A Phase 1/2 interventional study of Asciminib Pediatric formulation group and Asciminib Adult formulation group in Myeloid Leukemia, Philadelphia Positive, sponsored by Novartis Pharmaceuticals. Active, not recruiting at 23 sites in 12 countries. Open to participants aged 1 Year to 17 Years. Per ClinicalTrials.gov, last updated 2026-11-09.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 1/2, Interventional, and Treatment
Among 21 studies testing asciminib in Leukemia, Myelogenous, Chronic, BCR-ABL Positive — 8 open to participants now, 13 under way, 2 of them phase 3. asciminib · Leukemia, Myelogenous, Chronic, BCR-ABL Positive.
Among the 21 studies testing asciminib in Leukemia, Myelogenous, Chronic, BCR-ABL Positive, this one is the 17th largest by enrollment, is one of 5 that started in 2021, is the only one in phase 1/2, reaches primary completion later than 4 of the 12 other live ones, and was the 10th registered.
894 studies on the registry are indexed under Leukemia, Myelogenous, Chronic, BCR-ABL Positive; 109 are open to participants now.
Browse Leukemia, Myelogenous, Chronic, BCR-ABL Positive studies →8 of the 21 studies testing asciminib in Leukemia, Myelogenous, Chronic, BCR-ABL Positive are open now, in 12 countries. Recruiting first, the newest first:
Thomas Ernst, Prof. Dr. med. · Sites in Germany
Ages 18+
Novartis Pharmaceuticals · Sites in United States, Australia, Brazil and 14 more countries
Ages 1–18
Gruppo Italiano Malattie EMatologiche dell'Adulto · Sites in Spain
Ages 18+
Novartis Pharmaceuticals · Sites in France
Ages 18–99
M.D. Anderson Cancer Center · Sites in United States
Ages 18+
By country, each count listing its studies:
asciminib: first FDA approval 2021 (SCEMBLIX), first EMA approval 2022. What it is approved for, and how studies give it, is on its page →
Of the 79 Leukemia, Myelogenous, Chronic, BCR-ABL Positive studies recruiting now, 48 (61%) set no upper age limit and 20 (25%) stop at 75 or younger; 20 take children under 18; 7 take healthy volunteers.
This study: 1 Year to 17 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
21 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 3 · Phase 2 12 · Phase 3 5 · Phase 4 2
First in 2014; this study’s year is marked. 5 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 46 is close to the median of 44 across 681 interventional studies indexed under Leukemia, Myelogenous, Chronic, BCR-ABL Positive.
Median enrollment 100 across the 21 interventional studies. Of the 8 that completed or were terminated, 4 (50%) have results posted.
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12 of 21 have an industry lead sponsor. The 9 most frequent:
This study is registered under Novartis Pharmaceuticals, one of the names counted as Novartis. Under that name alone:
Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
92 studies in Leukemia, Myelogenous, Chronic, BCR-ABL Positive by Novartis: 9 open now, 13 under way, 2 of them phase 3. Of 73 finished, 35 (48%) have results posted.
Among the 92 studies in Leukemia, Myelogenous, Chronic, BCR-ABL Positive by Novartis, this one is the 39th largest by enrollment of the 63 interventional ones that give a count, is one of 8 that started in 2021, is one of 6 in phase 1/2, reaches primary completion later than 5 of the 12 other live ones, and was the 67th registered.
Novartis’s Leukemia, Myelogenous, Chronic, BCR-ABL Positive studies by the year they started, since 1999.
2 of the studies testing asciminib in Leukemia, Myelogenous, Chronic, BCR-ABL Positive that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT03906292University of JenaFrontline Asciminib Combination in Chronic Phase CML | 2027-03 |
| NCT04925479Novartis PharmaceuticalsStudy to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia | 2027-09-03 |
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Among the 21 studies testing asciminib in Leukemia, Myelogenous, Chronic, BCR-ABL Positive, from their registry updates:
Among Novartis’s 92 in Leukemia, Myelogenous, Chronic, BCR-ABL Positive:
This tab as a report to print or save as PDF → With an account.
