Esta página se tradujo automáticamente y no se garantiza la precisión de la traducción. por favor refiérase a versión inglesa para un texto fuente.

Estudio para determinar la eficacia y seguridad de Asciminib en pacientes pediátricos con LMC-CP Ph+

9 de noviembre de 2026 actualizado por: Novartis Pharmaceuticals

Un estudio de fase II, multicéntrico, abierto, de un solo brazo para evaluar la seguridad y eficacia de asciminib en participantes pediátricos recién diagnosticados o previamente tratados por leucemia mieloide crónica Filadelfia positiva en fase crónica (Ph+ LMC-FC) con o sin mutación T315I conocida

The aim of this study is to support development of asciminib in the pediatric population (1 to < 18 years) with Ph+ CML-CP. The study will evaluate the efficacy and safety of asciminib in pediatric formulation (weigh-based dose, fed state) or adult formulation (fasted) in newly diagnosed and resistant or intolerant Ph+ CML-CP with or without T315I mutation.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

This is a multi-center, open-label, single arm study of asciminib in pediatric participants aged

1 to <18 years old with Ph+ CML-CP newly diagnosed and previously treated with TKI treatment, with or without T315I mutation.

The study population will consist of three cohorts of Ph+ CML-CP pediatric participants:

  • Newly-diagnosed Ph+ CML-CP participants without known T315I mutation
  • Ph+ CML-CP participants resistant or intolerant to previous TKI without known T315I mutation
  • Ph+ CML-CP participants with known T315I mutation irrespective of prior TKI treatment

There is no fixed duration of study treatment for the participants. The study will end 5 years (240 weeks) after the last enrolled participants received their first dose of treatment in the study. The objective is to have enough follow up for safety, including growth and development and efficacy.

Tipo de estudio

Intervencionista

Inscripción (Estimado)

50

Fase

  • Fase 2

Acceso ampliado

Disponible fuera del ensayo clínico. Ver registro de acceso ampliado.

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Copia de seguridad de contactos de estudio

  • Nombre: Novartis Pharmaceuticals
  • Número de teléfono: +41613241111

Ubicaciones de estudio

  • Alemania
      • Erlangen, Alemania, 91054
        • Reclutamiento
        • Novartis Investigative Site
      • Hamburg, Alemania, 20246
        • Reclutamiento
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main, Hesse, Alemania, 60590
        • Reclutamiento
        • Novartis Investigative Site
  • Australia
    • Queensland
      • Brisbane, Queensland, Australia, 4101
        • Reclutamiento
        • Novartis Investigative Site
    • South Australia
      • North Adelaide, South Australia, Australia, 5066
        • Reclutamiento
        • Novartis Investigative Site
  • Brasil
    • Rio de Janeiro
      • Rio de Janeiro, Rio de Janeiro, Brasil, 20211-030
        • Reclutamiento
        • Novartis Investigative Site
    • São Paulo
      • Barretos, São Paulo, Brasil, 14784-400
        • Reclutamiento
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brasil, 04829-310
        • Reclutamiento
        • Novartis Investigative Site
  • Canadá
    • Alberta
      • Edmonton, Alberta, Canadá, T6G 1C9
        • Reclutamiento
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, Canadá, H3T 1C5
        • Reclutamiento
        • Novartis Investigative Site
  • Corea del Sur
      • Seoul, Corea del Sur, 03080
        • Reclutamiento
        • Novartis Investigative Site
      • Seoul, Corea del Sur, 06591
        • Reclutamiento
        • Novartis Investigative Site
  • España
      • Barcelona, España, 08035
        • Reclutamiento
        • Novartis Investigative Site
      • Madrid, España, 28046
        • Reclutamiento
        • Novartis Investigative Site
      • Madrid, España, 28009
        • Reclutamiento
        • Novartis Investigative Site
  • Estados Unidos
    • Illinois
      • Chicago, Illinois, Estados Unidos, 60611
        • Reclutamiento
        • Ann and Robert H Lurie Childs Hosp
        • Investigador principal:
          • Sara Zarnegar-Lumley
        • Contacto:
    • New Jersey
      • New Brunswick, New Jersey, Estados Unidos, 08901
        • Reclutamiento
        • Rutgers Cancer Institute of New Jersey
        • Investigador principal:
          • Richard DRACHTMAN
        • Contacto:
    • New York
      • New York, New York, Estados Unidos, 10032
        • Reclutamiento
        • Columbia University Medical Center New York Presbyterian
        • Investigador principal:
          • Nobuko Hijiya
        • Contacto:
    • Ohio
      • Cincinnati, Ohio, Estados Unidos, 45229-3039
        • Reclutamiento
        • Cinn Children Hosp Medical Center
        • Investigador principal:
          • Benjamin Mizukawa
        • Contacto:
    • Washington
      • Seattle, Washington, Estados Unidos, 98105
        • Reclutamiento
        • Seattle Childrens Hospital
        • Contacto:
        • Investigador principal:
          • Todd Cooper
  • Francia
      • Marseille, Francia, 13885
        • Reclutamiento
        • Novartis Investigative Site
      • Paris, Francia, 75019
        • Reclutamiento
        • Novartis Investigative Site
      • Poitiers, Francia, 86021
        • Reclutamiento
        • Novartis Investigative Site
      • Strasbourg, Francia, 67000
        • Reclutamiento
        • Novartis Investigative Site
  • Hungría
    • Hajdu Bihar Megye
      • Debrecen, Hajdu Bihar Megye, Hungría, 4032
        • Reclutamiento
        • Novartis Investigative Site
  • Italia
    • PD
      • Padova, PD, Italia, 35128
        • Reclutamiento
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italia, 00165
        • Reclutamiento
        • Novartis Investigative Site
  • Japón
      • Fukuoka, Japón, 8128582
        • Reclutamiento
        • Novartis Investigative Site
      • Saitama, Japón, 3308777
        • Reclutamiento
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama, Kanagawa, Japón, 2328555
        • Reclutamiento
        • Novartis Investigative Site
  • Países Bajos
      • Utrecht, Países Bajos, 3584 CS
        • Reclutamiento
        • Novartis Investigative Site
  • Polonia
      • Gdansk, Polonia, 80-214
        • Reclutamiento
        • Novartis Investigative Site
      • Wroclaw, Polonia, 50-556
        • Reclutamiento
        • Novartis Investigative Site
  • Porcelana
      • Tianjin, Porcelana, 300020
        • Reclutamiento
        • Novartis Investigative Site
    • Henan
      • Zhengzhou, Henan, Porcelana, 450003
        • Reclutamiento
        • Novartis Investigative Site
  • Singapur
      • Singapore, Singapur, 229899
        • Reclutamiento
        • Novartis Investigative Site
  • Tailandia
      • Bangkok, Tailandia, 10330
        • Reclutamiento
        • Novartis Investigative Site
      • Bangkok, Tailandia, 10700
        • Reclutamiento
        • Novartis Investigative Site
  • Vietnam
      • Hanoi, Vietnam, 100000
        • Reclutamiento
        • Novartis Investigative Site

