Actively Recruiting
Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer
Led by Theodore S. Johnson · Updated on 2026-01-09
37
Participants Needed
1
Research Sites
346 weeks
Total Duration
On this page
Sponsors
T
Theodore S. Johnson
Lead Sponsor
A
Augusta University
Collaborating Sponsor
AI-Summary
What this Trial Is About
Recent lab-based discoveries suggest that IDO (indoleamine 2,3-dioxygenase) and BTK (Bruton's tyrosine Kinase) form a closely linked metabolic checkpoint in tumor-associated antigen-presenting cells. The central clinical hypothesis for the GCC2020 study is that combining ibrutinib (BTK-inhibitor) with indoximod (IDO-inhibitor) during chemotherapy will synergistically enhance anti-tumor immune responses, leading to improvement in clinical response with manageable overlapping toxicity. The GCC2020 trial is a prospective open-label phase 1 trial to determine the best safe dose of the BTK-inhibitor ibrutinib to use in combination with previously studied chemo-immunotherapy regimens comprised of the investigational IDO-inhibitor indoximod plus oral palliative chemotherapy for participants, age 6 to 25 years, with relapsed or refractory primary brain cancer. Those previously treated with indoximod-based therapy may be eligible, including prior treatment via the phase 2 indoximod study (GCC1949, NCT04049669), the now closed phase 1 study (NLG2105, NCT02502708), or any expanded access (compassionate use) protocols. Ibrutinib will be combined with either indoximod plus oral cyclophosphamide and etoposide (Regimen A) or indoximod plus oral temozolomide (Regimen B). No cross-over between these two regimens will be allowed. Dose-escalation cohorts will determine the best safe dose of ibrutinib for each of these regimens. This will be followed by expansion cohorts, using ibrutinib at the best safe dose for each regimen, to allow assessment of preliminary evidence of efficacy.
CONDITIONS
Official Title
Chemo-immunotherapy Using Ibrutinib Plus Indoximod for Patients With Pediatric Brain Cancer
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 6 to 25 years with relapsed or refractory primary brain cancer including ependymoma, medulloblastoma, glioblastoma, or other central nervous system tumors
- Must have MRI confirmation of current active disease
- Must be able to swallow pills
- Lansky or Karnofsky performance status score of 50% or higher
- Adequate kidney function with creatinine clearance over 25 mL/min and creatinine within 1.5 times the age-adjusted normal
- Adequate liver function with ALT and AST levels not exceeding 3 times the upper limit of normal and total bilirubin no more than 1.5 times normal unless due to non-hepatic causes
- Adequate bone marrow function with ANC of at least 1000/mm3, platelets at least 100,000/mm3, and hemoglobin at least 8 g/dL
- Well-controlled seizure disorders on antiepileptic medication
- At least 21 days since any investigational or cytotoxic therapy (other than indoximod) and 28 days since antibody, vaccine, or cellular immune therapy
- At least 56 days since tumor-directed infectious agent therapies
- At least 90 days since radiation or proton therapy targeting all known disease sites
- No concurrent anti-cancer therapies other than those in this study
- Women of childbearing potential and sexually active men must use effective birth control during and after the study; men must not donate sperm during and for 3 months after the study
- Patients or guardians must provide informed consent
You will not qualify if you...
- Unable to swallow pills
- Known allergy to any drugs in the treatment plan
- Active autoimmune disease requiring systemic therapy (non-autoimmune allergic conditions allowed)
- Pregnant or breastfeeding women
- Major surgery or unhealed wound within 4 weeks prior to screening
- Known central nervous system lymphoma
- Active bleeding or recent stroke or intracranial hemorrhage within 6 months, except uncomplicated recent surgery blood products
- Need for anticoagulation with warfarin or equivalent drugs
- Chronic treatment with strong CYP3A inhibitors
- Significant cardiovascular disease including uncontrolled arrhythmias, heart failure, recent heart attack, or severe cardiac disease
- Prolonged QTc interval over 470 msec or congenital long QT syndrome
- Vaccination with live vaccines within 4 weeks prior to screening
- Known HIV infection or active Hepatitis B or C infection or uncontrolled systemic infection
- Any life-threatening illness or condition that may risk safety or interfere with the study treatments as judged by the investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Augusta University, Georgia Cancer Center
Augusta, Georgia, United States, 30912
Actively Recruiting
Research Team
T
Theodore S. Johnson, MD, PhD
CONTACT
R
Robin Dobbins, RN
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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