The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML in the pediatric population has the same pathogenesis, similar clinical characteristics and progression pattern as in adults.
The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (PH+ CML-CP) previously treated with one or more Tyrosine kinase inhibitor (TKIs).
The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients with the goal of identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
The pediatric formulation group will include at least 15 participants in each of the following two age categories: 1 to \<12 years and 12 to \<18 years; leading to at least 30 participants enrolled treated with the pediatric formulation. It will consist of a dose determination part (Part 1) and a cohort expansion (Part 2 BID regimen and Part 3 QD regimen).
In Part 1, 4-6 participants will be enrolled in order to obtain at least 4 participants evaluable for PK (these participants may be from either of the age categories described above). The initial starting dose will be based on body weight and will be administered BID with food.
Once the body weight adjusted dose has been determined in Part 1 of the study, the patients will be enrolled in Part 2 until at least 20 participants, including those who were included in Part 1, have been enrolled (10 per age group) in the pediatric formulation group. Once the interim safety and PK analysis 2 is completed for one of the age groups, the Part 3 QD regimen will open for the respective age group to enroll 10 patients (5 patients by age group).
Due to the fact that the pediatric formulation was in development and was not available, this study started with the recruitment of adolescent patients. These participants aged 14 to \<18 years, weighing at least 40 kg receive the adult formulation at a flat dose of 40 mg BID under fasted conditions.
The total duration of the treatment period of the study will be 5 years (260 weeks). Participants who, according to Investigator's judgement, are benefiting from study treatment will remain on treatment up to the completion of the treatment period (Week 260/5 years).
The primary analysis for the BID regimen is planned after all participants in Part 1 and 2 have completed at least 52 weeks of study treatment or discontinued earlier.
The primary analysis for combined regimen (BID+QD) is planned after all participants in Part 1, 2 and 3 have completed at least 52 weeks of study treatment or discontinued earlier.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Male or female participants:
Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
This arm consists of 2 groups: * The pediatric formulation group where the dose is based on body weight (1.3mg/kg BID or 2.6 mg/kg QD) * The adult formulation group where participants will receive a flat dose of 40mg BID
Drug: Asciminib Pediatric formulation group · Drug: Asciminib Adult formulation group
Asciminib Pediatric formulation group: 1 mg film-coated granules in a size 0 capsule will be supplied, taken orally (capsules are a container for the granules and are not ingested): 10 mg (10x 1 mg film-coated granules in capsule) 15 mg (15x 1 mg film-coated granules in capsule) 30 mg (30x 1 mg film-coated granules in capsule)
Also known as: ABL001
Asciminib Adult formulation group: 40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally.
Also known as: ABL001
Primary Pharmacokinetic (PK) parameter: AUClast
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Time frame: 52 weeks
Primary PK parameter: AUCtau
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Time frame: 52 weeks
Secondary PK parameter: Cmax
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Time frame: 52 weeks
Secondary PK parameter: Tmax
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Time frame: 52 weeks
Secondary PK parameter: Ctrough
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Time frame: 52 weeks
Hematologic responses
Complete hematological response will be defined as all of the following present for ≥ 4 weeks: * 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
Time frame: 52 weeks
Molecular responses
To assess pharmacodynamic markers of asciminib's anti-leukemic activity. Molecular response will be assessed by Breakpoint Cluster Region gene-Abelson proto-oncogene (BCR-ABL) 1 level.
Time frame: 52 weeks
Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks
To assess acceptability and palatability of the pediatric formulation
Time frame: after first dose at Week 1 Day 1, 4 weeks, 52 weeks
Plan to share: Yes — 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.
Data sharing details ↗ · www.clinicalstudydatarequest.com
No publications or documents are linked to this record.
Started Dec 2021 (actual). Primary completion is estimated for Sep 2027, 68 months after the start.
Of 26 phase 1/2 interventional studies under Leukemia, Myelogenous, Chronic, BCR-ABL Positive, half reached primary completion within 69 months of starting (the median), counting those still running; a quarter within 56 and three quarters within 94. This study's planned 68 months is close to that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Nov 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Asciminib
Leukemia, Myelogenous, Chronic, BCR-ABL Positive →