A Study of BXQ-350 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma (DIPG) or Diffuse Midline Glioma (DMG)

Stopped early · Phase 1

Conditions studied: Diffuse Intrinsic Pontine Glioma, Diffuse Midline Glioma, H3 K27M-Mutant

In brief

This study will evaluate the safety of BXQ-350 and determine the maximum tolerated dose (MTD) in children with newly diagnosed DIPG or DMG. All patients will receive BXQ-350 by intravenous (IV) infusion and radiation therapy. The study is divided into two parts: Part 1 will enroll patients at increasing dose levels of BXQ-350 in order to determine the MTD. Part 2 will enroll patients requiring a biopsy in order to assess BXQ-350 concentrations in the biopsied tumor.

Key facts

Study ID

NCT04771897

Run by

Bexion Pharmaceuticals, Inc.

People needed

10

Starts

2021-05-24

Expected to finish

2024-10-04

Last updated by the study team

2024-12-20

Who can join

Age: 1 and older, up to 30. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Each subject must meet the following criteria:
• Provide signed, written informed consent prior to the initiation of any study-specific procedures (consent from guardians for minor children and patient assent according to institution and Institutional Review Board (IRB) standards)
• Age ≥ 1 year of age and ≤ 30 years of age at the time of study entry
• Have radiographically suspected or histologically confirmed newly diagnosed DIPG or DMG with the following defining disease characteristics/features:
• Part 1 and Part 2:
• DIPG: radiographic imaging showing a diffuse expansion of the brainstem/pons by a tumor that is poorly defined but abnormally bright in signal on T2-weighted images and abnormally dark on T1-weighted images; contrast enhancement, when present, is typically mild; there may be some indication of necrosis, and the basilar artery is commonly engulfed by tumor.
• DMG: the same imaging characteristics as noted above for DIPG are present, but the lesion can extend superiorly up into the midbrain and thalami, and/or inferiorly to the medulla.
• In cases of disease that is not classic for DIPG or DMG, biopsy may be necessary in order to obtain histological confirmation of eligibility.
• Part 2: newly-diagnosed DIPG or DMG planning to undergo biopsy at the recommendation of the treating physician; to be considered evaluable for PK tissue concentration analysis, tumor samples must have at least 50% viable tumor cells with <30% necrosis or hemorrhage as determined by the study neuropathologist
• If receiving glucocorticoid therapy: dexamethasone allowed with maximum allowable dose 16mg/day
• Have measurable or non-measurable disease per RANO criteria
• Have Lansky (age 1 - 15) / Karnofsky (age ≥ 16) Performance Score of ≥ 50% or Eastern Cooperative Oncology Group (ECOG) Performance Status (age ≥ 18) of 0 - 2
• Subjects unable to walk because of paralysis, but who can sit without assistance/restraint and control a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
• Have acceptable liver function defined as:
• Total serum bilirubin ≤ 1.5 x upper limit of normal for the study site (ULN) (in subjects with known Gilbert Syndrome, total bilirubin ≤ 3 x ULN, with direct bilirubin ≤ 1.5 x ULN)
• Aspartate Transaminase (AST), Serum Glutamic Oxaloacetic Transaminase (SGOT), Alanine Transaminase (ALT), Serum Glutamic-Pyruvic Transamine (SGPT) ≤ 3 x ULN (if liver metastases are present, then ≤ 5 x ULN is allowed)
• Serum albumin ≥ 3 grams/deciliter (g/dL)
• Have acceptable renal function defined as:
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥70 milliliters (mL)/min/1.73 meters squared (m2) or a serum creatinine based on age/gender as follows: 1 to < 2 years: 0.6 (male); 0.6 (female) 2 to < 6 years: 0.8 (male); 0.8 (female) 6 to < 10 years: 1 (male); 1 (female) 10 to < 13 years: 1.2 (male); 1.2 (female) 13 to < 16 years: 1.5 (male); 1.4 (female)
• 16 years: 1.7 (male);1.4 (female)
• Threshold creatinine values derived from the Schwartz formula for estimating GFR utilizing child length and stature data published by the Center for Disease Control (Schwartz 2009)
• Have acceptable bone marrow function defined as:
• Absolute neutrophil count (ANC) ≥ 1,500 cells/ cubic millimeter (mm3)
• Platelet count ≥ 100,000 cells/mm3 (unsupported, no transfusion within 7 days of enrollment)
• Hemoglobin > 9.0 g/dL (unsupported, no transfusion within 7 days of enrollment)

You may not qualify if…

• Subjects must not meet any of the following criteria:
• Have a concurrent or secondary malignancy
• Have a low-grade glioma (Grade II or less)
• Have received prior treatment with radiation, chemotherapy, anti-neoplastic, or investigational agents
• Have had major surgery other than a minor outpatient procedure within 28 days prior to dose assignment or have not recovered from major side effects of the surgery if more than 4 weeks have elapsed since surgery
• Have poorly controlled hypertension despite the use of antihypertensive agents defined as blood pressure ≥95th percentile for age and weight or >160/90 on at least 2 repeated determinations on separate days within 2 weeks (14 days) prior to initiation of screening
• Have a history of cardiac dysfunction including:
• myocardial infarction within 6 months prior to initiation of screening
• history of documented congestive heart failure (New York Heart Association functional classification III-IV) within 6 months prior to initiation of screening
• active cardiomyopathy
• electrocardiogram (ECG) with QTc >470 msec at screening
• echocardiogram with ejection fraction <50% or a decrease in the left ventricular shortening fraction to <27%
• Have a known history of Human Immunodeficiency Virus (HIV) seropositivity
• Have active (acute or chronic) or uncontrolled severe infections
• Have active poor wound healing (delayed healing, wound infection or fistula)
• Have evidence of active clinically significant bleed (e.g., gastrointestinal bleed, hemoptysis, or gross hematuria) at screening
• Are pregnant or nursing, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive serum human chorionic gonadotropin (hCG) laboratory test
• Have a known sensitivity to any component of BXQ-350
• Have other concurrent severe and/or uncontrolled medical condition that would, in the Principal Investigator's judgment contraindicate the subject's participation in the clinical study or obscure assessment of toxicity

Where it is running

• Children's Hospital Colorado — Aurora, Colorado, United States
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
• Nationwide Children's — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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