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CABL001I12201 = Asciminib in Pediatric Patients with CML [Europe, Japan, Republic of Korea, Russian Federation, Thailand, Turkey]

Type of study

Pediatric trial

Phase

1/2

Current status

Recruiting

Study title

CABL001I12201 = Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia

Scientific title

A multi-center, open-label study to determine the dose and safety of oral asciminib in pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with one or more tyrosine kinase inhibitors (ClinicalTrials.gov NCT04925479), EudraCT no. 2021-001286-20)

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What is the purpose of the study

The aim of this study is to support development of asciminib in patients aged 1 to <18 years with PH+ CML-CP previously treated with one or more tyrosine kinase inhibitors (TKIs). Asciminib is not yet available for pediactric patients. The study will be conducted to identify the dose and safety of asciminib as pediatric formulation.

What will happen during the study

There will be two treatment groups in this study:

  • A pediatric formulation group with patients aged 1 to <18 years who will receive a dose based on body weight (1.3 mg/kg). The dose will be given orally with food.
  • An adult formulation group with adolescent patients aged 14 to <18 years, weighing at least 40 kg, who will receive a flat dose of 40 mg orally twice daily under fasted conditions.

The total duration of the treatment period of the study will be 5 years (260 weeks). Participants who, according to Investigator’s judgement, are benefiting from study treatment will remain on treatment up to the completion of the treatment period.

Key inclusion criteria

This study includes male or female patients:

  • with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (PH+ CML-CP)
  • in the pediatric formulation group: who are at least 1 and less than 18 years of age at study entry;
    in the adult formulation group: who are at least 14 and less than 18 years of age with a body weight of at least 40 kg at study entry
  • who meet all laboratory values specified in the study protocol.
  • who have previously been treated with at least one TKI.

Key exclusion criteria

This study does not include patients:

  • with a known presence of the T315I mutation prior to study entry
  • in second chronic phase of CML after previous progression to advanced phase (AP) or blast crisis (BC)
  • who have undergone hematopoietic stem cell transplantation or are planning to undergo allogeneic stem cell transplantation.

Further criteria may apply. Please discuss these with your doctor or study staff.

Estimated primary completion date

December 2025

Where can I find additional information

Study description in the US register ClinicalTrials.gov, a service of the U. S. National Institutes of Health

Study description in the EU Clinical Trials Register which is hosted by the European Medicines Agency (EMA)

Study sponsor

Novartis Pharmaceuticals

Scientific lead / contact

Novartis Pharmaceuticals

Principal investigator

Novartis Pharmaceuticals

Study centers / principal investigators

France

Novartis Investigative Site
Bordeaux, 33076

Novartis Investigative Site
Lille, 59000

Novartis Investigative Site
Poitiers, 86021


Germany

Novartis Investigative Site
Erlangen, 91054

Novartis Investigative Site
Essen, 45147

Novartis Investigative Site
Hamburg, 20246


Greece

Novartis Investigative Site
Athens, 115 27


Hungary

Novartis Investigative Site
Budapest, 1094


Italy

Novartis Investigative Site
Genova, 16147

Novartis Investigative Site
Monza, 20900


Japan

Novartis Investigative Site
Osaka, 534-0021

Novartis Investigative Site
Okohama-city, Kanagawa, 232-8555

Novartis Investigative Site
Shinjuku-ku, Tokyo, 160 8582


Korea, Republic of

Novartis Investigative Site
Seoul, 03080

Novartis Investigative Site
Wrocław, 50367


Russian Federation

Novartis Investigative Site
Moscow, 117198

Novartis Investigative Site
Saint Petersburg, 197022


Thailand

Novartis Investigative Site
Bangkok, 10400

Novartis Investigative Site
Khon Kaen


Turkey

Novartis Investigative Site
Bursa, Gorukle, 16059

Novartis Investigative Site
Istanbul, 34093