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Studie för att fastställa effektiviteten och säkerheten hos Asciminib hos pediatriska patienter med Ph+ CML-CP

9 november 2026 uppdaterad av: Novartis Pharmaceuticals

En fas II, multicenter, öppen, enarmsstudie för att utvärdera säkerheten och effekten av asciminib hos barn- och ungdomsdeltagare som nyligen diagnostiserats eller tidigare behandlats för Philadelphia-positiv kronisk myelogen leukemi i kronisk fas (Ph+ CML-CP) med eller utan känd T315I-mutation

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.

Studieöversikt

Status

Rekrytering

Betingelser

Intervention / Behandling

Detaljerad beskrivning

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.

Studietyp

Interventionell

Inskrivning (Beräknad)

50

Fas

  • Fas 2

Utökad åtkomst

Tillgängligt utanför den kliniska prövningen. Se utökad åtkomstpost.

Kontakter och platser

Det här avsnittet innehåller kontaktuppgifter för dem som genomför studien och information om var denna studie genomförs.

Studiekontakt

Studera Kontakt Backup

  • Namn: Novartis Pharmaceuticals
  • Telefonnummer: +41613241111

Studieorter

  • Australien
    • Queensland
      • Brisbane, Queensland, Australien, 4101
        • Rekrytering
        • Novartis Investigative Site
    • South Australia
      • North Adelaide, South Australia, Australien, 5066
        • Rekrytering
        • Novartis Investigative Site
  • Brasilien
    • Rio de Janeiro
      • Rio de Janeiro, Rio de Janeiro, Brasilien, 20211-030
        • Rekrytering
        • Novartis Investigative Site
    • São Paulo
      • Barretos, São Paulo, Brasilien, 14784-400
        • Rekrytering
        • Novartis Investigative Site
      • São Paulo, São Paulo, Brasilien, 04829-310
        • Rekrytering
        • Novartis Investigative Site
  • Frankrike
      • Marseille, Frankrike, 13885
        • Rekrytering
        • Novartis Investigative Site
      • Paris, Frankrike, 75019
        • Rekrytering
        • Novartis Investigative Site
      • Poitiers, Frankrike, 86021
        • Rekrytering
        • Novartis Investigative Site
      • Strasbourg, Frankrike, 67000
        • Rekrytering
        • Novartis Investigative Site
  • Förenta staterna
    • Illinois
      • Chicago, Illinois, Förenta staterna, 60611
        • Rekrytering
        • Ann and Robert H Lurie Childs Hosp
        • Huvudutredare:
          • Sara Zarnegar-Lumley
        • Kontakt:
    • New Jersey
      • New Brunswick, New Jersey, Förenta staterna, 08901
        • Rekrytering
        • Rutgers Cancer Institute of New Jersey
        • Huvudutredare:
          • Richard DRACHTMAN
        • Kontakt:
    • New York
      • New York, New York, Förenta staterna, 10032
        • Rekrytering
        • Columbia University Medical Center New York Presbyterian
        • Huvudutredare:
          • Nobuko Hijiya
        • Kontakt:
    • Ohio
      • Cincinnati, Ohio, Förenta staterna, 45229-3039
        • Rekrytering
        • Cinn Children Hosp Medical Center
        • Huvudutredare:
          • Benjamin Mizukawa
        • Kontakt:
    • Washington
      • Seattle, Washington, Förenta staterna, 98105
  • Italien
    • PD
      • Padova, PD, Italien, 35128
        • Rekrytering
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italien, 00165
        • Rekrytering
        • Novartis Investigative Site
  • Japan
      • Fukuoka, Japan, 8128582
        • Rekrytering
        • Novartis Investigative Site
      • Saitama, Japan, 3308777
        • Rekrytering
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama, Kanagawa, Japan, 2328555
        • Rekrytering
        • Novartis Investigative Site
  • Kanada
    • Alberta
      • Edmonton, Alberta, Kanada, T6G 1C9
        • Rekrytering
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, Kanada, H3T 1C5
        • Rekrytering
        • Novartis Investigative Site
  • Kina
      • Tianjin, Kina, 300020
        • Rekrytering
        • Novartis Investigative Site
    • Henan
      • Zhengzhou, Henan, Kina, 450003
        • Rekrytering
        • Novartis Investigative Site
  • Nederländerna
      • Utrecht, Nederländerna, 3584 CS
        • Rekrytering
        • Novartis Investigative Site
  • Polen
      • Gdansk, Polen, 80-214
        • Rekrytering
        • Novartis Investigative Site
      • Wroclaw, Polen, 50-556
        • Rekrytering
        • Novartis Investigative Site
  • Singapore
      • Singapore, Singapore, 229899
        • Rekrytering
        • Novartis Investigative Site
  • Spanien
      • Barcelona, Spanien, 08035
        • Rekrytering
        • Novartis Investigative Site
      • Madrid, Spanien, 28046
        • Rekrytering
        • Novartis Investigative Site
      • Madrid, Spanien, 28009
        • Rekrytering
        • Novartis Investigative Site
  • Sydkorea
      • Seoul, Sydkorea, 03080
        • Rekrytering
        • Novartis Investigative Site
      • Seoul, Sydkorea, 06591
        • Rekrytering
        • Novartis Investigative Site
  • Thailand
      • Bangkok, Thailand, 10330
        • Rekrytering
        • Novartis Investigative Site
      • Bangkok, Thailand, 10700
        • Rekrytering
        • Novartis Investigative Site
  • Tyskland
      • Erlangen, Tyskland, 91054
        • Rekrytering
        • Novartis Investigative Site
      • Hamburg, Tyskland, 20246
        • Rekrytering
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main, Hesse, Tyskland, 60590
        • Rekrytering
        • Novartis Investigative Site
  • Ungern
    • Hajdu Bihar Megye
      • Debrecen, Hajdu Bihar Megye, Ungern, 4032
        • Rekrytering
        • Novartis Investigative Site
  • Vietnam
      • Hanoi, Vietnam, 100000
        • Rekrytering
        • Novartis Investigative Site

