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