Study to evaluate the safety and efficacy of asciminib in pediatric participants with chronic myeloid leukemia.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Decision date (initial)

2026-05-26

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2025-522138-29-00

WHO UTN

U1111-1323-0860

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Efficacy, Others

To estimate the efficacy of asciminib in pediatric participants with:
• Newly diagnosed Ph+ CML-CP without known T315I mutation
• Ph+ CML-CP without known T315I mutation resistant or intolerant to previous TKI
• Ph+ CML-CP with known T315I mutation irrespective of prior TKI treatment

Secondary objectives 4

1. To estimate additional parameters of the efficacy of asciminib in pediatric participants with: • Newly diagnosed Ph+ CML-CP without known T315I mutation • Ph+ CML-CP without known T315I mutation resistant or intolerant to previous TKI • Ph+ CML-CP with known T315I mutation irrespective of prior TKI treatment
2. To characterize the safety and tolerability of asciminib
3. To assess growth and sexual maturation
4. To characterize the PK profile of asciminib in pediatric participants

Conditions and MedDRA coding

Philadelphia positive Chronic Myeloid leukemia in Chronic Phase with newly diagnosed and resistant/intolerant to previous TKIs, with or without the T315I mutation

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Signed informed consent must be obtained prior to participation in the study
2. Male or female participants 1and <18 years of age at study enrollment
3. Diagnosis of CML-CP (Apperley et al 2025) with cytogenic confirmation of Philadelphia positive (Ph+) chromosome
4. For participants with CML-CP newly diagnosed within 3 months of screening
5. For participants with CML – CP with high risk of developing resistance or intolerance to previous TKI a. Unfavourable response to TKI is defined following the Apperley et al 2025 Guidelines as: · At three months after the initiation of therapy: BCR::ABL1 ratio > 10% IS (if confirmed within 1-3 months) · At six months after the initiation of therapy: BCR::ABL1 ratio > 10% IS · At twelve months after initiation of therapy: BCR::ABL1 ratio > 1% IS · At any time loss of previous response · At any time emergent resistant BCR::ABL1 mutations or high-risk ACA from prior TKI treatment as per local test results b. Intolerance to TKI is defined as: · Non-hematologic intolerance: participants with grade 3 or 4 toxicity while on therapy (in which case the patient is eligible whether or not there was a dose reduction); or with persistent grade 2 toxicity unresponsive to optimal management including dose adjustments (unless dose reduction is not considered in the best interest of the patient if response is already suboptimal) · Hematologic intolerance: participants with grade 3 or 4 toxicity (absolute neutrophil count [ANC] or platelets) while on therapy that is recurrent after dose reduction to the lowest doses of the TKI
6. Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.
7. Performance status: Karnofsky ≥ 50% for participants ≥ 16 years of age, and Lansky ≥ 50 for participants < 16 years of age at the time of screening.

Exclusion criteria 4

1. Known second chronic phase of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
2. Previous treatment with a hematopoietic stem-cell transplantation.
3. Patient planned to undergo allogeneic hematopoietic stem cell transplantation
4. Known presence of a BCR::ABL mutation with known resistance to study treatment in accordance with the most recent public version of international CML clinical guidelines (e.g. NCCN CML treatment guidelines v 1.2026 and Apperley et al 2025) at any time prior to study entry.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Major Molecular Response (MMR) at Week 48

Secondary endpoints 5

1. • MMR at Week 96 • MMR at and by scheduled timepoints
2. • Hematologic, cytogenetic and other molecular responses (MRs) at and by scheduled timepoints • Time to response and duration of response (limited to the binary response endpoints), time to treatment failure, time to disease progression, event free survival, overall survival
3. • Incidence of treatment-emergent adverse events (TEAEs) and other safety data as considered appropriate
4. • Height/length, weight, bone age measured by X-Ray, Tanner staging
5. • PK parameters of asciminib: AUClast, AUCtau, Cmax, Tmax, Ctrough

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 13

Locations

6 EU/EEA countries · 19 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 63 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications