Acute Myeloid LeukemiaNCT04788420
Influence of Co-existing Mutations on Sorafenib Maintenance Therapy After Allo-HSCT for Patients With FLT3-ITD AML
An observational study of Acute Myeloid Leukemia and Acute Myeloid Leukemia With FLT3/ITD Mutation, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial summary
The purpose of this study is to reveal the influence of co-existing mutations on the efficacy of sorafenib maintenance after allogeneic hematopoietic stem cell transplantation for patients with FLT3-ITD AML. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Hematopoietic Stem Cell Transplantation
- What is being tested?
- Sorafenib
- Type of treatment
- Pill or oral medicine
- How many people
- 456 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2012to 31 Dec 2020
- Phase
- Observational
- 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 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Study population: Cases included in this study were from 4 previously published studies of our study group, including 3 retrospective and 1 prospective studies.
Who may be able to join
- FLT3-ITD Positive AML
- Allo-HSCT Recipients
Who cannot join
- cardiac dysfunction (particularly congestive heart failure)
- hepatic abnormalities (bilirubin ≥ 3 mg/dL, aminotransferase> 2 times the upper limit of normal)
- renal dysfunction (creatinine clearance rate < 30 mL/min)
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (according to the investigators' decision)
Trial locations and contacts
1 site in 1 country.
China1 site
Department of Hematology,Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
About this study
Official title: Influence of Co-existing Mutations on Sorafenib Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation for Patients With FLT3-ITD Positive Acute Myeloid Leukemia
The purpose of this study is to reveal the influence of co-existing mutations on the efficacy of sorafenib maintenance after allogeneic hematopoietic stem cell transplantation for patients with FLT3-ITD AML.
Read the detailed description
Internal tandem duplication of FMS-like tyrosine kinase 3 (FLT3-ITD) mutations have been reported in 20%-30% of patients with acute myeloid leukemia (AML). FLT3-ITD-positive AML patients have an inferior survival, primarily due to lower complete remission (CR) rate and higher relapse rate. Our previous studies demonstrated that post-transplantation sorafenib maintenance could improve the outcomes of FLT3-ITD-positive AML patients. However, whether other co-existing mutations influence the efficacy of post-allo-HSCT sorafenib maintenance remains unknown.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Hematopoietic Stem Cell Transplantation
- Being tested
- Sorafenib
- Keywords
- Acute Myeloid Leukemia, FLT3-ITD, Hematopoietic Stem Cell Transplantation, sorafenib
- 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.
SorafenibDrug
Also called Nexavar, BAY 43-9006, BAY-673472, BAY 545-9085
The initial dose of sorafenib is 400 mg orally twice daily and is adjusted in case of suspected toxicity or resistance (dose range, 200-800 mg daily).
| Study group | Role | What they receive |
|---|---|---|
| Sorafenib group | Patients assigned to this group received sorafenib post-transplantation. Receives: Drug: Sorafenib | |
| Control group | Patients assigned to this group did not receive sorafenib or any other FLT3 inhibitor post-transplantation until reaching the primary outcome. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of leukemia relapse
Measured: 2 year
Secondary outcomes (2)
Overall survival
Measured: 2 year
Leukemia-free survival
Measured: 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 Mar 2021
- Study start1 Jan 2012
- Primary completion21 Jul 2018
- Study completion31 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Acute Myeloid LeukemiaStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical UniversityThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 456 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University People's Hospital, Third Affiliated Hospital, Sun Yat-Sen University, Zhujiang Hospital, Xiangya Hospital of Central South University, First People's Hospital of Chenzhou, The First Affiliated Hospital of Guangzhou Medical University, Institute of Hematology & Blood Diseases Hospital, China, The First Affiliated Hospital of Soochow University, Xinqiao Hospital of Chongqing, First Affiliated Hospital of Zhejiang University
- Responsible party
- Principal investigator, Qifa Liu, Nanfang Hospital, Southern Medical University
- Investigators
- Qifa Liu, MD, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04788420
- Sponsor's protocol ID
- Sorafenib-Flt3 AML-2020
- Status last verified
- May 2022
- First submitted
- 5 Mar 2021
- Last updated
- 1 Jun 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Xuan L, Wang Y, Huang F, Jiang E, Deng L, Wu B, Fan Z, Liang X, Xu N, Ye J, Lin R, Yin C, Zhang Y, Sun J, Han M, Huang X, Liu Q. Effect of sorafenib on the outcomes of patients with FLT3-ITD acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation. Cancer. 2018 May 1;124(9):1954-1963. doi: 10.1002/cncr.31295. Epub 2018 Mar 6.
