NCT02828358|CompletedPhase 2ResultsFDA Drug

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

A Groupwide Pilot Study to Test the Tolerability and Biologic Activity of the Addition of Azacitidine (IND# 133688, NSC# 102816) to Chemotherapy in Infants With Acute Lymphoblastic Leukemia (ALL) and KMT2A(MLL) Gene Rearrangement

National Cancer Institute (NCI)ClinicalTrials.gov

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4 other identifiers

NCI-2016-00973s17-00488AALL15P1U10CA180886

Study Type

interventional

Target

Locations

4 countries

Sites

170

Timeline

RegisteredJul 2016

StartedApr 2017

CompletionDec 2024

Brief 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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P50-P75 for phase_2

Timeline

Completed

Started Apr 2017

Longer than P75 for phase_2

Geographic Reach

4 countries

170 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

7.8 years study duration

First Submitted

Initial submission to the registry

July 7, 2016

Completed

4 days until next milestone

First Posted

Study publicly available on registry

July 11, 2016

Completed

9 months until next milestone

Study Start

First participant enrolled

April 1, 2017

Completed

3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2020

Completed

1.1 years until next milestone

Results Posted

Study results publicly available

October 26, 2021

Completed

3.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2024

Completed

Last Updated

February 27, 2025

Status Verified

January 1, 2025

Enrollment Period

3.5 years

First QC Date

July 7, 2016

Results QC Date

October 4, 2021

Last Update Submit

February 26, 2025

Conditions

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

Outcome Measures

Primary Outcomes (1)

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)
Proportion of KMT2A-Rearranged patients treated with azacitidine with Dose Limiting Toxicities (DLTs) from the first course of azacitidine administration up to fourth course of azacitidine administration.
6 months

Secondary Outcomes (4)

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
Week 6, Day 1 (Following the induction phase (35 days), the first course of Azacitidine began around Week 6 of therapy)
Biologic Activity, Defined as Global Deoxyribonucleic Acid (DNA) Methylation Change in Peripheral Blood Mononuclear Cells (PBMC)s; Day 5 of First Course of Azacitidine
Week 6, Day 5 (Following the induction phase (35 days), the first course of Azacitidine began around Week 6 of therapy)
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
Week 13, Day 1 (Following induction phase (5 weeks), the first course of Azacitidine (1 week), and consolidation (6 weeks), the second course of Azacitidine began around Week 13 of therapy)
Biologic Activity, Defined as Global Deoxyribonucleic Acid (DNA) Methylation Change in Peripheral Blood Mononuclear Cells (PBMC)s; Day 5 of Second Course of Azacitidine
Week 13, Day 5 (Following induction phase (5 weeks), the first course of Azacitidine (1 week), and consolidation (6 weeks), the second course of Azacitidine began around Week 13 of therapy)

Other Outcomes (5)

Event-free Survival (EFS)
Five years
Minimal Residual Disease (MRD)
Five years
Pharmacokinetic (PK) Parameters of Azacitidine
Two months
+2 more other outcomes

Study Arms (1)

Treatment (azacitidine, combination chemotherapy)

EXPERIMENTAL

See Detailed Description

Drug: AzacitidineProcedure: Biospecimen CollectionProcedure: Computed TomographyDrug: CyclophosphamideDrug: CytarabineDrug: DaunorubicinDrug: Daunorubicin HydrochlorideDrug: DexamethasoneProcedure: EchocardiographyDrug: Hydrocortisone Sodium SuccinateDrug: LeucovorinDrug: Leucovorin CalciumProcedure: Magnetic Resonance ImagingDrug: MercaptopurineDrug: MethotrexateProcedure: Multigated Acquisition ScanDrug: PegaspargaseDrug: PrednisoloneDrug: ThioguanineDrug: VincristineDrug: Vincristine Sulfate

Interventions

AzacitidineDRUG

Given IV

Also known as: 5 AZC, 5-AC, 5-Azacitidine, 5-Azacytidine, 5-AZC, Azacytidine, Azacytidine, 5-, Ladakamycin, Mylosar, U-18496, Vidaza