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

1 año a 18 años (Niño, Adulto)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Key Inclusion Criteria:

Participants eligible for inclusion in this study must meet all of the following criteria:

  1. Signed informed consent must be obtained prior to participation in the study.

  2. Male or female participants 1 and < 18 years of age at study enrollment

  3. Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome

  4. For participants with CML-CP newly diagnosed within 3 months of screening OR 5 For participants with CML - CP with high risk of developing resistance or intolerance to previous TKI:

    1. 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

    2. 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.

Key Exclusion Criteria:

  1. Known second chronic phase (CP) 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::ABL1 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) any time prior to study entry

Other inclusion/exclusion criteria may apply.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: N / A
  • Modelo Intervencionista: Asignación de un solo grupo
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Single arm study

This study will enroll pediatric patients (≥ 1 and < 18 years of age) with newly diagnosed or previously treated with Philadelphia positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) with or without known T315I mutation
Droga: Asciminib single agent

Asciminib (labelled as ABL001) administered as 40 mg tablet (adult formulation) or as 1 mg film-coated granules mini-tablets (pediatric formulation)

Otros nombres:
  • ABL001

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
MMR at Week 48
Periodo de tiempo: 48 weeks

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
48 weeks

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Tiempo hasta la progresión de la enfermedad
Periodo de tiempo: 5 años
5 años
MMR at Week 96
Periodo de tiempo: 96 weeks

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
96 weeks
MMR at and by scheduled timepoints
Periodo de tiempo: Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96

MMR is defined as BCR::ABL1 IS ≤0.1%. MMR is defined as a ≥ 3.0 log reduction in BCR::ABL1 transcripts compared to the standardized baseline equivalent to ≤ 0.1 % BCR::ABL1/ABL % by international scale as measured by RQ-PCR.
Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Hematologic response at and by scheduled timepoints
Periodo de tiempo: Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96

Complete hematologic response (CHR) is defined as all of the following present for ≥ 4 weeks (which means present at least at 2 visits 4-week apart, with no intermediate visit showing no CHR):

  • White blood cell(s) (WBC) count < 10 x 10^9/L
  • Platelet count < 450 x 10^9/L
  • Basophils < 5%
  • No blasts and promyelocytes in peripheral blood
  • Myelocytes + metamyelocytes < 5% in peripheral blood
  • No evidence of extramedullary disease, including spleen and liver
Weeks 4, 8, 12, 24, 36, 48, 60, 72, 84, and 96
Cytogenetic response at and by scheduled timepoints
Periodo de tiempo: 5 years

Cytogenetic response is defined as follows:

  • Complete (CCyR) - 0% Ph+ metaphases
  • Partial (PCyR) - >0 to 35% Ph+ metaphases
  • Major* (MCyR) - 0 to 35% Ph+ metaphases
  • Minor (mCyR) - >35 to 65% Ph+ metaphases
  • Minimal - >65 to 95% Ph+ metaphases
  • None - >95 to 100% Ph+ metaphases Bone marrow aspirate for cytogenetic analyses will be performed at screening, and upon unfavorable response to treatment at the physician's discretion.
5 years
Time to response
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

