Core Binding Factor Acute Myeloid LeukemiaPhase 2NCT05821738
Avapritinib in CBF-AML With KIT Mutations
A Phase 2 study testing Avapritinib in people with Core Binding Factor Acute Myeloid Leukemia and KIT Mutation-Related Tumors, run by The First Affiliated Hospital of Soochow University at 1 site in China.
Trial summary
AML with t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as CBF-AML. KIT mutations are common in CBF-AML, which have a worse prognosis.This study is aimed to evaluate the efficacy of Avapritinib, an highly specific inhibitor of the KIT gene, in CBF-AML with KIT mutations. Read more about this study
- Who is it for?
- Ages 15 years and older
- Conditions the study is for
- Core Binding Factor Acute Myeloid Leukemia, KIT Mutation-Related Tumors
- What is being tested?
- Avapritinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteChina
- Study dates
- 1 Jun 2022to 31 Dec 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 15 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with acute myeloid leukemia accompanied by t(8; 21)/RUNX1-RUNX1T1, or inv(16)/t(16; 16)/CBFβ-MYH11;
- Accompanied by KIT mutation
- Disease recurrence after the first remission, or the mol-MRD remains positive after the morphologic remission of AML.
- No active infection.
- Liver function: TBIL≤ 2×ULN,ALT/AST≤ 3×ULN, CCr ≥ 50ml/min，NYHA grading ≤2; SaO2 >92%.
- ECOG <2;
- Predicted survival > 12 weeks.
Who cannot join
- Accept other AML targeted therapies, such as dasatinib, sorafenib, gilteritinib, etc. simultaneously;
- The presence of uncontrolled and active infections (including bacterial, fungal or viral infections).
- Underlying diseases such as myocardial infarction, chronic heart failure, decompensated liver dysfunction, renal failure, etc.
- Pregnant or lactating women;
- Accompanied by other malignant tumors requiring treatment;
- Other interventional clinical studies have been enrolled.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, 215000
Suning Chen, PhD, +8613814881746, chensuning@sina.com
Haiping Dai, 13914086271, daihaiping@126.com
About this study
Official title: Avapritinib in Relapsed Refractory or MRD-positive CBF-AML With KIT Mutations
AML with t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as CBF-AML. KIT mutations are common in CBF-AML, which have a worse prognosis.This study is aimed to evaluate the efficacy of Avapritinib, an highly specific inhibitor of the KIT gene, in CBF-AML with KIT mutations.
Read the detailed description
Acute Myeloid Leukemia (AML) with the chromosomal abnormality of t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as the Core Binding Factor AML (CBF-AML). KIT mutation is a common mutation in CBF-AML, which is more likely to relapse and have a worse prognosis.
Avapritinib is an oral tyrosine kinase inhibitor (TKI) with selective inhibitory activity against KIT and PDGFRA. Avapritinib has been approved by FDA for the treatment of gastrointestinal stromal tumors(GIST) with PDGFRA mutations and Advanced systemic mastocytosis (AdvSM). However, the efficacy of avapritinib in AML with KIT mutations is uncertain.
This prospective, multicenter clinical study of the efficacy and safety of avapritinib in relapsed refractory or molecular minimal residual disease (MRD)-positive AML with KIT mutations.
- Being tested
- Avapritinib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvapritinibDrug
administered orally
| Study group | Role | What they receive |
|---|---|---|
| Treatment Group | Experimental | The treatment group will receive avapritinib orally. The dosage is 100mg to 300mg qd, allowed to combine with other chemotheray drugs. Receives: Drug: Avapritinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite complete remission (CRc)
The proportion of participants who achieve Composite complete remission (CRc)，which includes complete remission (CR)、CR with partial hematologic recovery (CRh)、CR with incomplete blood count recovery (CRi) and morphology leukemia free (MLFS) based on response criteria for AML.
Measured: Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.
Secondary outcomes (4)
MRD-negative rate
The proportion of participants who achieve a negative molecular MRD.
Measured: Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.
Progression-free survival (PFS)
The Kaplan-Meier method will be used to assess PFS probabilities.
Measured: From the first day of treatment until any failure (resistant disease, relapse, or death), assessed up to 1 to 3 years.
Overall survival (OS)
The Kaplan-Meier method will be used to assess OS probabilities.
Measured: From the first day of treatment to time of death from any cause, assessed up 1 to 3 years.
Incidence of adverse events (AEs)
Terminology Criteria for Adverse Events (CTCAE) version (v) 5.0. The proportion of patients with AEs will be estimated, along with the 95% credible interval.
