A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, Pā¦ (NCT06191978) | Clinical Trial Compass
RecruitingPhase 1
A Phase Ia/Ib Open-label, Multiple Dose, Study to Determine the Recommended Dose, Evaluate PKs, PDs, Safety, and Activity of Venetoclax in Combination With Oral Decitabine/Cedazuridine (ASTX727) in Pediatric Patients With Relapsed/Refractory Acute Myeloid Leukemia (AML)
United States40 participantsStarted 2024-03-07
Plain-language summary
To find a recommended dose of ASTX727 (cedazuridine/decitabine) in combination with venetoclax for pediatric patients with relapsed AML.
Who can participate
Age range2 Years ā 18 Years
SexALL
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Inclusion criteria
ā. Pediatric Participants with Relapsed/refractory AML by WHO criteria.
ā. Participants must have ā„ 5% blasts in the bone marrow as assessed by morphology or flow cytometry. However, if an adequate bone marrow sample cannot be obtained, participants may be enrolled if there is unequivocal evidence of leukemia with ā„ 5% blasts in the peripheral blood or an AML defining genetic abnormality as specified by the WHO 2022 criteria.
ā. Performance status: Lansky ā„ 50 for participants who are ā¤ 16 years old and Karnofsky ā„ 50% for participants who are \> 16 years old.
ā. Age ā„2 years of age and ā¤18 years of age
ā. Able to swallow pills
ā. The following baseline laboratory data:
ā. Total serum bilirubin ā¤1.5 x upper limit of normal (ULN) for age, unless the increased value is likely attributed to disease involvement. Participants with known Gilbert's syndrome may have a total bilirubin up to ā¤5 x ULN for age.
ā. Creatinine clearance (Schwartz-Formula) or radioisotope GFR ā„ 60ml/min/1.73 m2 or a serum creatinine based on age/sex as follows:
Exclusion criteria
ā. Concomitant other anti-cancer therapy and/or participation in any other investigational clinical trials except for hydroxyurea. Concurrent hydroxyurea use should be limited to the first 2 cycles of therapy only.
ā. History of another primary invasive malignancy that has not been definitively treated and is in remission. Participants with non-melanoma skin cancers or with carcinomas in situ are eligible regardless of the time from diagnosis (including concomitant diagnoses).
What they're measuring
1
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Timeframe: Through study completion; an average of 1 year.
ā. Presence of clinically significant uncontrolled central nervous system (CNS) pathology such as epilepsy, childhood seizure, paresis, aphasia, stroke, severe brain injuries, organic brain syndrome, or psychosis.
ā. Evidence of active cerebral/meningeal disease. Participants may have history of CNS leukemic involvement if definitively treated with prior therapy and no evidence of active disease at the time of consent with at least 2 consecutive spinal fluid negative assessments for residual leukemia and negative imaging (imaging required only if previously showing evidence of CNS leukemia not otherwise documented by spinal fluid assessment).
ā. Participants with uncontrolled, active infections (viral, bacterial, or fungal) or other disease expected to interfere with the ability of the PI to assess the efficacy of the study drug. Infections controlled on concurrent anti-microbial agents are acceptable, and anti-microbial prophylaxis per institutional guidelines are acceptable.
ā. Known active hepatitis B (eg, HBsAg reactive), or hepatitis C (eg, HCV RNA \[qualitative\] is detected), or chronic hepatitis B or C infection or human immunodeficiency virus (HIV) infection in medical history (historical results within 6 months prior to informed consent is acceptable), with the following exceptions:
ā. Liver cirrhosis or other serious active liver disease or with suspected active alcohol abuse.
ā. Exclude participants with active GVHD and those on immunosuppressive drugs for treatment of GVHD. Require that participants be off calcineurin inhibitors for at least 4 weeks to be eligible.