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Phase 2 N=78 Treatment

Azacitidine and Combination Chemotherapy in Treating Infants With Acute Lymphoblastic Leukemia and KMT2A Gene Rearrangement

Acute Leukemia of Ambiguous Lineage · B Acute Lymphoblastic Leukemia · Mixed Phenotype Acute Leukemia

Enrolled (actual)
78
Serious AEs
44.9%
Results posted
Oct 2021
Primary outcome: Primary: Tolerability of Azacitidine in Combination With Interfant-06 Standard Chemotherapy in Evaluable Infant Patients With Newly Diagnosed ALL With KMT2A Gene Rearrangement (KMT2A-R). KMT2A Gene Rearrangement (KMT2A-R) — 6.45 percentage of participants

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Azacitidine (Drug); Biospecimen Collection (Procedure); Computed Tomography (Procedure); Cyclophosphamide (Drug); Cytarabine (Drug); Daunorubicin (Drug); Daunorubicin Hydrochloride (Drug); Dexamethasone (Drug); Echocardiography (Procedure); Hydrocortisone Sodium Succinate (Drug); Leucovorin (Drug); Leucovorin Calcium (Drug); Magnetic Resonance Imaging (Procedure); Mercaptopurine (Drug); Methotrexate (Drug); Multigated Acquisition Scan (Procedure); Pegaspargase (Drug); Prednisolone (Drug); Thioguanine (Drug); Vincristine (Drug); Vincristine Sulfate (Drug)
Age
Pediatric
Sex
All
Sponsor
National Cancer Institute (NCI)
Primary completion
Sep 2020

Outcome Measures

OutcomeResultp-value
PRIMARY
Tolerability of Azacitidine in Combination With Interfant-06 Standard Chemotherapy in Evaluable Infant Patients With Newly Diagnosed ALL With KMT2A Gene Rearrangement (KMT2A-R). KMT2A Gene Rearrangement (KMT2A-R)
6.45
SECONDARY
Biologic Activity, Defined as Global Deoxyribonucleic Acid (DNA) Methylation Change in Peripheral Blood Mononuclear Cells (PBMC)s; Day 1 Prior to First Course of Azacitidine
78.17
SECONDARY
Biologic Activity, Defined as Global Deoxyribonucleic Acid (DNA) Methylation Change in Peripheral Blood Mononuclear Cells (PBMC)s; Day 5 of First Course of Azacitidine
75.54
SECONDARY
Biologic Activity, Defined as Global Deoxyribonucleic Acid (DNA) Methylation Change in Peripheral Blood Mononuclear Cells (PBMC)s; Day 1 Prior to Second Course of Azacitidine
76.5
SECONDARY
Biologic Activity, Defined as Global Deoxyribonucleic Acid (DNA) Methylation Change in Peripheral Blood Mononuclear Cells (PBMC)s; Day 5 of Second Course of Azacitidine
74.52

Summary

This pilot phase II trial studies the side effects of azacitidine and combination chemotherapy in infants with acute lymphoblastic leukemia and KMT2A gene rearrangement. Drugs used in chemotherapy, such as methotrexate, prednisolone, daunorubicin hydrochloride, cytarabine, dexamethasone, vincristine sulfate, pegaspargase, hydrocortisone sodium succinate, azacitidine, cyclophosphamide, mercaptopurine, leucovorin calcium, and thioguanine work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug may kill more cancer cells.

Eligibility Criteria

Inclusion Criteria

  • Infants must be > 36 weeks gestational age at the time of enrollment
  • Patients must have newly diagnosed B lymphoblastic leukemia (2008 World Health Organization [WHO] classification) (also termed B-precursor acute lymphoblastic leukemia) or acute leukemia of ambiguous lineage (ALUL), which includes mixed phenotype acute leukemia (MPAL); for patients with ALUL, the morphology and immunophenotype must be at least 50% B lymphoblastic
  • Central nervous system (CNS) status must be determined based on a sample obtained prior to the administration of any systemic or intrathecal chemotherapy, with the exception of steroid pretreatment

Exclusion Criteria

  • Patients with known absence of KMT2A-rearrangement leukemia prior to enrollment
  • Patients with Down syndrome
  • Patients with secondary B acute lymphoblastic leukemia (B-ALL) that developed after treatment of a prior malignancy with cytotoxic chemotherapy
  • With the exception of steroid pretreatment or the administration of intrathecal methotrexate or intrathecal cytarabine, receipt of any other prior cytotoxic chemotherapy for either the current diagnosis of B-ALL or any cancer diagnosed prior to the initiation of protocol therapy on AALL15P1
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02828358). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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