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Lo studio clinico NCT07354074 per Leucemia Mieloide Cronica, Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive è non ancora in arruolamento. Consulti la vista a schede del Radar degli Studi Clinici e gli strumenti di scoperta IA per tutti i dettagli. Oppure, ponga pure una domanda qui. | ||
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NovartisStudy to Determine the Efficacy and Safety of Asciminib in Pediatric Patients With Ph+ CML-CP Fase II 50 Pediatrico
I dettagli dello studio clinico sono disponibili principalmente in inglese. Tuttavia, Trial Radar IA può essere d'aiuto! Basta cliccare su 'Spiega lo studio' per visualizzare e discutere le informazioni sullo studio nella lingua selezionata.
La sperimentazione clinica NCT07354074 è uno studio interventistico di Fase II volto a esaminare il trattamento per Leucemia Mieloide Cronica, Leukemia, Myelogenous, Chronic, Philadelphia Chromosome Positive, attualmente non ancora in arruolamento. L'arruolamento dovrebbe iniziare il 4 maggio 2026, con l'obiettivo di raggiungere 50 partecipanti. Sotto la guida di Novartis, dovrebbe concludersi entro il 23 febbraio 2033. I dati più recenti da ClinicalTrials.gov sono stati aggiornati l'ultima volta il 23 marzo 2026.
Sommario breve
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.
Descrizione dettagliata
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 resista...
Titolo ufficiale
A Phase II, Multicenter, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Asciminib in Pediatric Participants Newly Diagnosed or Previously Treated With Philadelphia Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP) With or Without Known T315I Mutation
Patologie
Leucemia Mieloide CronicaLeukemia, Myelogenous, Chronic, Philadelphia Chromosome PositiveAltri ID dello studio
- CABL001I12202
- 2025-522138-29 (Identificativo del registro) (EU CTIS)
Numero NCT
Data di inizio (effettiva)
2026-05-04
Ultimo aggiornamento pubblicato
2026-03-23
Data di completamento (stimata)
2033-02-23
Arruolamento (previsto)
50
Tipo di studio
Interventistico
FASE
Fase II
Stato
Non ancora in arruolamento
Parole chiave
Asciminib
ABL001
Pediatric participants
Philadelphia chromosome positive chronic myeloid leukemia in chronic phase
Ph+ CML-CP
tyrosine kinase inhibitor
TKI
Molecular Response
MR
CML
Chronic phase
T3151
Ph+
ABL001
Pediatric participants
Philadelphia chromosome positive chronic myeloid leukemia in chronic phase
Ph+ CML-CP
tyrosine kinase inhibitor
TKI
Molecular Response
MR
CML
Chronic phase
T3151
Ph+
Scopo principale
Trattamento
Allocazione
N.D.
Modello di intervento
A gruppo singolo
Mascheramento
Nessuno (studio in aperto)
Bracci / Interventi
| Gruppo/Braccio di partecipanti | Intervento/Trattamento |
|---|---|
SperimentaleSingle 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 | 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) |
Esito primario
Esito secondario
| Misure di esito | Descrizione della misura | Arco temporale |
|---|---|---|
MMR at Week 48 | 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 |
| Misure di esito | Descrizione della misura | Arco temporale |
|---|---|---|
MMR at Week 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. | 96 weeks |
MMR at and by scheduled timepoints | 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 | 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 | 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 | 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) | 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) | 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 |
Time to disease progression | 5 years | |
Event-free survival (EFS) | 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) | 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 | 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 | 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 | 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 | 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 | 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 | 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 | 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) |
Assistente alla partecipazione
Criteri di eleggibilità
Età idonea
Bambino, Adulto
Età minima
1 Year
Sessi idonei
Tutti
Participants eligible for inclusion in this study must meet all of the following criteria:
Signed informed consent must be obtained prior to participation in the study.
Male or female participants 1 and < 18 years of age at study enrollment
Diagnosis of CML-CP (Apperley et al 2025) with cytogenetic confirmation of Philadelphia positive (Ph+) chromosome
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:
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
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.
- Known second chronic phase (CP) of CML after previous progression to Accelerated Phase (AP)/Blast Phase (BP).
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planned to undergo allogeneic hematopoietic stem cell transplantation
- 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.
NovartisContatti principali dello studio
Contatto: Novartis Pharmaceuticals, 1-888-669-6682, [email protected]
Contatto: Novartis Pharmaceuticals, +41613241111
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