ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma
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|ClinicalTrials.gov Identifier: NCT05233397|
Recruitment Status : Recruiting
First Posted : February 10, 2022
Last Update Posted : January 10, 2023
|Condition or disease||Intervention/treatment||Phase|
|Adamantinomatous Craniopharyngioma Recurrent Adamantinomatous Craniopharyngioma||Drug: Tocilizumab||Phase 2|
Adamantinomatous Craniopharyngioma (ACP) is a highly debilitating pediatric brain tumor that lacks medical anti-tumor therapies. Current therapy, which depends largely on surgery and radiation, is associated with poor quality of life and becomes more challenging and risky in the setting of recurrent disease. Recent discoveries regarding the biological characteristics of ACP indicate that available agents, including IL-6 pathway blockers may have efficacy in the control of ACP. We hypothesize that the IL6- receptor antagonist ACTEMRA (tocilizumab) will be safe and effective at inducing tumor response in children with residual ACP.
In this study, up to 38 patients will receive tocilizumab at the dose approved for pediatric Systemic Juvenile Idiopathic Arthritis (< 30 kg: 12 mg/kg IV every 2 weeks; ≥30 kg: 8 mg/kg IV every 2 weeks). Therapy may continue for up to two years (26 cycles).
It will be a multi-center Phase 2 trial with two strata for patients aged >1 year and <25 years with unresectable ACP who may have been previously treated with radiation (Stratum 1, 18 patients) or without radiation (Stratum 2, 18 patients).
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||38 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||ACTEMRA® (tocilizumab)|
|Masking:||None (Open Label)|
|Official Title:||Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (Tocilizumab) for the Treatment of Progressive/Recurrent Pediatric Adamantinomatous Craniopharyngioma|
|Actual Study Start Date :||December 16, 2022|
|Estimated Primary Completion Date :||December 2024|
|Estimated Study Completion Date :||December 2026|
Experimental: Stratum 1 and Stratum 2
Stratum 1: Patients with progressive or recurrent adamantinomatous craniopharyngiomas following radiation therapy.
Stratum 2: Patients with measurable adamantinomatous craniopharyngioma who have undergone surgery but have not previously received radiation therapy. Progressive disease is allowed but not required
For < 30 kg: 12 mg/kg IV every 2 weeks; For ≥30 kg: 8 mg/kg IV every 2 weeks
Other Name: ACTEMRA®
- Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with systemic tocilizumab [ Time Frame: 24 months ]To calculate the number of patients who experience sustained objective response rate [minor response (MR) + partial response (PR) + complete response (CR)] of patients with recurrent/progressive previously irradiated Adamantinomatous Craniopharyngioma to treatment with systemic tocilizumab (Stratum 1).
- 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 [ Time Frame: 24 months ]To calculate the number of patients who experience sustained objective response rate (MR + PR + CR) of patients with measurable Adamantinomatous Craniopharyngioma who have undergone surgery but have not been previously treated with radiation to treatment with systemic tocilizumab (Stratum 2).
- Biological effects of tocilizumab on ACP tumor tissue and cyst fluid. [ Time Frame: 24 months ]To measure the concentrations of IL-6, IL-8, IL-10, CXCL1, CXCR2, IDO-1 and IL-6R using a combination of ELISA, RNAseq, immunohistochemistry and immunofluorescence in cyst fluid or tumor tissue or blood. Comparisons will be made with known levels in the literature and among patient samples from within the study.
- Toxicities associated with tocilizumab in children with ACP [ Time Frame: 24 months ]To calculate the number of participants with, as well as frequency and severity of, tocilizumab-related Adverse Events as assessed by CTCAE v5.0 in children with recurrent or refractory ACP.
- PFS of ACP patients treated with tocilizumab after radiation [ Time Frame: 12 months ]To assess one-year progression-free survival (PFS) rates for patients with ACP who are treated with tocilizumab following progression after radiation (Stratum 1).
- PFS of ACP patients treated with tocilizumab who have not received radiation [ Time Frame: 12 months ]To assess one-year progression-free survival (PFS) rates for patients with ACP who are treated with tocilizumab who have not previously received radiation (Stratum 2).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05233397
|Contact: Leonie Mikael, PhDfirstname.lastname@example.org|
|Contact: Dorothy Crabtree, BS||16147228693||Dorothy.Crabtree@nationwidechildrens.org|
|Study Chair:||Kathleen H Dorris, MD||Children's Hospital Colorado|
|Study Chair:||Todd C Hankinson, MD||Children's Hospital Colorado|
|Principal Investigator:||Maryam Fouladi, MD||Nationwide Children's Hospital|