AML, AdultPhase 2NCT06765915
Avapritinib Maintenance for AML With KIT Mutations
A Phase 2 study testing Avapritinib in people with AML, Adult, run by Ruijin Hospital at 1 site in China.
Trial summary
A multicenter, single-arm clinical study of evaluate the efficacy and safety of avapritinib as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients with KIT mutation. Read more about this study
- Who is it for?
- Ages 14 to 70 years
- Conditions the study is for
- AML, Adult
- 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
- 47 participants
- Where
- 1 siteChina
- Study dates
- 1 Feb 2025to 1 Feb 2028
- Phase
- Phase 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 14 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age≥ 14 years old;
- First allo-HSCT for AML (including secondary AML) ;
- KIT mutation at diagnosis (no restriction on locus for kit mutation
- CR and negative MFC-MRD prior to initiation of maintenance therapy;
- Absolute neutrophil count ≥ 1.0 x 109 /L, platelets ≥ 75 x 109 /L, hemoglobin ≥ 80 g/L before maintenance;
- Normal functioning of major organs and laboratory findings in accordance with the following criteria:AST and ALT) ≤ 3x ULN; Total serum bilirubin ≤ 1.5x ULN unless the patient has Gilbert syndrome; patients with Gilbert-Meulengracht syndrome with bilirubin ≤ 3.0 times the upper limit of normal and direct bilirubin ≤ 1.5 times the upper limit of normal may be included; HB ≥ 70 g/L (had not received a red blood cell transfusion within 1 week prior to administration); ANC ≥ 0.8 x 10^9/L (had not received long-acting colony-stimulating factor (LACSF) within 1 week prior to administration and short-acting colony-stimulating factor (SACSF) within 3 days prior to administration); Platelet count ≥ 20 x 10^9/L (had not received a platelet transfusion within 1 week prior to administration); serum creatinine ≤ 1.5x ULN or creatinine clearance ≥ 60 mL/min; Coagulation function: International Normalized Ratio (INR) ≤1.5×ULN, Activated Partial Thromboplastin Time (APTT) ≤1.5×ULN; Left ventricular ejection fraction (LVEF) ≥45%;
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- ECOG PS 0-2 points;
- Expected survival ≥ 3 months;
- Patient consent
Who cannot join
- concurrently receiving other targeted therapies for AML;
- Prior treatment with a TKI inhibitor that proved ineffective;
- with concurrent FLT3-ITD mutations at enrollment;
- Acute/chronic graft-versus-host disease requiring systemic immunosuppressive therapy prior to maintenance therapy;
- Accompanied by other malignant tumors requiring treatment;
- Have important organ-based diseases: e.g., myocardial infarction, chronic cardiac insufficiency, decompensated hepatic insufficiency, renal failure;
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- Active, uncontrolled infection;
- HIV-positive, active hepatitis B (HBV) or active hepatitis C (HCV) requiring antiviral therapy;
- Other interventional clinical studies have been enrolled;
- Men and women of childbearing potential are unwilling to use contraception during and for 12 months after treatment;
- The investigator believes that there are other conditions that make the patient unsuitable for participation in this study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Ruijin Hospital of Shanghai Jiaotong University
Shanghai, Shanghai Municipality
About this study
Official title: Avapritinib Maintenance Following Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia With KIT Mutations
A multicenter, single-arm clinical study of evaluate the efficacy and safety of avapritinib as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients with KIT mutation.
Read the detailed description
It has been reported that in patients with CBF-associated acute myeloid leukemia (CBF-AML), c-kit mutations occur preferentially in patients with core-binding factor ((8; 21) and inv(16) or t(16; 16) (referred to as inv(16)) rearrangements. However, c-KIT gene mutations, which occur more frequently in patients with CBF-AML, have an incidence of 10% to 45%, which in turn leads to relapse, suggesting a poor prognosis. Nearly 50% of AML patients with concomitant t(8;21) have the c-KIT-D816 mutation. Among AML patients with t(8; 21), patients with c-KIT D816 mutation had significantly shorter OS and EFS than those without this mutation.
Avapritinib, is a novel inhibitor of KIT and PDGFRA-activating ring mutants and a potent and selective inhibitor of KIT D816V, and preclinical studies have demonstrated that compared to Midostauin, Avapritinib is 10-fold more potent against this mutant kinase was 10-fold more potent. A retrospective study [ 14 ] analyzed the efficacy of Avapritinib in patients with t (8;21) AML with KIT mutations who failed allo-HSCT treatment. Among the 13 patients in the D816 mutation, 8 cases reduced RUNX1-RUNX1T1 transcript levels by ≥1 log after 1 month of treatment, and 3 cases turned negative. However, whether avapritinib is effective for maintenance therapy in CBF-AML patients who harbor KIT mutations is unknown.
There is a lack of prospective, controlled studies to clarify the efficacy and safety of XPO1 inhibitor combined with venetoclax as maintenance therapy after allo-HSCTin patients with intermediate- to high-risk AML/MDS, especially those with out specific gene mutation which would be targeted with commerically available inhibitors. Therefore, this multicenter, single-arm study is designed to assess the efficacy and safety of avapritinib as maintenance therapy in CBF-AML patients who harbor KIT mutations after allo-HSCT, with the aim of providing a reference for clinical treatment.