PubMed 29509276Xuan L, Wang Y, Huang F, Fan Z, Xu Y, Sun J, Xu N, Deng L, Li X, Liang X, Luo X, Shi P, Liu H, Wang Z, Jiang L, Yu C, Zhou X, Lin R, Chen Y, Tu S, Huang X, Liu Q. Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial. Lancet Oncol. 2020 Sep;21(9):1201-1212. doi: 10.1016/S1470-2045(20)30455-1. Epub 2020 Aug 10.
PubMed 32791048Xuan L, Wang Y, Chen J, Jiang E, Gao L, Wu B, Deng L, Liang X, Huang F, Fan Z, Tang X, Sun J, Zhang X, Han M, Wu D, Huang X, Liu Q. Sorafenib Therapy Is Associated with Improved Outcomes for FMS-like Tyrosine Kinase 3 Internal Tandem Duplication Acute Myeloid Leukemia Relapsing after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2019 Aug;25(8):1674-1681. doi: 10.1016/j.bbmt.2019.04.018. Epub 2019 Apr 19.
PubMed 31009704Shi J, Cao L, Luo Y, Zhao Y, Tan Y, Yu J, Lai X, Zhu Y, Hu Y, He J, Sun J, Zheng W, Wei G, Huang H. Maintenance sorafenib is superior to prophylactic donor lymphocyte infusion at improving the prognosis of acute myeloid leukemia with FMS-like tyrosine kinase 3 internal tandem duplication after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2021 Jan;56(1):293-296. doi: 10.1038/s41409-020-01015-w. Epub 2020 Aug 4. No abstract available.
PubMed 32753705
Study at a glance
From the registry record, last updated by the sponsor on 1 Jun 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Influence of Co-existing Mutations on Sorafenib Maintenance Therapy After Allogeneic Hematopoietic Stem Cell Transplantation for Patients With FLT3-ITD Positive Acute Myeloid Leukemia
- ClinicalTrials.gov ID
- NCT04788420
- Acronym / sponsor ID
- Sorafenib-Flt3 AML-2020
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Hematopoietic Stem Cell Transplantation
- Keywords
- Acute Myeloid Leukemia, FLT3-ITD, Hematopoietic Stem Cell Transplantation, sorafenib
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 456 participants (actual)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Collaborators
- Peking University People's Hospital, Third Affiliated Hospital, Sun Yat-Sen University, Zhujiang Hospital, Xiangya Hospital of Central South University, First People's Hospital of Chenzhou, The First Affiliated Hospital of Guangzhou Medical University, Institute of Hematology & Blood Diseases Hospital, China, The First Affiliated Hospital of Soochow University, Xinqiao Hospital of Chongqing, First Affiliated Hospital of Zhejiang University
- Study start
- 1 Jan 2012 (actual)
- Primary completion
- 21 Jul 2018 (actual)
- Study completion
- 31 Dec 2020 (actual)
- First posted
- 9 Mar 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 purpose of this study is to reveal the influence of co-existing mutations on the efficacy of sorafenib maintenance after allogeneic hematopoietic stem cell transplantation for patients with FLT3-ITD AML.
Detailed description
Internal tandem duplication of FMS-like tyrosine kinase 3 (FLT3-ITD) mutations have been reported in 20%-30% of patients with acute myeloid leukemia (AML). FLT3-ITD-positive AML patients have an inferior survival, primarily due to lower complete remission (CR) rate and higher relapse rate. Our previous studies demonstrated that post-transplantation sorafenib maintenance could improve the outcomes of FLT3-ITD-positive AML patients. However, whether other co-existing mutations influence the efficacy of post-allo-HSCT sorafenib maintenance remains unknown.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sorafenib group | – | Drug: SorafenibPatients assigned to this group received sorafenib post-transplantation. |
| Control group | – | –Patients assigned to this group did not receive sorafenib or any other FLT3 inhibitor post-transplantation until reaching the primary outcome. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Cases included in this study were from 4 previously published studies of our study group, including 3 retrospective and 1 prospective studies.
