NCT03031249

Brief Summary

In this open-label, randomized, prospective clinical trial, nucleophosmin-1(NPM1) mutated acute myeloid leukemia (AML) patients who have reached CR are randomized into two groups.The control group receive high-dose cytarabine(HDAC) regimen while the experimental group receive high dose of cytarabine plus tretinoin(ATRA) and arsenic trioxide(ATO) treatment.The safety and efficacy of ATRA and ATO is evaluated.

Trial Health

75
On Track

Trial Health Score

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

Enrollment
80

participants targeted

Target at P75+ for phase_1

Timeline
17mo left

Started Feb 2017

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
enrolling by invitation

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

Study Progress87%
Feb 2017Oct 2027

First Submitted

Initial submission to the registry

January 12, 2017

Completed
13 days until next milestone

First Posted

Study publicly available on registry

January 25, 2017

Completed
14 days until next milestone

Study Start

First participant enrolled

February 8, 2017

Completed
9.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 15, 2026

Expected
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

October 15, 2027

Last Updated

May 30, 2025

Status Verified

February 1, 2025

Enrollment Period

9.3 years

First QC Date

January 12, 2017

Last Update Submit

May 26, 2025

Conditions

AML

Outcome Measures

Primary Outcomes (1)

  • Relapse-Free Survival Rate (RFS)

    RFS is defined as the time from the date of complete remission (CR) after entry in this trial until the date of documented relapse or death for NPM1 mutated leukemia patients who achieve CR.

    Within 5 years after randomization

Secondary Outcomes (3)

  • Non-relapse Mortality

    through treatment completion, an average of 5 months

  • Overall Survival Rate (OS)

    Within 5 years after randomization

  • Cumulative incidence of relapse

    Within 5 years after randomization

Study Arms (2)

High Dose of Cytarabine

ACTIVE COMPARATOR

Patients receive high dose of cytarabine.

Drug: Cytarabine

HDAC + ATRA + ATO

EXPERIMENTAL

Patients receive high dose of cytarabine plus ATRA and ATO treatment.

Drug: CytarabineDrug: all-trans retinoic acidDrug: Arsenic Trioxide

Interventions

CytarabineDRUG

Cytarabine at a dose of 3g/㎡/d on the first, third and fifth day.

Also known as: HDAC
HDAC + ATRA + ATOHigh Dose of Cytarabine
all-trans retinoic acidDRUG

ATRA at a dose of 30mg/㎡/d on day 1-14.

Also known as: ATRA
HDAC + ATRA + ATO
Arsenic TrioxideDRUG

ATO at a dose of 10mg/d on day 1-14

Also known as: ATO
HDAC + ATRA + ATO

Eligibility Criteria

Age14 Years - 55 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age of 14 to 55 years old;
  • Patients that meet the diagnostic criteria(WHO 2008 criteria) of AML (except APL subtypes) and with NPM1-mutated.
  • Reached CR after induction regimen.
  • ECOG score of ≤ 2;
  • Patients with eligible laboratory examination including liver,renal and heart function.
  • Adult patients are willing to participate in the study and sign the informed consent by themselves or by their immediate family. Patients under 18 years old willing to participate should have their legal guardians sign the informed consent.

You may not qualify if:

  • Secondary leukemia.
  • Patients had other tumor at active stage or had received radiotherapy or chemotherapy in the last 6 months due to other tumor.
  • Patients with other blood diseases(for example, haemophiliacs) are excluded.However, patients with abnormal blood count, but with undiagnosed MDS or MPD patients are included.
  • Acute panmyelosis with myelofibrosis and myeloid sarcoma patients;
  • With BCR-ABL fusion gene;
  • Pregnant or lactating women;
  • With ineligible renal or liver function;
  • With active cardiovascular disease;
  • Severe infection disease including uncured tuberculosis pulmonary aspergillosis;
  • AIDS;
  • Patients had central nervous system involvement when they were diagnosed as AML.
  • Patients with epilepsy or dementia or other mental disease who couldn't understand or follow the research.
  • Drugs, medical, mental or social situation may distract patients from following the research or being evaluated the results.
  • Patients with other factors which were considered unsuitable to participate in the study by the investigators.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China

Location

MeSH Terms

Interventions

CytarabineTretinoinArsenic Trioxide

Intervention Hierarchy (Ancestors)

CytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsArabinonucleosidesNucleosidesNucleic Acids, Nucleotides, and NucleosidesVitamin ARetinoidsCarotenoidsPolyenesAlkenesHydrocarbons, AcyclicHydrocarbonsOrganic ChemicalsCyclohexenesCyclohexanesCycloparaffinsHydrocarbons, AlicyclicHydrocarbons, CyclicTerpenesDiterpenesPigments, BiologicalBiological FactorsArsenicalsInorganic ChemicalsOxidesOxygen Compounds

Study Officials

  • Jianxiang Wang

    Institute of Hematology & Blood Diseases Hospital, China

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 12, 2017

First Posted

January 25, 2017

Study Start

February 8, 2017

Primary Completion (Estimated)

May 15, 2026

Study Completion (Estimated)

October 15, 2027

Last Updated

May 30, 2025

Record last verified: 2025-02

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)