A Study of BXQ-350 in Children and Young Adults With Relapsed Solid Tumors

Stopped early · Phase 1

Conditions studied: Neoplasms

In brief

This study will evaluate the safety of BXQ-350 and determine the maximum tolerated dose (MTD) in children and young adults with relapsed solid tumors, including recurrent malignant brain tumors. All patients will receive BXQ-350 by intravenous (IV) infusion. 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 use the MTD to further assess the safety of BXQ-350 as well as preliminary anti-tumor activity.

Key facts

Study ID

NCT03967093

Run by

Bexion Pharmaceuticals, Inc.

People needed

9

Starts

2019-04-15

Expected to finish

2020-01-17

Last updated by the study team

2021-07-22

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)
• Are male or female aged ≥ 1 to 30 years
• Have histologically or cytologically confirmed relapsed solid tumor cancer, including recurrent malignant brain tumors, for which there is no further standard therapy or when standard therapy is contraindicated • Recurrent malignant brain tumors: must have shown unequivocal evidence for recurrence or progression by MRI scan or must have histologically proven tumor recurrence • Recurrent malignant brain tumors: must have previously received standard of care treatment at initial diagnosis (radiation and/or chemotherapy)
• Recurrent malignant brain tumors receiving glucocorticoid therapy: must be on stable or decreasing equivalent daily dose of glucocorticoids for 2 weeks (14 days) prior to dose assignment
• Recurrent embryonal tumors or atypical teratoid rhabdoid tumors (AT/RT): must have previously received standard of care therapy including either chemotherapy and radiation therapy or high dose chemotherapy with autologous hematopoietic stem cell support
• Grade II or III recurrent ependymoma, including RELA fusion-positive ependymoma: must have previously received radiation therapy
• Have measurable or non-measurable disease per RECIST v1.1 for relapsed solid tumors, RRC for recurrent malignant brain tumors, and INRC for recurrent neuroblastomas
• Have Lansky (age 1 - 15) / Karnofsky (age ≥ 16) Performance Score of >50 or Eastern Cooperative Oncology Group Performance Status (ECOG PS) (age ≥ 18) of 0 - 2
• Have acceptable liver function defined as:
• Total serum bilirubin ≤ 1.5 × upper limit of normal (ULN) for the study site (in subjects with known Gilbert Syndrome, total bilirubin ≤ 3 × ULN, with direct bilirubin ≤ 1.5 × ULN)
• Aspartate Transaminase (AST), Serum Glutamic Oxaloacetic Transaminase (SGOT), Alanine Transaminase (ALT), Serum Glutamic-Pyruvic Transamine (SGPT) ≤ 3 × ULN (if liver metastases are present, then ≤ 5 × ULN is allowed)
• Serum albumin ≥ 3 g/dL
• Have acceptable renal function defined as:
• Creatinine clearance or radioisotope glomerular filtration rate (GFR) ≥70 mL/min/1.73m² or a maximum serum creatinine (mg/dL)* 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 CDC (Schwartz 2009)
• Have acceptable bone marrow function defined as:
• Absolute neutrophil count (ANC) ≥ 750 cells/mm3
• Platelet count ≥ 75,000 cells/mm3

You may not qualify if…

• Subjects must not meet any of the following criteria:
• Have a concurrent or second malignancy
• Have lymphoma
• Have Grade I ependymoma
• Relapsed solid tumors: have symptomatic brain metastases or leptomeningeal disease
• Relapsed solid tumors: have received
• anticancer therapies within 2 weeks prior to dose assignment (including radiation therapy, cytotoxic agents, targeted agents or endocrine therapy)
• myelosuppressive agents within 3 weeks prior to dose assignment
• monoclonal antibodies within 4 weeks prior to dose assignment
• growth factors within 2 weeks of dose assignment
• other immunotherapy (tumor vaccine, cytokines) within 4 weeks of dose assignment
• Recurrent malignant brain tumors: have received
• anticancer therapies including: radiation therapy to current site of disease within 3 weeks dose assignment; targeted agent therapy within 2 weeks of dose assignment; nitrosoureas within 6 weeks of dose assignment; procarbazine within 3 weeks of dose assignment; other cytotoxic agents withing 4 weeks of dose assignment
• myelosuppressive agents within 4 weeks prior to dose assignment
• monoclonal antibodies within 4 weeks prior to dose assignment
• other immunotherapy (tumor vaccine, cytokines, or growth factor) within 2 weeks prior to dose assignment
• Have not recovered from toxicity of prior therapy defined as a return to ≤ grade 1 at the time of dose assignment, graded according to CTCAE v5.0 (excluding alopecia, neuropathy, and lymphopenia)
• 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 any of the following:
• Myocardial infarction within 6 months prior to initiation of screening
• History of documented congestive heart failure (New York Heart Association functional classification III-IV, see Appendix 6) within 6 months prior to initiation of screening
• Active cardiomyopathy
• Electrocardiogram (ECG) with QTc >480 msec at screening
• Echocardiogram with ejection fraction <50% or a decrease in the left ventricular shortening fraction to <27%

Where it is running

• 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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