A Phase 1 Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Pat… (NCT02141828) | Clinical Trial Compass
CompletedPhase 1
A Phase 1 Dose Escalation and Expanded Cohort Study of EPZ-5676 in the Treatment of Pediatric Patients With Relapsed/Refractory Leukemias Bearing a Rearrangement of the MLL Gene
United States18 participantsStarted 2014-05
Plain-language summary
A subset of patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) harbor rearrangements of the MLL gene, which are detected either by cytogenetic or fluorescent in situ hybridization evaluation at the time of diagnosis. A protein called DOT1L plays an important role in the malignant process in these leukemias. EPZ-5676 is a molecule that blocks the activity of DOT1L, and is therefore being evaluated in the treatment of patients with MLL-rearranged leukemias.
Who can participate
Age range3 Months – 18 Years
SexALL
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Inclusion criteria
✓. Age: \>3 months to \<18 years of age.
✓. Diagnosis: Patients must have documented relapsed/refractory ALL, AML, or acute leukemia of ambiguous lineage and meet the following criteria:
✓. Therapeutic Options: Patients must be ineligible or inappropriate for other treatment regimens known to have curative potential.
✓. Performance Level: Karnofsky \> 50% for pts \> 12 years; Lansky \> 50% for pts \< 12 years of age.
✓. Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
✓. Renal and Hepatic Function: Patient must have adequate renal and hepatic functions as indicated by the following laboratory values:
✓. Cardiac Function: Patient must have a shortening fraction (SF) of \> 27% or an ejection fraction (EF) of \> 50% by echocardiogram or MUGA scan.
Exclusion criteria
✕. Patients with CNS 3 disease or symptomatic CNS disease
What they're measuring
1
Determine the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of EPZ-5676.
Timeframe: 12 months
2
To assess the safety and tolerability of EPZ-5676 administered as a continuous intravenous (CIV) infusion
✕. Clinically active heart disease including prolonged QTc or prolonged PR interval, or history of arrhythmias
✕. On immunosuppressive or other anti-leukemic therapy, excluding patients receiving glucocorticoids for management of circulating blast count or patients on a stable dose (\<20mg/m2/day prednisone or equivalent) of systemic or topical glucocorticoid therapy with ≤ Grade 1 GvHD or tapering dose of calcineurin inhibitor
✕. Patients with known bleeding diathesis or prothrombin time (PT) or aPTT \>1.5 x ULN or fibrinogen \<0.5 x LLN
✕. Receiving prophylactic use of hematopoietic colony stimulating factors
✕. Known history of infection with human immunodeficiency virus (HIV) or chronic infection with hepatitis B virus (HBsAg positive) or hepatitis C virus (anti-HCV positive)
✕. Being actively treated for another concurrent malignancy