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Ph+ CML-CP 소아 환자에서 Asciminib의 효능과 안전성을 평가하는 연구

2026년 11월 9일 업데이트: Novartis Pharmaceuticals

신규 진단 또는 기존 치료 경험이 있는 필라델피아 양성 만성기 만성골수성백혈병(Ph+ CML-CP) 소아 환자에서 Asciminib의 안전성과 유효성을 평가하기 위한 제2상, 다기관, 개방형, 단일군 연구(T315I 돌연변이 유무에 관계없음)

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.

연구 개요

상태

모병

정황

개입 / 치료

상세 설명

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.

연구 유형

중재적

등록 (추정된)

50

단계

  • 2 단계

확장된 액세스

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연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 연락처

연구 연락처 백업

  • 이름: Novartis Pharmaceuticals
  • 전화번호: +41613241111

연구 장소

  • 네덜란드
      • Utrecht, 네덜란드, 3584 CS
        • 모병
        • Novartis Investigative Site
  • 대한민국
      • Seoul, 대한민국, 03080
        • 모병
        • Novartis Investigative Site
      • Seoul, 대한민국, 06591
        • 모병
        • Novartis Investigative Site
  • 독일
      • Erlangen, 독일, 91054
        • 모병
        • Novartis Investigative Site
      • Hamburg, 독일, 20246
        • 모병
        • Novartis Investigative Site
    • Hesse
      • Frankfurt am Main, Hesse, 독일, 60590
        • 모병
        • Novartis Investigative Site
  • 미국
    • Illinois
      • Chicago, Illinois, 미국, 60611
        • 모병
        • Ann and Robert H Lurie Childs Hosp
        • 수석 연구원:
          • Sara Zarnegar-Lumley
        • 연락하다:
    • New Jersey
      • New Brunswick, New Jersey, 미국, 08901
        • 모병
        • Rutgers Cancer Institute of New Jersey
        • 수석 연구원:
          • Richard DRACHTMAN
        • 연락하다:
    • New York
      • New York, New York, 미국, 10032
        • 모병
        • Columbia University Medical Center New York Presbyterian
        • 수석 연구원:
          • Nobuko Hijiya
        • 연락하다:
    • Ohio
      • Cincinnati, Ohio, 미국, 45229-3039
        • 모병
        • Cinn Children Hosp Medical Center
        • 수석 연구원:
          • Benjamin Mizukawa
        • 연락하다:
    • Washington
      • Seattle, Washington, 미국, 98105
  • 베트남
      • Hanoi, 베트남, 100000
        • 모병
        • Novartis Investigative Site
  • 브라질
    • Rio de Janeiro
      • Rio de Janeiro, Rio de Janeiro, 브라질, 20211-030
        • 모병
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    • São Paulo
      • Barretos, São Paulo, 브라질, 14784-400
        • 모병
        • Novartis Investigative Site
      • São Paulo, São Paulo, 브라질, 04829-310
        • 모병
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  • 스페인
      • Barcelona, 스페인, 08035
        • 모병
        • Novartis Investigative Site
      • Madrid, 스페인, 28046
        • 모병
        • Novartis Investigative Site
      • Madrid, 스페인, 28009
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        • Novartis Investigative Site
  • 싱가포르
      • Singapore, 싱가포르, 229899
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        • Novartis Investigative Site
  • 이탈리아
    • PD
      • Padova, PD, 이탈리아, 35128
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        • Novartis Investigative Site
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      • Roma, RM, 이탈리아, 00165
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        • Novartis Investigative Site
  • 일본
      • Fukuoka, 일본, 8128582
        • 모병
        • Novartis Investigative Site
      • Saitama, 일본, 3308777
        • 모병
        • Novartis Investigative Site
    • Kanagawa
      • Yokohama, Kanagawa, 일본, 2328555
        • 모병
        • Novartis Investigative Site
  • 중국
      • Tianjin, 중국, 300020
        • 모병
        • Novartis Investigative Site
    • Henan
      • Zhengzhou, Henan, 중국, 450003
        • 모병
        • Novartis Investigative Site
  • 캐나다
    • Alberta
      • Edmonton, Alberta, 캐나다, T6G 1C9
        • 모병
        • Novartis Investigative Site
    • Quebec
      • Montreal, Quebec, 캐나다, H3T 1C5
        • 모병
        • Novartis Investigative Site
  • 태국
      • Bangkok, 태국, 10330
        • 모병
        • Novartis Investigative Site
      • Bangkok, 태국, 10700
        • 모병
        • Novartis Investigative Site
  • 폴란드
      • Gdansk, 폴란드, 80-214
        • 모병
        • Novartis Investigative Site
      • Wroclaw, 폴란드, 50-556
        • 모병
        • Novartis Investigative Site
  • 프랑스
      • Marseille, 프랑스, 13885
        • 모병
        • Novartis Investigative Site
      • Paris, 프랑스, 75019
        • 모병
        • Novartis Investigative Site
      • Poitiers, 프랑스, 86021
        • 모병
        • Novartis Investigative Site
      • Strasbourg, 프랑스, 67000
        • 모병
        • Novartis Investigative Site
  • 헝가리
    • Hajdu Bihar Megye
      • Debrecen, Hajdu Bihar Megye, 헝가리, 4032
        • 모병
        • Novartis Investigative Site
  • 호주
    • Queensland
      • Brisbane, Queensland, 호주, 4101
        • 모병
        • Novartis Investigative Site
    • South Australia
      • North Adelaide, South Australia, 호주, 5066
        • 모병
        • Novartis Investigative Site

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

1년 (어린이, 성인)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

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.

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 해당 없음
  • 중재 모델: 단일 그룹 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: 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
의약품: 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)

다른 이름들:
  • ABL001

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
MMR at Week 48
기간: 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

2차 결과 측정

결과 측정
측정값 설명
기간
질병 진행까지의 시간
기간: 5년
5년
MMR at Week 96
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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)
기간: 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)
기간: 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)
기간: 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)
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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
기간: 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)

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Novartis Pharmaceuticals

수사관

  • 연구 책임자: Novartis Pharmaceuticals, Novartis Pharmaceuticals

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2026년 4월 28일

기본 완료 (추정된)

2033년 2월 23일

연구 완료 (추정된)

2033년 2월 23일

연구 등록 날짜

최초 제출

2026년 1월 15일

QC 기준을 충족하는 최초 제출

2026년 1월 16일

처음 게시됨 (실제)

2026년 1월 21일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 11월 10일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 11월 9일

마지막으로 확인됨

2026년 11월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • CABL001I12202
  • 2025-522138-29 (레지스트리 식별자: EU CTIS)
  • 2025 (미국 NIH 보조금/계약)

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

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.

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

구독하다