Myeloid Leukemia, Philadelphia PositivePhase 1/2NCT04925479
Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia
A Phase 1/2 study testing Asciminib Pediatric formulation group and Asciminib Adult formulation group in people with Myeloid Leukemia, Philadelphia Positive, run by Novartis Pharmaceuticals at 39 sites in 14 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Myeloid Leukemia, Philadelphia Positive
- What is being tested?
- Asciminib Pediatric formulation group and Asciminib Adult formulation group
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 39 sitesin 14 countries
- Study dates
- 27 Dec 2021to 1 Oct 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female participants:
- Pediatric formulation group: ≥ 1 and less than 18 years of age at study entry.
- Adult formulation group: ≥ 14 and less than 18 years of age and body weight of ≥ 40 kg at study entry.
- 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.
- < 15% blasts in peripheral blood and bone marrow
- < 30% combined blasts plus promyelocytes in peripheral blood and bone marrow
Show all 15Show fewer
- < 20% basophils in the peripheral blood
- Neutrophils ≥ 1.5 x 10^9/L (or WBC ≥ 3 x 10^9/L if neutrophils are not available) and platelet count ≥ 100 x 10^9/L
- No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly
- Prior treatment with a minimum of one TKI
- Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening.
- Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients < 16 years of age at the time of screening
- Participants must have adequate renal, hepatic, pancreatic and cardiac function
- Participants must have electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication:
- Evidence of typical BCR::ABL1 transcript [e14a2 and/or e13a2] at the time of screening which are amenable to standardized RQ-PCR quantification.
Who cannot join
- Known presence of the T315I mutation prior to study entry or a BCR::ABL mutation with known resistance to study treatment any time prior to study entry.
- Known second chronic phase of CML after previous progression to AP/BC.
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planning to undergo allogeneic hematopoietic stem cell transplantation.
- Cardiac or cardiac repolarization abnormality
- Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol
Show all 11Show fewer
- History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis.
- History of acute or chronic liver disease.
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug
- Pregnant or nursing (lactating) females.
- Other protocol-defined inclusion/exclusion may apply.
Trial locations and contacts
39 sites in 14 countries. Contact the study team or a site near you to ask about taking part.
Novartis Pharmaceuticals contact
39 sites in 14 countries
United States8 sites, 1 recruiting
- Active, not recruiting
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
- Withdrawn
Cinn Children Hosp Medical Center
Cincinnati, Ohio, 45229-3039
- Withdrawn
Uni Of Texas MD Anderson Cancer Ctr
Houston, Texas, 77024
- Active, not recruiting
Indiana UH Riley H for CIU
Indianapolis, Indiana, 46202-5167
- Recruiting
University of Mississippi Medical Center
Jackson, Mississippi, 39216-4505
Rachel Lowery, 601-984-5220, rlowery@umc.edu
Dereck B Davis
- Active, not recruiting
Columbia University Medical Center New York Presbyterian
New York, New York, 10032
- Withdrawn
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104-4399
- Withdrawn
University Of Utah
Salt Lake City, Utah, 84132
China4 sites
- Active, not recruiting
Novartis Investigative Site
Beijing, 100044
- Withdrawn
Novartis Investigative Site
Hangzhou, Zhejiang, 310052
- Withdrawn
Novartis Investigative Site
Shanghai, 200127
- Withdrawn
Novartis Investigative Site
Tianjin, 300020
France4 sites
- Withdrawn
Novartis Investigative Site
Bordeaux, 33076
- Active, not recruiting
Novartis Investigative Site
Lille, 59000
- Active, not recruiting
Novartis Investigative Site
Paris, 75019
- Withdrawn
Novartis Investigative Site
Poitiers, 86021
Italy4 sites
- Active, not recruiting
Novartis Investigative Site
Genova, GE, 16147
- Withdrawn
Novartis Investigative Site
Monza, MB, 20900
- Active, not recruiting
Novartis Investigative Site
Roma, RM, 00165
- Withdrawn
Novartis Investigative Site
Torino, TO, 10126
Germany3 sites
- Active, not recruiting
Novartis Investigative Site
Erlangen, 91054
- Active, not recruiting
Novartis Investigative Site
Essen, 45147
- Active, not recruiting
Novartis Investigative Site
Hamburg, 20246
Japan3 sites
- Active, not recruiting
Novartis Investigative Site
Osaka, 5340021
- Active, not recruiting
Novartis Investigative Site
Shinjuku-ku, Tokyo, 1608582
- Active, not recruiting
Novartis Investigative Site
Yokohama, Kanagawa, 2328555
Thailand3 sites
- Withdrawn
Novartis Investigative Site
Bangkok, 10400
- Active, not recruiting
Novartis Investigative Site
Chiang Mai, 50200
- Active, not recruiting
Novartis Investigative Site
Khon Kaen, THA, 40002
Russia2 sites
- Withdrawn
Novartis Investigative Site
Moscow, 117198
- Withdrawn
Novartis Investigative Site
Saint Petersburg, 197022
South Korea2 sites
- Active, not recruiting
Novartis Investigative Site
Seoul, 03080
- Active, not recruiting
Novartis Investigative Site
Seoul, 05505
Turkey (Türkiye)2 sites
- Active, not recruiting
Novartis Investigative Site
Bursa, Nilufer, 16059
- Active, not recruiting
Novartis Investigative Site
Istanbul, Fatih, 34093
Netherlands1 site
- Active, not recruiting
Novartis Investigative Site
Utrecht, 3584 CS
About this study
Official title: A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors
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.