Key inclusion criteria (2 listed)
- FLT3-ITD Positive AML
- Allo-HSCT Recipients
Key exclusion criteria (5 listed)
- cardiac dysfunction (particularly congestive heart failure)
- hepatic abnormalities (bilirubin ≥ 3 mg/dL, aminotransferase> 2 times the upper limit of normal)
- renal dysfunction (creatinine clearance rate < 30 mL/min)
- Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
- Patients with any conditions not suitable for the trial (according to the investigators' decision)
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 |
|---|---|---|
| SorafenibAlso: Nexavar, BAY 43-9006, BAY-673472, BAY 545-9085The initial dose of sorafenib is 400 mg orally twice daily and is adjusted in case of suspected toxicity or resistance (dose range, 200-800 mg daily). | Drug | Sorafenib group |
Primary outcome
Incidence of leukemia relapse
Time frame: 2 year
2 secondary outcomes
Overall survival
Time frame: 2 year
Leukemia-free survival
Time frame: 2 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jun 2022.
- Background: PubMed 29509276Xuan L, Wang Y, Huang F, Jiang E, Deng L, Wu B, Fan Z, Liang X, Xu N, Ye J, Lin R, Yin C, Zhang Y, Sun J, Han M, Huang X, Liu Q. Effect of sorafenib on the outcomes of patients with FLT3-ITD acute myeloid leukemia undergoing allogeneic hematopoietic stem cell transplantation. Cancer. 2018 May 1;124(9):1954-1963. doi: 10.1002/cncr.31295. Epub 2018 Mar 6.
- Background: PubMed 32791048Xuan L, Wang Y, Huang F, Fan Z, Xu Y, Sun J, Xu N, Deng L, Li X, Liang X, Luo X, Shi P, Liu H, Wang Z, Jiang L, Yu C, Zhou X, Lin R, Chen Y, Tu S, Huang X, Liu Q. Sorafenib maintenance in patients with FLT3-ITD acute myeloid leukaemia undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised phase 3 trial. Lancet Oncol. 2020 Sep;21(9):1201-1212. doi: 10.1016/S1470-2045(20)30455-1. Epub 2020 Aug 10.
- Background: PubMed 31009704Xuan L, Wang Y, Chen J, Jiang E, Gao L, Wu B, Deng L, Liang X, Huang F, Fan Z, Tang X, Sun J, Zhang X, Han M, Wu D, Huang X, Liu Q. Sorafenib Therapy Is Associated with Improved Outcomes for FMS-like Tyrosine Kinase 3 Internal Tandem Duplication Acute Myeloid Leukemia Relapsing after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2019 Aug;25(8):1674-1681. doi: 10.1016/j.bbmt.2019.04.018. Epub 2019 Apr 19.
- Background: PubMed 32753705Shi J, Cao L, Luo Y, Zhao Y, Tan Y, Yu J, Lai X, Zhu Y, Hu Y, He J, Sun J, Zheng W, Wei G, Huang H. Maintenance sorafenib is superior to prophylactic donor lymphocyte infusion at improving the prognosis of acute myeloid leukemia with FMS-like tyrosine kinase 3 internal tandem duplication after allogeneic hematopoietic stem cell transplantation. Bone Marrow Transplant. 2021 Jan;56(1):293-296. doi: 10.1038/s41409-020-01015-w. Epub 2020 Aug 4. No abstract available.
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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.
Qifa Liu, MD principal investigator
Nanfang Hospital, Southern Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acute Myeloid Leukemia and Acute Myeloid Leukemia With FLT3/ITD Mutation, run by Nanfang Hospital, Southern Medical 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 2 inclusion and 5 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: finished, as of the sponsor's last update on 1 Jun 2022. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of leukemia relapse (time frame: 2 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04788420, which also lists 4 linked publications.
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 1 Jun 2022.