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AMLPhase 1/2NCT03031249

Efficacy and Safety of ATO Plus ATRA in Nucleophosmin-1 Mutated Acute Myeloid Leukemia

A Phase 1/2 study testing Cytarabine, all-trans retinoic acid and Arsenic Trioxide in people with AML, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.

Enrolling by invitation

Enrolling by invitation. The study team selects and invites participants, usually from an existing group of patients.

Sites and contacts
Official record
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Trial 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… Read more about this study

Who is it for?
Ages 14 to 55 years
Conditions the study is for
AML
What is being tested?
Cytarabine, all-trans retinoic acid and Arsenic Trioxide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
80 participants
Where
1 siteChina
Study dates
8 Feb 2017to 15 Oct 2027
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 14 to 55 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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.

Who cannot join

  • 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;
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  • 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.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

China1 site

  • Institute of Hematology & Blood Diseases Hospital

    Tianjin, Tianjin Municipality

Other trials in China

About this study

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.

Read the detailed description

In this open-label, randomized, prospective clinical trial, NPM1- mutated AML patients who have reached CR are randomized into two groups.

In experimental group, patients receive cytarabine at a dose of 3g/㎡/d on the first, third and fifth day, ATRA at a dose of 30mg/㎡/d on day 1-14 and ATO at a dose of 0.15mg/kg/d (maximum, 10mg/d) on day 1-14. Patients in control group only receive high dose of cytarabine.

The safety and efficacy of ATRA plus ATO regimen is evaluated.The primary outcome is relapse-free survival rate after treatment.

Conditions
AML
Being tested
Cytarabine, all-trans retinoic acid, Arsenic Trioxide

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CytarabineDrug

    Also called HDAC

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

  • all-trans retinoic acidDrug

    Also called ATRA

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

  • Arsenic TrioxideDrug

    Also called ATO

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

Study groups in NCT03031249
Study groupRoleWhat they receive
High Dose of CytarabineActive comparatorPatients receive high dose of cytarabine.
Receives: Drug: Cytarabine
HDAC + ATRA + ATOExperimentalPatients receive high dose of cytarabine plus ATRA and ATO treatment.
Receives: Drug: Cytarabine; Drug: all-trans retinoic acid; Drug: Arsenic Trioxide

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Within 5 years after randomization

Secondary outcomes (3)

  1. Non-relapse Mortality

    Measured: through treatment completion, an average of 5 months

  2. Overall Survival Rate (OS)

    Measured: Within 5 years after randomization

  3. Cumulative incidence of relapse

    Measured: Within 5 years after randomization

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Jan 2017 (estimated)
  2. Study start8 Feb 2017
  3. Primary completion15 May 2026 (estimated)
  4. Study completion15 Oct 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
80 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03031249
Sponsor's protocol ID
IIT2016007（ATO + ATRA）
Status last verified
Feb 2025
First submitted
12 Jan 2017
Last updated
30 May 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Cytarabine, all-trans retinoic acid and Arsenic Trioxide in people with AML, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: enrolling by invitation, as of the sponsor's last update on 30 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Relapse-Free Survival Rate (RFS) (time frame: Within 5 years after randomization). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03031249.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 May 2025; AzTrials last refreshed its copy on 8 Nov 2026.

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