Time to the first response is defined as: date of the first documented occurrence of the response or time from first study drug intake to first response - date of the first study treatment intake + 1.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Duration of response (limited to the binary response endpoints)
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

Duration of response is defined as the time between the date of the first documented occurrence of the response and the earliest date of confirmed loss of the response, progression to accelerated phase/blast crisis (AP/BC) or CML-related death for participants in the respective Responder Set.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Time to treatment failure (TTF)
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

TTF is defined as the time from the first study drug intake to the first/earliest documented date of any of the following events: unfavorable response to treatment, confirmed loss of MMR at any time while on study treatment and discontinuation from study treatment due to any reason.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Event-free survival (EFS)
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

EFS is defined as the time from the first study drug intake to the earliest occurrence of the following events: unfavorable response to treatment, confirmed loss of MMR at any time while on study treatment, discontinuation from the study treatment due to an adverse event (AE) and death due to any cause.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Overall survival (OS)
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

OS is defined as the time from the first study drug intake to the date of death from any cause during the study (including death observed during the survival follow-up period after discontinuation of study treatment).
240 weeks after the last participant enrolled in the study received the first dose of treatment
Growth: Height/length, weight, bone age measured by X-Ray
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

To assess growth and sexual maturation. An X-ray of the left hand and wrist to assess bone age will be performed at screening and every 48 weeks until end of treatment (EOT). This assessment will no longer be required for participants once bone maturity in post-puberty stage is confirmed by the last X-ray.
240 weeks after the last participant enrolled in the study received the first dose of treatment
Sexual maturation: Height/length, weight, bone age measured by Tanner staging
Periodo de tiempo: 240 weeks after the last participant enrolled in the study received the first dose of treatment

To assess growth and sexual maturation. Tanner staging will be based on external genitalia, breast and pubic hair examination. Tanner staging will be assessed for all study participants at screening. Participants who do not have a baseline Tanner Stage of 5 will continue to be assessed every 24 weeks until achievement of Tanner Stage 5 on both secondary sexual characteristics or EOT, whichever comes first. Once a participant reaches stage 5, examinations do not need to be conducted anymore. For all male participants, Tanner Stage 5 will be achieved once the participant has demonstrated both stage 5 genitalia development and stage 5 pubic hair development. For all female participants, Tanner Stage 5 will be achieved once the participant has demonstrated both stage 5 breast development and stage 5 pubic hair development.
240 weeks after the last participant enrolled in the study received the first dose of treatment
PK parameters of asciminib: AUClast
Periodo de tiempo: Week 2, Day 1 at 1, 3 and 6 hours post dose

AUClast: The AUC from time zero to the last measurable concentration sampling time.
Week 2, Day 1 at 1, 3 and 6 hours post dose
PK parameters of asciminib: AUCtau
Periodo de tiempo: Week 2, Day 1: pre dose (0 hour), Week 2: Day 1 at 1, 2, 3, and 6 hours post-dose

AUCtau: The AUC calculated to the end of a dosing interval (tau) at steady-state.
Week 2, Day 1: pre dose (0 hour), Week 2: Day 1 at 1, 2, 3, and 6 hours post-dose
PK parameter of asciminib: Cmax
Periodo de tiempo: Week 2, Day 1 at 1, 3, and 6 hours post dose

Cmax is the maximum (peak) observed plasma drug concentration after single dose administration.
Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Tmax
Periodo de tiempo: Week 2, Day 1 at 1, 3, and 6 hours post dose

Tmax is the time to reach maximum (peak) plasma drug concentration (h) after administration.
Week 2, Day 1 at 1, 3, and 6 hours post dose
PK parameter of asciminib: Ctrough
Periodo de tiempo: Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)

Ctrough refers to the lowest concentration a drug reaches in the bloodstream immediately before the next dose is administered.
Week 2, Day 1: pre-dose (0 hr), Week 24: pre-dose (0 hr), Week 48: Pre-dose (0 hr)

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Novartis Pharmaceuticals

Investigadores

  • Director de estudio: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Actual)

28 de abril de 2026

Finalización primaria (Estimado)

23 de febrero de 2033

Finalización del estudio (Estimado)

23 de febrero de 2033

Fechas de registro del estudio

Enviado por primera vez

15 de enero de 2026

Primero enviado que cumplió con los criterios de control de calidad

16 de enero de 2026

Publicado por primera vez (Actual)

21 de enero de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

10 de noviembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

9 de noviembre de 2026

Última verificación

1 de noviembre de 2026

Más información

Términos relacionados con este estudio

Palabras clave

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • CABL001I12202
  • 2025-522138-29 (Identificador de registro: EU CTIS)
  • 2025 (Subvención/contrato del NIH de EE. UU.)

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

SÍ

Descripción del plan IPD

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data is currently available according to the process described on www.clinicalstudydatarequest.com.

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

Sí

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .

Suscribir