Read the detailed description
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.
- Conditions
- Myeloid Leukemia, Philadelphia Positive
- Being tested
- Asciminib Pediatric formulation group, Asciminib Adult formulation group
- Keywords
- Asciminib, pediatric patients, Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, Ph+ CML-CP, ABL001
- Medical subject headings
- Leukemia, Myelogenous, Chronic, BCR-ABL Positive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Asciminib Pediatric formulation groupDrug
Also called ABL001
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)
Asciminib Adult formulation groupDrug
Also called ABL001
Asciminib Adult formulation group:
40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally.
| Study group | Role | What they receive |
|---|---|---|
| Asciminib | Experimental | 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 Receives: Drug: Asciminib Pediatric formulation group; Drug: Asciminib Adult formulation group |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 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).
Measured: 52 weeks
Secondary outcomes (3)
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
Measured: 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.
Measured: 52 weeks
Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks
To assess acceptability and palatability of the pediatric formulation
Measured: after first dose at Week 1 Day 1, 4 weeks, 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2021
- Study start27 Dec 2021
- Primary completion2 Sep 2027 (estimated)
- Study completion1 Oct 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myeloid Leukemia, Philadelphia PositiveStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 34 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04925479
- Sponsor's protocol ID
- CABL001I12201
- Other IDs
- 2023- 508129-28, 2023
- Status last verified
- Sep 2026
- First submitted
- 18 May 2021
- Last updated
- 25 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- 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.
- Expanded access
- Available
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors
- ClinicalTrials.gov ID
- NCT04925479
- Acronym / sponsor ID
- CABL001I12201
- Conditions
- Myeloid Leukemia, Philadelphia Positive
- Keywords
- Asciminib, pediatric patients, Philadelphia chromosome positive chronic myeloid leukemia in chronic phase, Ph+ CML-CP, ABL001
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 34 participants (estimated)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 27 Dec 2021 (actual)
- Primary completion
- 2 Sep 2027 (estimated)
- Study completion
- 1 Oct 2031 (estimated)
- First posted
- 14 Jun 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Asciminib | Experimental | Drug: Asciminib Pediatric formulation group, Drug: Asciminib Adult formulation groupThis 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 |
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (15 listed)
- 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.
- Prior treatment with a minimum of one TKI
- Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening.
- Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients < 16 years of age at the time of screening
Key exclusion criteria (11 listed)
- Known presence of the T315I mutation prior to study entry or a BCR::ABL mutation with known resistance to study treatment any time prior to study entry.
- Known second chronic phase of CML after previous progression to AP/BC.
- Previous treatment with a hematopoietic stem-cell transplantation.
- Patient planning to undergo allogeneic hematopoietic stem cell transplantation.
- Cardiac or cardiac repolarization abnormality
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Asciminib Pediatric formulation groupAlso: ABL001Asciminib 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) | Drug | Asciminib |
| Asciminib Adult formulation groupAlso: ABL001Asciminib Adult formulation group: 40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally. | Drug | Asciminib |
Primary outcome
Primary Pharmacokinetic (PK) parameter: AUClast
Time frame: 52 weeks
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Primary PK parameter: AUCtau
Time frame: 52 weeks
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Secondary PK parameter: Cmax
Time frame: 52 weeks
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Secondary PK parameter: Tmax
Time frame: 52 weeks
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
Secondary PK parameter: Ctrough
Time frame: 52 weeks
Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted).
3 secondary outcomes
Hematologic responses
Time frame: 52 weeks
Molecular responses
Time frame: 52 weeks
Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks
Time frame: after first dose at Week 1 Day 1, 4 weeks, 52 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2026.
- Individual participant dataSharing planned: 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Novartis Pharmaceuticals contact
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
39 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Asciminib Pediatric formulation group and Asciminib Adult formulation group in people with Myeloid Leukemia, Philadelphia Positive, run by Novartis Pharmaceuticals at 39 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 39 sites in United States, China, France, Italy, Germany and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Novartis Pharmaceuticals (1-888-669-6682, novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Pharmacokinetic (PK) parameter: AUClast (time frame: 52 weeks). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2026.