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Promising new pill for kids with chronic leukemia enters testing

NCT ID NCT04925479

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 28, 2026 · Updated 4 times

Summary

This study is testing an oral medication called asciminib in children aged 1 to 18 with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML-CP) who have already tried other treatments. The goal is to find the right dose that works as well as it does in adults and to check that it is safe. About 34 children will take part in this early-phase trial.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 34 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2021

Expected to finish

Nov 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 year to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male or female participants: * Pediatric formulation group: ≥ 1 and less than 18 years of age at study entry. * Adult formulation group: ≥ 14 and less than 18 years of age and body weight of ≥ 40 kg at study entry. * Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test. 1. \< 15% blasts in peripheral blood and bone marrow 2. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow 3. \< 20% basophils in the peripheral blood 4. Neutrophils ≥ 1.5 x 10\^9/L (or WBC ≥ 3 x 10\^9/L if neutrophils are not available) and platelet count ≥ 100 x 10\^9/L 5. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly * Prior treatment with a minimum of one TKI * Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening. * Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients \< 16 years of age at the time of screening * Participants must have adequate renal, hepatic, pancreatic and cardiac function * Participants must have electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication: * Evidence of typical BCR::ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification. Exclusion Criteria: * Known presence of the T315I mutation prior to study entry or a BCR::ABL mutation with known resistance to study treatment any time prior to study entry. * Known second chronic phase of CML after previous progression to AP/BC. * Previous treatment with a hematopoietic stem-cell transplantation. * Patient planning to undergo allogeneic hematopoietic stem cell transplantation. * Cardiac or cardiac repolarization abnormality * Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol * History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis. * History of acute or chronic liver disease. * Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug * Pregnant or nursing (lactating) females. Other protocol-defined inclusion/exclusion may apply.

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Conditions

The condition(s) this trial relates to.

chronic myelogenous leukemia, BCR-ABL1 positive Leukemia, Myelogenous, Chronic, BCR-ABL Positive Philadelphia-positive myelogenous leukemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Myeloid leukemia, philadelphia positive

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    38 sites in 13 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Childrens Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104-4399, United States

  • Cinn Children Hosp Medical Center

    RECRUITING

    Cincinnati, Ohio, 45229-3039, United States

  • Columbia University Medical Center New York Presbyterian

    RECRUITING

    New York, New York, 10032, United States

  • Dana Farber Cancer Institute

    RECRUITING

    Boston, Massachusetts, 02215, United States

  • Indiana UH Riley H for CIU

    RECRUITING

    Indianapolis, Indiana, 46202-5167, United States

  • Novartis Investigative Site

    RECRUITING

    Hangzhou, Zhejiang, 310052, China

  • Novartis Investigative Site

    RECRUITING

    Beijing, 100044, China

  • Novartis Investigative Site

    RECRUITING

    Shanghai, 200127, China

  • Novartis Investigative Site

    RECRUITING

    Tianjin, 300020, China

  • Novartis Investigative Site

    RECRUITING

    Bordeaux, 33076, France

  • Novartis Investigative Site

    RECRUITING

    Lille, 59000, France

  • Novartis Investigative Site

    RECRUITING

    Paris, 75019, France

  • Novartis Investigative Site

    RECRUITING

    Poitiers, 86021, France

  • Novartis Investigative Site

    RECRUITING

    Erlangen, 91054, Germany

  • Novartis Investigative Site

    RECRUITING

    Essen, 45147, Germany

  • Novartis Investigative Site

    RECRUITING

    Hamburg, 20246, Germany

  • Novartis Investigative Site

    RECRUITING

    Athens, 115 27, Greece

  • Novartis Investigative Site

    WITHDRAWN

    Budapest, 1083, Hungary

  • Novartis Investigative Site

    RECRUITING

    Genova, GE, 16147, Italy

  • Novartis Investigative Site

    RECRUITING

    Monza, MB, 20900, Italy

  • Novartis Investigative Site

    RECRUITING

    Roma, RM, 00165, Italy

  • Novartis Investigative Site

    RECRUITING

    Torino, TO, 10126, Italy

  • Novartis Investigative Site

    RECRUITING

    Yokohama, Kanagawa, 2328555, Japan

  • Novartis Investigative Site

    RECRUITING

    Shinjuku-ku, Tokyo, 1608582, Japan

  • Novartis Investigative Site

    RECRUITING

    Osaka, 5340021, Japan

  • Novartis Investigative Site

    RECRUITING

    Utrecht, 3584 CS, Netherlands

  • Novartis Investigative Site

    RECRUITING

    Wroclaw, 50367, Poland

  • Novartis Investigative Site

    RECRUITING

    Moscow, 117198, Russia

  • Novartis Investigative Site

    RECRUITING

    Saint Petersburg, 197022, Russia

  • Novartis Investigative Site

    RECRUITING

    Seoul, 03080, South Korea

  • Novartis Investigative Site

    RECRUITING

    Seoul, 05505, South Korea

  • Novartis Investigative Site

    RECRUITING

    Khon Kaen, THA, 40002, Thailand

  • Novartis Investigative Site

    RECRUITING

    Bangkok, 10400, Thailand

  • Novartis Investigative Site

    RECRUITING

    Chiang Mai, 50200, Thailand

  • Novartis Investigative Site

    RECRUITING

    Istanbul, Fatih, 34093, Turkey (Türkiye)

  • Novartis Investigative Site

    RECRUITING

    Bursa, Nilufer, 16059, Turkey (Türkiye)

  • Uni Of Texas MD Anderson Cancer Ctr

    RECRUITING

    Houston, Texas, 77024, United States

  • University Of Utah

    RECRUITING

    Salt Lake City, Utah, 84132, United States

  • University of Mississippi Medical Center

    RECRUITING

    Jackson, Mississippi, 39216-4505, United States

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