Phase 2 Study of Systemic IL-6 Receptor Antagonist ACTEMRA® (Tocilizumab) for the Treatment of Progressive/Recurrent Pediatric Adamantinomatous Craniopharyngioma

Official Summary

ACTEMRA (tocilizumab) is an IL-6 receptor antagonist used for the treatment of adult Rheumatoid Arthritis as well as Polyarticular (PJIA) and Systemic (SJIA) Juvenile Idiopathic Arthritis. In this Phase II, the drug will be used to treat pediatric patients diagnosed with recurrent Adamantinomatous Craniopharyngioma including patients who have undergone surgery and/or radiation therapy.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 38 participants

Interventions

• DRUG: Tocilizumab — For \< 30 kg: 12 mg/kg IV every 2 weeks; For ≥30 kg: 8 mg/kg IV every 2 weeks

Primary Outcomes

• Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with systemic tocilizumab (From Day 1 of treatment through 30 days following end of protocol treatment)
• 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 (From Day 1 of treatment through 30 days following end of protocol treatment)

Secondary Outcomes

• Biological effects of tocilizumab on ACP tumor tissue and cyst fluid. (From Day 1 of treatment through 30 days following end of protocol treatment)
• Toxicities associated with tocilizumab in children with ACP (From Day 1 of treatment through 30 days following end of protocol treatment)
• PFS of ACP patients treated with tocilizumab after radiation (12 months)
• PFS of ACP patients treated with tocilizumab who have not received radiation (12 months)

Trial Locations

• Children's Hospital Colorado, Aurora, Colorado, United States
• Children's National Medical Center, Washington D.C., District of Columbia, United States
• Nicklaus Children's Hospital, Miami, Florida, United States
• Duke Children's Hospital, Durham, North Carolina, United States
• Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
• Nationwide Children's Hospital, Columbus, Ohio, United States
• Texas Children's Hospital, Houston, Texas, United States
• Seattle Children's Hospital, Seattle, Washington, United States
• Sydney Children's Hospital, Randwick, New South Wales, Australia
• Queensland Children's Hospital, South Brisbane, Queensland, Australia
• ...and 4 more locations

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