CABL001I12202 = Asciminib in Pediatric Patients With Ph+ CML-CP [Australia, Canada, China, France, Italy, Japan, Netherlands, Poland, South Korea, Spain, USA]

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. The study will evaluate the efficacy and safety of asciminib in a pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in participants with newly diagnosed and resistant or intolerant Ph+ CML-CP.

What will happen during the study

Asciminib will be given as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets after (pediatric formulation).

Key inclusion criteria

This study includes male or female participants:

• with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP)
• who are at least 1 and less than 18 years of age at study entry
• are newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation
• who have evidence of a typical BCR::ABL1 transcript at screening
• who have a Karnofsky performance status ≥ 50% for participants ≥ 16 years of age, and aLansky performance status ≥ 50 for participants < 16 years of age at the time of screening.

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

Key exclusion criteria

This study does not include particpants:

• who are 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
• with a known presence of a BCR::ABL1 mutation with known resistance to study treatment any time prior to study entry

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

Estimated primary completion date

Where can I find additional information

You can find a study description in the US register ClinicalTrials.gov. This is a database provided by the U. S. National Institutes of Health.
You can also find a study description in the EU Clinical Trials Information System (CTIS). This is a database hosted by the European Medicines Agency (EMA).

Study centers / principal investigators

Australia

Novartis Investigative Site
Brisbane, Queensland, 4101

Novartis Investigative Site
North Adelaide, South Australia, 5066

Canada

Novartis Investigative Site
Edmonton, Alberta, T6G 1C9

Novartis Investigative Site
Montrieal, Quebec, H3T 1C5

China

Novartis Investigative Site
Zhengzhou, Henan, 450003

Novartis Investigative Site
Tianjin, 300020

France

Novartis Investigative Site
Marseille, 13885

Novartis Investigative Site
Paris, 75019

Novartis Investigative Site
Poitiers, 86021

Novartis Investigative Site
Strasbourg, 67000

Italy

Novartis Investigative Site
Roma, 00165

Japan

Novartis Investigative Site
Fukuoka, 812-8582

Novartis Investigative Site
Saitama, 330-8777

Novartis Investigative Site
Yokohama, 232-8555

Netherlands

Novartis Investigative Site
Utrecht, 3584 CS

Poland

Novartis Investigative Site
Gdansk, 80-214

Novartis Investigative Site
Wroclaw, 50-556

South Korea

Novartis Investigative Site
Yokohama, 232-8555
Seoul, 03080

Novartis Investigative Site
Seoul, 06591

Spain

Novartis Investigative Site
Barcelona, 08035

Novartis Investigative Site
Madrid, 28009

United States

New Jersey

Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, 08901
Principal Investigator: Richard Drachtman

New York

Columbia University Medical Center New York Presbyterian
New York, New York, 10032
Principal Investigator: Nobuko Hijiya

Washington

Seattle Childrens Hospital
Seattle, Washington, 98105