |
What portion of applicants met pre-screening criteria?
| Met criteria | 100.0% |
References
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- Donson, Andrew M, John Apps, Andrea M Griesinger, Vladimir Amani, Davis A Witt, Richard C E Anderson, Toba N Niazi, et al.. 2017. “Molecular Analyses Reveal Inflammatory Mediators in the Solid Component and Cyst Fluid of Human Adamantinomatous Craniopharyngioma”. Journal of Neuropathology & Experimental Neurology. Oxford University Press (OUP). doi:10.1093/jnen/nlx061.
- Choy, E. H. S., D. A. Isenberg, T. Garrood, S. Farrow, Y. Ioannou, H. Bird, N. Cheung, et al.. 2002. “Therapeutic Benefit of Blocking Interleukin-6 Activity with an Anti-interleukin-6 Receptor Monoclonal Antibody in Rheumatoid Arthritis: A Randomized, Double-blind, Placebo-controlled, Dose-escalation Trial”. Arthritis & Rheumatism. Wiley. doi:10.1002/art.10623.
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- Yokota S, Miyamae T, Imagawa T, Iwata N, Katakura S, Mori M, Woo P, Nishimoto N, Yoshizaki K, Kishimoto T. Therapeutic efficacy of humanized recombinant anti-interleukin-6 receptor antibody in children with systemic-onset juvenile idiopathic arthritis. Arthritis Rheum. 2005 Mar;52(3):818-25. doi: 10.1002/art.20944.
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- Gust J, Hay KA, Hanafi LA, Li D, Myerson D, Gonzalez-Cuyar LF, Yeung C, Liles WC, Wurfel M, Lopez JA, Chen J, Chung D, Harju-Baker S, Ozpolat T, Fink KR, Riddell SR, Maloney DG, Turtle CJ. Endothelial Activation and Blood-Brain Barrier Disruption in Neurotoxicity after Adoptive Immunotherapy with CD19 CAR-T Cells. Cancer Discov. 2017 Dec;7(12):1404-1419. doi: 10.1158/2159-8290.CD-17-0698. Epub 2017 Oct 12.
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- 2022. "ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05233397.
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