A Phase 1/2 interventional study of Biospecimen Collection and Bone Marrow Aspirate in Diffuse Midline Glioma, H3 K27-Altered, Metastatic Malignant Neoplasm in the Central Nervous System and Recurrent Diffuse Intrinsic Pontine Glioma, sponsored by Children's Oncology Group. Recruiting at 35 sites in 2 countries. Open to participants aged 12 Months to 21 Years. Per ClinicalTrials.gov, last updated 2026-09-11.
Sponsored by Children's Oncology Group · Phase 1/2, Interventional, and Treatment
This phase I/II trial evaluates the best dose, side effects and possible benefit of CBL0137 in treating patients with solid tumors, including central nervous system (CNS) tumors or lymphoma that has come back (relapsed) or does not respond to treatment (refractory). Drugs, such as CBL0137, block signals passed from one molecule to another inside a cell. Blocking these signals can affect many functions of the cell, including cell division and cell death, and may kill cancer cells.
PRIMARY OBJECTIVES:
I. To estimate the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of Facilitates Chromatin Transcription (FACT) complex-targeting curaxin CBL0137 (CBL0137) administered via infusion on Day 1 and Day 8 of a 21-day cycle to children with recurrent or refractory solid tumors, including CNS tumors and lymphoma. (Phase 1 Dose Escalation) II. To preliminarily determine the antitumor effects as measured by objective response rate of CBL0137 in children with progressive/recurrent diffuse intrinsic pontine glioma (DIPG) and other H3 K27-altered diffuse midline gliomas (DMG). (Phase 2)
SECONDARY OBJECTIVES:
I. To preliminarily determine the antitumor effects of CBL0137 in children with refractory solid tumors and other CNS tumors, to the extent possible in the context of a Phase 1 study.
II. To define and describe the toxicities of CBL0137 in children with recurrent or refractory solid tumors, including CNS tumors.
III. To characterize the pharmacokinetics of CBL0137 in children with recurrent or refractory solid tumors, including CNS tumors.
EXPLORATORY OBJECTIVES:
I. To measure biologic marker FACT in tumor specimens with potential for correlation with disease response.
II. To evaluate the effect of CBL0137 on immune response by measuring the effects on the interferon response pathway in peripheral blood mononuclear cells.
III. To preliminarily determine the effect of treatment with CBL0137 on overall survival of children with DIPG or other diffuse midline gliomas, H3 K27-altered, in comparison with historical controls.
OUTLINE: This is a phase I, dose-escalation study followed by a phase II study.
Patients receive CBL0137 intravenously (IV) over 30 minutes on days 1 and 8. Treatment repeats every 21 days for up to 17 cycles in the absence of disease progression or unacceptable toxicity. Patients with pseudoprogression may remain on treatment, as per the treating physician. Patients also undergo echocardiography (ECHO) collection of blood samples throughout the trial. Patients may also undergo bone marrow aspirate and/or biopsy as clinically indicated.
After completion of study treatment, patients are followed up at 3, 6, 9, 12, 18, 24, 36, 48 and 60 months.
Patients must have had histologic verification of malignancy at original diagnosis or relapse, except in patients with diffuse intrinsic brain stem tumors, or patients with pineal tumors and elevations of cerebrospinal fluid (CSF) or serum tumor markers, including alpha-fetoprotein or beta-human chorionic gonadotropin (HCG)
Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. If after the required timeframe, the numerical eligibility criteria are met, e.g., blood count criteria, the patient is considered to have recovered adequately
Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive
Stem cell Infusions (with or without total body irradiation [TBI]):
For patients with solid tumors without known bone marrow involvement:
For patients with solid tumors without known bone marrow involvement:
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m\^2 or a creatinine based on age/sex as follows (performed within 7 days prior to enrollment unless otherwise indicated):
Patients with solid tumors:
Patients with solid tumors:
Exclusion Criteria:
Patients receive CBL0137 IV over 30 minutes on days 1 and 8. Treatment repeats every 21 days for up to 17 cycles in the absence of disease progression or unacceptable toxicity. Patients with pseudoprogression may remain on treatment, as per the treating physician. Patients also undergo ECHO collection of blood samples throughout the trial. Patients may also undergo bone marrow aspirate and/or biopsy as clinically indicated.
Procedure: Biospecimen Collection · Procedure: Bone Marrow Aspirate · Procedure: Bone Marrow Biopsy · Procedure: Echocardiography Test · Drug: FACT Complex-targeting Curaxin CBL0137
Undergo collection of blood samples
Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo bone marrow aspirate
Also known as: BONE MARROW, LIQUID, Human Bone Marrow Aspirate
Undergo bone marrow biopsy
Also known as: Biopsy of Bone Marrow, Biopsy, Bone Marrow
Undergo ECHO
Also known as: EC, Echocardiography
Given IV
Also known as: CBL0137
Maximum tolerated dose and/or Recommended Phase 2 dose of CBL0137
Maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of CBL0137 in children with relapsed or refractory solid tumors including central nervous system (CNS) tumors and lymphoma.
Time frame: Up to 21 days
Frequency of dose limiting toxicities of CBL0137 (Phase I)
The frequency (%) of patients experiencing a cycle 1 dose limiting toxicity attributable to CBL0137 by study part and dose level.
Time frame: Up to 21 days
Anti-tumor effect of CBL0137 in children with diffuse intrinsic pontine glioma (DIPG) or other H3 K27-altered diffuse midline gliomas (Phase II)
Frequency (%) of patients with at least partial response to CBL0137 at the maximum tolerated dose/recommended phase II dose (MTD/RP2D) in children with progressive or recurrent diffuse intrinsic pontine glioma (DIPG).
Time frame: Up to 4 months
Anti-tumor effect of CBL0137 in children with solid tumors (Phase I)
Frequency (%) of patients with at least partial response to CBL0137 at the MTD/RP2D in children with refractory solid tumors and other central nervous system (CNS) tumors.
Time frame: Up to 4 months
Frequency of adverse events attributable to CBL0137
The frequency (%) of patients experiencing adverse events that are at least possibly attributable to CBL0137 by study part and dose level.
Time frame: Up to 60 months
Area under the drug concentration curve of CBL0137
The median (min, max) of the area under the drug concentration curve for CBL0137 by study part and dose level.
Time frame: Up to 3 days
FACT in tumor specimens
A descriptive analysis of biomarkers will assess associations with disease response. The parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit). All these analyses will be descriptive and exploratory and hypotheses generating in nature. FACT expression in tumor specimens will be determined by immunohistochemistry.
Time frame: Up to 60 months
Immune response
Will measure effects on the interferon response pathway. A descriptive analysis of biomarkers will assess associations with disease response. The parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit). All these analyses will be descriptive and exploratory and hypotheses generating in nature. Peripheral blood lymphocytes will be assessed for expression of genes in the interferon response pathway, in order to determine whether treatment with CBL0137 leads to increase gene expression.
Time frame: Up to 60 months
Overall survival
Will be compared with historical controls in children with DIPG or other diffuse midline gliomas, H3 K27-altered.
Time frame: Up to 60 months
Progression-free survival
An exploratory analysis of progression-free survival using Kaplan-Meier curves and the log-rank test statistic will compare overall time-to-disease progression (or death) versus historical cohort for the DIPG cohort.
Time frame: Up to 60 months
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Diffuse Intrinsic Pontine Glioma→
Children's Oncology Group