ACTEMRA® for the Treatment of Pediatric Adamantinomatous Craniopharyngioma

Phase 2

Recruiting

• Conditions: Adamantinomatous Craniopharyngioma; Recurrent Adamantinomatous Craniopharyngioma

• Registration Number: NCT05233397
• Lead Sponsor: Nationwide Children's Hospital

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-1-25
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 38

Inclusion Criteria

Inclusion Criteria:<br><br> 1. Age: Patients must be = 12 months and = 25 years of age at the time of study<br> enrollment.<br><br> 2. Diagnosis: Patients with histologically-confirmed adamantinomatous craniopharyngioma<br> (ACP) Histologic confirmation of ACP may be made on solid tumor or, if no solid<br> tumor can be safely obtained, cyst fluid with classic ACP characteristics of thick,<br> cholesterol-rich, greenish-brown liquid in the context of imaging features<br> consistent with craniopharyngioma, including lobulated, cystic/solid mass with<br> calcifications that originates in the sellar/suprasellar region.<br><br> 3. Disease Status: Patients must have measurable disease.<br><br> - Stratum 1: Patients with progressive or recurrent ACP who demonstrate cystic<br> and/or solid recurrence or progression at least 6 months post completion of<br> radiation therapy<br><br> - Stratum 2: Patients with measurable ACP who have undergone surgery but have NOT<br> previously undergone irradiation (but may have received prior systemic or<br> intracystic therapy). Progressive disease is allowed but not required.<br><br> 4. Performance Level: Karnofsky = 50% for patients > 16 years of age and Lansky = 50<br> for patients = 16 years of age (See Appendix I). Note: Neurologic deficits in<br> patients with CNS tumors must have been stable for at least 7 days prior to study<br> enrollment. Patients who are unable to walk because of paralysis, but who are up in<br> a wheelchair, will be considered ambulatory for the purpose of assessing the<br> performance score.<br><br> 5. Prior Therapy: Patients must have recovered or stabilized from the acute toxic<br> effects of prior treatments<br><br> - Biologic (anti-neoplastic agent): At least 7 days must have elapsed after the<br> last (systemic or intracystic) dose of a biologic agent. For agents that have<br> known adverse events occurring beyond 7 days after administration, this period<br> must be extended beyond the time during which adverse events are known to<br> occur. The duration of this interval must be discussed with the study chair<br><br> - Immunotherapy: At least 42 days after the completion of any type of systemic<br> immunotherapy, e.g. tumor vaccines.<br><br> - Monoclonal antibodies: At least 21 days after the last dose of a monoclonal<br> antibody.<br><br> - Radiation therapy: Patients must have had their last (conventional or<br> hypofractionated) fraction of: a) Focal irradiation > 6 months prior to<br> enrollment and b) No prior craniospinal irradiation is permitted.<br><br> - Corticosteroids: Patients receiving dexamethasone must be on a stable or<br> decreasing dose for at least 1 week prior to enrollment<br><br> - Myelosuppressive systemic therapy: At least 21 days must have elapsed after the<br> last systemic myelosuppressive therapy.<br><br> - Surgery: At least 6 weeks must have elapsed since surgery.<br><br> 6. Organ Function Requirements<br><br> Adequate Bone Marrow Function Defined as:<br><br> - Peripheral absolute neutrophil count (ANC) =1000/mm3<br><br> - Platelet count =100,000/mm3 (transfusion independent, defined as not receiving<br> platelet transfusions for at least 7 days prior to enrollment)<br><br> - Hemoglobin >8 g/dL (may be transfused)<br><br> Adequate Renal Function Defined as:<br><br> - Creatinine clearance or radioisotope GFR > 70ml/min/1.73 m2 or<br><br> - A serum creatinine based on (Schwartz et al. J. Peds, 106:522, 1985) age/gender<br> as follows:<br><br> 1 to < 2 years: maximum serum creatinine 0.6 mg/dL for males and females. 2 to<br> < 6 years: maximum serum creatinine 0.8 mg/dL for males and females. 6 to < 10<br> years: maximum serum creatinine 1.0 mg/dL for males and females. 