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Asciminib Pediatric formulation group

Phase 1

Myeloid Leukemia, Philadelphia Positive | Small molecule | Oncology |Novartis AG|Last Updated: Aug 27, 2026

Target and mechanism

Molecular targetABL1, BCR
Target classInhibitor
ModalitySmall molecule

Also known as Asciminib Pediatric formulation

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials1
Total Enrollment34

FDA Designations

No designations recorded

Clinical trial landscape

Asciminib Pediatric formulation group · 1 trial · 1 indication

Phase 1 1
NCT04925479Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid LeukemiaMyeloid Leukemia, Philadelphia Positive
RECRUITING34 Analytics
PHASE1RECRUITING
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
Myeloid Leukemia, Philadelphia PositiveUnlock trial analytics

Study Endpoints

Primary Endpoints

Primary Pharmacokinetic (PK) parameter: AUClast
52 weeks

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Primary PK parameter: AUCtau
52 weeks

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary PK parameter: Cmax
52 weeks

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary PK parameter: Tmax
52 weeks

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary PK parameter: Ctrough
52 weeks

Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).

Secondary Endpoints

Hematologic responses
52 weeks
Molecular responses
52 weeks
Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks
after first dose at Week 1 Day 1, 4 weeks, 52 weeks
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
AsciminibEXPERIMENTALThis arm consists of 2 groups: * The pediatric formulation group where the dose is based on body weight (1.3mg/kg BID or 2.6 mg/kg QD) * The adult formulation group where participants will receive a flat dose of 40mg BID

Interventions

NameTypeDescription
Asciminib Pediatric formulation groupDRUGAsciminib Pediatric formulation group: 1 mg film-coated granules in a size 0 capsule will be supplied, taken orally (capsules are a container for the granules and are not ingested): 10 mg (10x 1 mg film-coated granules in capsule) 15 mg (15x 1 mg film-coated granules in capsule) 30 mg (30x 1 mg film-coated granules in capsule)
Asciminib Adult formulation groupDRUGAsciminib Adult formulation group: 40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally.
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Eligibility Criteria

Age Range1 Year to 17 Years
SexALL
Healthy VolunteersNo
Study Sites39

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 follo...

Countries:United StatesChinaFranceGermanyGreeceHungaryItalyJapanNetherlandsPolandRussiaSouth KoreaThailandTurkey (Türkiye)
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Recent Changes (Last 90 Days)

LOWAug 27, 2026NCT04925479lastUpdatePostDate: changed
LOWAug 27, 2026NCT04925479lastUpdatePostDate: changed
MEDIUMJun 30, 2026NCT04925479primaryCompletionDate: changed
MEDIUMJun 30, 2026NCT04925479primaryCompletionDate: changed
MEDIUMJun 30, 2026NCT04925479primaryCompletionDate: changed

Frequently asked questions about Asciminib Pediatric formulation group

What is Asciminib Pediatric formulation used for?

Asciminib Pediatric formulation is an investigational small molecule being studied for the treatment of Philadelphia positive myeloid leukemia, a type of blood cancer. It is currently in Phase 1 clinical development, meaning it has not yet been approved by regulatory authorities and remains under investigation for safety and dosing in pediatric patients.

Who is developing Asciminib Pediatric formulation?

Asciminib Pediatric formulation is being developed by Novartis AG, a global pharmaceutical company listed on the stock exchange under the ticker symbol NVS. The company is conducting clinical research to evaluate the drug's safety and appropriate dosing in pediatric patients with Philadelphia positive myeloid leukemia.

What phase is Asciminib Pediatric formulation in?

Asciminib Pediatric formulation is currently in Phase 1 clinical development. This early-stage trial is designed to determine the appropriate dose and assess the safety of the drug in pediatric patients with Philadelphia positive myeloid leukemia. The drug is investigational and has not yet received regulatory approval.

What clinical trials is Asciminib Pediatric formulation in?

Asciminib Pediatric formulation is being studied in a Phase 1 clinical trial registered as NCT04925479, titled "Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia." The trial is currently recruiting participants and aims to enroll 34 patients across multiple countries, including the United States, China, France, Germany, and Japan.

Is Asciminib Pediatric formulation the same as Asciminib?

Asciminib Pediatric formulation is also known as Asciminib Pediatric formulation, and it is a pediatric-specific formulation of the drug asciminib. The pediatric formulation is being developed specifically for use in children and adolescents, with a dedicated clinical trial focused on determining the appropriate dose and safety in this younger patient population.

What does Asciminib Pediatric formulation target?

Asciminib Pediatric formulation belongs to the kinase inhibitor class of drugs, specifically a -nib type kinase inhibitor. Kinase inhibitors work by blocking the activity of specific enzymes involved in cancer cell growth. In the context of Philadelphia positive myeloid leukemia, this mechanism is intended to interfere with the signaling pathways that drive the disease.