- Conditions
- AML, Adult
- Being tested
- Avapritinib
- Keywords
- AML, KIT mutation, avapritinib
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AvapritinibDrug
Also called KIT inhibitor
After allo-HSCT, CBF-AML patients who have kit mutation would receive avapritinib for maintenance therapy.
| Study group | Role | What they receive |
|---|---|---|
| Experimental arm | Experimental | Allo-HSCT recipients will begin maintenance therapy with oral Avapritinib tablets at a dose of 100 mg/day, starting 2-4 months post-transplantation. Each treatment cycle lasts 28 days, and therapy will continue for up to 2 years or until disease progression or unacceptable toxicity occurs. Receives: Drug: Avapritinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cumulative incidence of relapse
disease relapse
Measured: through study completion, an average of 2 year
Secondary outcomes (2)
Overall survival
death as a result of any causes
Measured: through study completion, an average of 2 year
Non relapse mortality
death without disease progression or relapse
Measured: through study completion, an average of 2 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2025
- Study start1 Feb 2025 (estimated)
- Primary completion1 Feb 2026 (estimated)
- Study completion1 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AML, AdultStudies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 47 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hu Xiaoxia, Ruijin Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06765915
- Sponsor's protocol ID
- RJ-BMT-011
- Status last verified
- Jan 2025
- First submitted
- 5 Jan 2025
- Last updated
- 9 Jan 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Avapritinib Maintenance Following Allogeneic Hematopoietic Stem Cell Transplantation in Acute Myeloid Leukemia With KIT Mutations
- ClinicalTrials.gov ID
- NCT06765915
- Acronym / sponsor ID
- RJ-BMT-011
- Conditions
- AML, Adult
- Keywords
- AML, KIT mutation, avapritinib
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 47 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 1 Feb 2025 (estimated)
- Primary completion
- 1 Feb 2026 (estimated)
- Study completion
- 1 Feb 2028 (estimated)
- First posted
- 9 Jan 2025 (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.
A multicenter, single-arm clinical study of evaluate the efficacy and safety of avapritinib as maintenance therapy following allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia patients with KIT mutation.
Detailed description
It has been reported that in patients with CBF-associated acute myeloid leukemia (CBF-AML), c-kit mutations occur preferentially in patients with core-binding factor ((8; 21) and inv(16) or t(16; 16) (referred to as inv(16)) rearrangements. However, c-KIT gene mutations, which occur more frequently in patients with CBF-AML, have an incidence of 10% to 45%, which in turn leads to relapse, suggesting a poor prognosis. Nearly 50% of AML patients with concomitant t(8;21) have the c-KIT-D816 mutation. Among AML patients with t(8; 21), patients with c-KIT D816 mutation had significantly shorter OS and EFS than those without this mutation.
Avapritinib, is a novel inhibitor of KIT and PDGFRA-activating ring mutants and a potent and selective inhibitor of KIT D816V, and preclinical studies have demonstrated that compared to Midostauin, Avapritinib is 10-fold more potent against this mutant kinase was 10-fold more potent. A retrospective study [ 14 ] analyzed the efficacy of Avapritinib in patients with t (8;21) AML with KIT mutations who failed allo-HSCT treatment. Among the 13 patients in the D816 mutation, 8 cases reduced RUNX1-RUNX1T1 transcript levels by ≥1 log after 1 month of treatment, and 3 cases turned negative. However, whether avapritinib is effective for maintenance therapy in CBF-AML patients who harbor KIT mutations is unknown.
There is a lack of prospective, controlled studies to clarify the efficacy and safety of XPO1 inhibitor combined with venetoclax as maintenance therapy after allo-HSCTin patients with intermediate- to high-risk AML/MDS, especially those with out specific gene mutation which would be targeted with commerically available inhibitors. Therefore, this multicenter, single-arm study is designed to assess the efficacy and safety of avapritinib as maintenance therapy in CBF-AML patients who harbor KIT mutations after allo-HSCT, with the aim of providing a reference for clinical treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental arm | Experimental | Drug: AvapritinibAllo-HSCT recipients will begin maintenance therapy with oral Avapritinib tablets at a dose of 100 mg/day, starting 2-4 months post-transplantation. Each treatment cycle lasts 28 days, and therapy will continue for up to 2 years or until disease progression or unacceptable toxicity occurs. |
Who may qualify
- Ages
- 14 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- Age≥ 14 years old;
- First allo-HSCT for AML (including secondary AML) ;
- KIT mutation at diagnosis (no restriction on locus for kit mutation
- CR and negative MFC-MRD prior to initiation of maintenance therapy;
- Absolute neutrophil count ≥ 1.0 x 109 /L, platelets ≥ 75 x 109 /L, hemoglobin ≥ 80 g/L before maintenance;
Key exclusion criteria (11 listed)
- concurrently receiving other targeted therapies for AML;
- Prior treatment with a TKI inhibitor that proved ineffective;
- with concurrent FLT3-ITD mutations at enrollment;
- Acute/chronic graft-versus-host disease requiring systemic immunosuppressive therapy prior to maintenance therapy;
- 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 |
|---|---|---|
| AvapritinibAlso: KIT inhibitorAfter allo-HSCT, CBF-AML patients who have kit mutation would receive avapritinib for maintenance therapy. | Drug | Experimental arm |
Primary outcome
Cumulative incidence of relapse
Time frame: through study completion, an average of 2 year
disease relapse
2 secondary outcomes
Overall survival
Time frame: through study completion, an average of 2 year
Non relapse mortality
Time frame: through study completion, an average of 2 year
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 9 Jan 2025.
- Individual participant dataSharing planned: Undecided
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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.
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 AML, Adult, run by Ruijin Hospital 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 9 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: opening soon, as of the sponsor's last update on 9 Jan 2025. 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?
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 Xiaoxia HU, Doctor (02164370045, hxx12276@rjh.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Cumulative incidence of relapse (time frame: through study completion, an average of 2 year). 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 Jan 2025.