10 to < 13<br> years: maximum serum creatinine 1.2 mg/dL for males and females. 13 to < 16<br> years: maximum serum creatinine 1.5 mg/dL for males and 1.4 mg/dL for females.<br><br> = 16 years: maximum serum creatinine 1.7 mg/dL for males and 1.4 mg/dL for<br> females.<br><br> Adequate Liver Function Defined as:<br><br> - Total bilirubin within normal institutional limits<br><br> - AST (SGOT) = 2.5 × institutional upper limit of normal<br><br> - ALT (SGPT) = 2.5 × institutional upper limit of normal<br><br> Adequate Neurologic Function Defined as:<br><br> - Patients with neurological deficits should have deficits that are stable for a<br> minimum of 1 week prior to enrollment.<br><br> - Patients with current seizure disorders may be enrolled if seizures are<br> well-controlled on antiepileptic therapies.<br><br> 7. Informed Consent: All patients and/or their parents or legally authorized<br> representatives must sign a written informed consent. Assent, when appropriate, will<br> be obtained according to institutional guidelines.<br><br>Exclusion Criteria:<br><br> 1. Pregnancy or Breast-Feeding: Pregnant or breast-feeding women will not be entered on<br> this study due to unknown risks of fetal and teratogenic adverse events as seen in<br> animal/human studies. Pregnancy tests must be obtained in girls who are<br> post-menarchal. Males or females of reproductive potential may not participate<br> unless they have agreed to use an effective contraceptive method for at least 90<br> days after discontinuation of drug for females and at least 60 days for males. For<br> females of childbearing potential, agreement to remain abstinent (refrain from<br> heterosexual intercourse) or use contraceptive methods (bilateral tubal ligation,<br> male sterilization, hormonal contraceptives that inhibit ovulation,<br> hormone-releasing intrauterine devices, and copper intrauterine devices; hormonal<br> contraceptive methods must be supplemented by a barrier method) and agreement to<br> refrain from donating eggs are required. For males of reproductive potential,<br> agreement to remain abstinent (refrain from heterosexual intercourse) or use a<br> condom, and agreement to refrain from donating sperm.<br><br> 2. Gastrointestinal Disease: Patients with a history of serious gastrointestinal<br> disease, including inflammatory bowel disease or gastrointestinal perforation<br><br> 3. Concomitant Medications<br><br> - Corticosteroids: Patients receiving corticosteroids who have not been on a<br> stable or decreasing dose of corticosteroid for at least 7 days prior to<br> enrollment are not eligible.<br><br> - Investigational Drugs: Patients who are currently receiving another<br> investigational drug are not eligible.<br><br> - Anti-cancer Agents: Patients who are currently receiving other anti-cancer<br> agents are not eligible.<br><br> 4. Study Specific:<br><br> - Patients who have an uncontrolled infection are not eligible.<br><br> - Patients who have received any live or attenuated vaccinations within three<br> months prior to start of therapy are not eligible.<br><br> - Any significant concurrent medical or surgical condition that would jeopardize<br> the patient's safety or ability to complete the study, including, but not<br> limited to, disease of the nervous, renal, hepatic, cardiac (such as<br> symptomatic congestive heart failure, unstable angina pectoris, cardiac<br> arrhythmia), pulmonary, or endocrine system<br><br> - Patients who have a history o

Exclusion Criteria

Not provided

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Sustained objective response rate of patients with recurrent/progressive previously irradiated ACP to treatment with systemic tocilizumab;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

Secondary Outcome Measures

Name	Time	Method
Biological effects of tocilizumab on ACP tumor tissue and cyst fluid.;Toxicities associated with tocilizumab in children with ACP;PFS of ACP patients treated with tocilizumab after radiation;PFS of ACP patients treated with tocilizumab who have not received radiation