Measured: Up to approximately 1 to 3 years.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Apr 2023
- Study start1 Jun 2022
- Primary completion31 Dec 2024 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Core Binding Factor Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The First Affiliated Hospital of Soochow University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Chen Suning, The First Affiliated Hospital of Soochow University
- Investigators
- Suning Chen, Principal investigator, First Affiliated Hospital of Soochow University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05821738
- Sponsor's protocol ID
- SZ-AML-KIT
- Status last verified
- May 2023
- First submitted
- 7 Apr 2023
- Last updated
- 9 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Avapritinib in Relapsed Refractory or MRD-positive CBF-AML With KIT Mutations
- ClinicalTrials.gov ID
- NCT05821738
- Acronym / sponsor ID
- SZ-AML-KIT
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- The First Affiliated Hospital of Soochow UniversityUniversity, hospital or other
- Study start
- 1 Jun 2022 (actual)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 20 Apr 2023 (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.
AML with t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as CBF-AML. KIT mutations are common in CBF-AML, which have a worse prognosis.This study is aimed to evaluate the efficacy of Avapritinib, an highly specific inhibitor of the KIT gene, in CBF-AML with KIT mutations.
Detailed description
Acute Myeloid Leukemia (AML) with the chromosomal abnormality of t(8; 21)(q22; q22) or inv(16)(p13; q22)/t(16; 16)(p13; q22) is known as the Core Binding Factor AML (CBF-AML). KIT mutation is a common mutation in CBF-AML, which is more likely to relapse and have a worse prognosis.
Avapritinib is an oral tyrosine kinase inhibitor (TKI) with selective inhibitory activity against KIT and PDGFRA. Avapritinib has been approved by FDA for the treatment of gastrointestinal stromal tumors(GIST) with PDGFRA mutations and Advanced systemic mastocytosis (AdvSM). However, the efficacy of avapritinib in AML with KIT mutations is uncertain.
This prospective, multicenter clinical study of the efficacy and safety of avapritinib in relapsed refractory or molecular minimal residual disease (MRD)-positive AML with KIT mutations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | Drug: AvapritinibThe treatment group will receive avapritinib orally. The dosage is 100mg to 300mg qd, allowed to combine with other chemotheray drugs. |
Who may qualify
- Ages
- 15 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Patients with acute myeloid leukemia accompanied by t(8; 21)/RUNX1-RUNX1T1, or inv(16)/t(16; 16)/CBFβ-MYH11;
- Accompanied by KIT mutation
- Disease recurrence after the first remission, or the mol-MRD remains positive after the morphologic remission of AML.
- No active infection.
- Liver function: TBIL≤ 2×ULN,ALT/AST≤ 3×ULN, CCr ≥ 50ml/min，NYHA grading ≤2; SaO2 >92%.
Key exclusion criteria (6 listed)
- Accept other AML targeted therapies, such as dasatinib, sorafenib, gilteritinib, etc. simultaneously;
- The presence of uncontrolled and active infections (including bacterial, fungal or viral infections).
- Underlying diseases such as myocardial infarction, chronic heart failure, decompensated liver dysfunction, renal failure, etc.
- Pregnant or lactating women;
- Accompanied by other malignant tumors requiring treatment;
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 |
|---|---|---|
| Avapritinibadministered orally | Drug | Treatment Group |
Primary outcome
Composite complete remission (CRc)
Time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.
The proportion of participants who achieve Composite complete remission (CRc)，which includes complete remission (CR)、CR with partial hematologic recovery (CRh)、CR with incomplete blood count recovery (CRi) and morphology leukemia free (MLFS) based on response criteria for AML.
4 secondary outcomes
MRD-negative rate
Time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.
Progression-free survival (PFS)
Time frame: From the first day of treatment until any failure (resistant disease, relapse, or death), assessed up to 1 to 3 years.
Overall survival (OS)
Time frame: From the first day of treatment to time of death from any cause, assessed up 1 to 3 years.
Incidence of adverse events (AEs)
Time frame: Up to approximately 1 to 3 years.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 May 2023.
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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. This study is not recruiting at the moment.
Suning Chen principal investigator
First Affiliated Hospital of Soochow University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Avapritinib in people with Core Binding Factor Acute Myeloid Leukemia and KIT Mutation-Related Tumors, run by The First Affiliated Hospital of Soochow University at 1 site in China.
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 7 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 9 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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 phase mean?
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Suning Chen (+8613814881746, chensuning@sina.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Composite complete remission (CRc) (time frame: Assessed at protocol-defined timepoints through end of study, up to approximately 36 months.). 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 9 May 2023; AzTrials last refreshed its copy on 26 Oct 2026.