Deltagandekriterier

Forskare letar efter personer som passar en viss beskrivning, så kallade behörighetskriterier. Några exempel på dessa kriterier är en persons allmänna hälsotillstånd eller tidigare behandlingar.

Urvalskriterier

Åldrar som är berättigade till studier

1 år till 18 år (Barn, Vuxen)

Tar emot friska volontärer

Nej

Kön som är behöriga för studier

Allt

Beskrivning

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.

Studieplan

Det här avsnittet ger detaljer om studieplanen, inklusive hur studien är utformad och vad studien mäter.

Hur är studien utformad?

Designdetaljer

  • Primärt syfte: Behandling
  • Tilldelning: N/A
  • Interventionsmodell: Enskild gruppuppgift
  • Maskning: Ingen (Open Label)

Vapen och interventioner

Deltagargrupp / Arm
Intervention / Behandling
Experimentell: 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
Läkemedel: 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)

Andra namn:
  • ABL001

Vad mäter studien?

Primära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
MMR at Week 48
Tidsram: 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

Sekundära resultatmått

Resultatmått
Åtgärdsbeskrivning
Tidsram
Tid till sjukdomsframsteg
Tidsram: 5 år
5 år
MMR at Week 96
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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)
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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
Tidsram: 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)

Samarbetspartners och utredare

Det är här du hittar personer och organisationer som är involverade i denna studie.

Sponsor

Novartis Pharmaceuticals

Utredare

  • Studierektor: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Studieavstämningsdatum

Dessa datum spårar framstegen för inlämningar av studieposter och sammanfattande resultat till ClinicalTrials.gov. Studieposter och rapporterade resultat granskas av National Library of Medicine (NLM) för att säkerställa att de uppfyller specifika kvalitetskontrollstandarder innan de publiceras på den offentliga webbplatsen.

Studera stora datum

Studiestart (Faktisk)

28 april 2026

Primärt slutförande (Beräknad)

23 februari 2033

Avslutad studie (Beräknad)

23 februari 2033

Studieregistreringsdatum

Först inskickad

15 januari 2026

Först inskickad som uppfyllde QC-kriterierna

16 januari 2026

Första postat (Faktisk)

21 januari 2026

Uppdateringar av studier

Senaste uppdatering publicerad (Faktisk)

10 november 2026

Senaste inskickade uppdateringen som uppfyllde QC-kriterierna

9 november 2026

Senast verifierad

1 november 2026

Mer information

Termer relaterade till denna studie

Nyckelord

Ytterligare relevanta MeSH-villkor

Andra studie-ID-nummer

  • CABL001I12202
  • 2025-522138-29 (Registeridentifierare: EU CTIS)
  • 2025 (U.S.S. NIH-anslag/kontrakt)

Plan för individuella deltagardata (IPD)

Planerar du att dela individuella deltagardata (IPD)?

JA

IPD-planbeskrivning

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.

Läkemedels- och apparatinformation, studiedokument

Studerar en amerikansk FDA-reglerad läkemedelsprodukt

Ja

Studerar en amerikansk FDA-reglerad produktprodukt

Nej

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