Tocilizumab in Children With ACP

Study Purpose

This study will be conducted in two phases. The first phase (phase 0) will be looking at patients with new or recurrent/ progressed craniopharyngioma tumors. These patients will be given one dose of tocilizumab before they have SOC surgery of their tumor. The objective of this phase is to see if drug reaches the tumor. If phase 0 is favorable and shows that drug is penetrating the tumor, the second phase of the study (feasibility phase) will open. Both phases will remain open concurrently and patients will be able to enroll on the Phase 0 then "roll over" and enroll on the feasibility phase. During the feasibility phase patients will be administered tocilizumab every two weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years in the feasibility phase.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 2 Years - 21 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria. Phase 0 Eligibility: 1. Tumor biopsy/resection and/or cyst aspiration planned for the clinical care of the patient independent of study participation by the treating pediatric neurosurgeon and neuro-oncologist. 2. Must meet one of the following criteria: 1. Presumed craniopharyngioma based on imaging features and best judgement of treating medical team (if newly diagnosed) 2. Previous histologically confirmed ACP that has progressed or recurred at the time of enrollment. Feasibility Eligibility: 1. Must meet one of the following criteria: 1. Recurrent or progressive* ACP treated with surgery alone without radiation. 2. Recurrent or progressive* ACP treated with surgery and radiation. * Progressive disease for eligibility purposes will be defined as follows: Solid disease: any growth deemed progression based on discretion of the investigator regardless of timing from RT Cystic disease: must be at least 6 months from last day of RT. Patients demonstrating isolated cyst growth >6 months after RT must show a continued increase in the cystic component on two serial MRI scans performed at least 4 weeks apart OR at least partial reaccumulation of the cyst following one or more cyst aspirations. 3. Newly diagnosed, by histology or imaging ACP with unresectable residual cystic and/or solid disease that is measurable in 2 dimensions. 2. Subjects who participated in the Phase 0 portion and meet eligibility, may enroll in the Feasibility Phase of the study once open. Overall Study

Inclusion Criteria:

1. Age: ≥ 2 years and < 21 years. 2. Subjects may have received prior tocilizumab or other IL6 or IL6R inhibitor. 3. Organ Function Requirements. 1. Adequate bone marrow function defined as:
  • - Platelet count ≥100,000/μl (transfusion independent) - Absolute neutrophil count (ANC) ≥1500/μl.
2. Adequate renal function defined as:
  • - Creatinine clearance or radioisotope GFR ≥70 ml/min/1.73 m2 or.
  • - A serum creatinine based on age/gender as follows: (Age, Male, Female) 3 to < 6 years, 0.8, 0.8; 6 to < 10 years, 1, 1; 10 to < 13 years, 1.2, 1.2; 13 to < 16 years, 1.5, 1.4; 16 years to < 18 years, 1.7, 1.4.
3. Adequate liver function defined as:
  • - SGOT (AST) and SGPT (ALT) <1.5x ULN for age.
4. Subjects must meet one of the following performance scores: 1. ECOG performance status scores of 0, 1, or 2; 2. Karnofsky score of ≥60 for patients > 16 years of age; or. 3. Lansky score of ≥60 for patients ≤16 years of age. 5. Subjects of childbearing or child fathering potential must be willing to use a medically acceptable form of birth control, which includes abstinence, while being treated on this study. 6. Informed consent and assent obtained as appropriate. Exclusion Criteria. 1. Pregnant or breastfeeding. 2. Uncontrolled intercurrent illness including, but not limited to: 1. ongoing or active infection (including active tuberculosis) 2. symptomatic congestive heart failure. 3. unstable angina pectoris. 4. cardiac arrhythmia. 5. psychiatric illness/social situations that would limit compliance with study requirements are not eligible. 3. Known hypersensitivity or history of anaphylaxis to tocilizumab. 4. Received any live vaccinations within 3 months prior to start of therapy. 5. Evidence of metastatic disease or other cancer. 6. Inability to return for follow up visits or obtain required follow-up studies to assess toxicity of therapy

Trial Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT03970226
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Early Phase 1
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

University of Colorado, Denver
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Kathleen Dorris, MD
Principal Investigator Affiliation University of Colorado, Denver
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Other, NIH
Overall Status Recruiting
Countries United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Adamantinomatous Craniopharyngioma
Arms & Interventions

Arms

Experimental: Tocilizumab Administration: Phase 0

In Phase 0, patients will receive one dose of tocilizumab prior to surgery.

Experimental: Tocilizumab Administration: Feasibility Phase

During the Feasibility Phase, patients will receive tocilizumab every 2 weeks for up to 13 cycles (approximately 1 year). Patients will be followed for up to 5 years.

Interventions

Drug: - Tocilizumab

Phase 0: One dose of tocilizumab prior to surgery Feasibility phase: Tocilizumab administered every 2 weeks for up to 13 cycles (approximately 1 year).

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Children's Hospital Colorado, Aurora, Colorado

Status

Recruiting

Address

Children's Hospital Colorado

Aurora, Colorado, 80045

Site Contact

Kathleen Dorris, MD

kathleen.dorris@childrenscolorado.org

720-777-5305

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