Acute Myeloid LeukemiaPhase 1NCT04272957
A Study of HMPL-306 in Patients With IDH1 and/or IDH2 Mutation of Relapsed/Refractory Myeloid Leukemia/Neoplasms
A Phase 1 study testing HMPL-306 in people with Acute Myeloid Leukemia, run by Hutchison Medipharma Limited at 1 site in China.
Trial summary
Phase I, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms with IDH1 and/or IDH2 Mutation. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia
- What is being tested?
- HMPL-306
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteChina
- Study dates
- 14 May 2020to 30 Dec 2022
- Phase
- Phase 1
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥18 years of age;
- Signed Informed Consent Form;
- Relapsed/refractory Acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia(CMML) and others myeloid neoplasm;
- IDH1 and/or IDH2 mutated disease status as assessed by local laboratory;
- Cooperative Oncology Group (ECOG) performance status of 0-2;
- Subjects must be amenable to serial bone marrow biopsies, peripheral blood sampling, and urine sampling during the study.
Who cannot join
- Previously treated with any prior IDH1 inhibitor, IDH2 inhibitor, or IDH1/IDH2 double-targeted therapy and had disease progression during treatment;
- with known involvement or clinical symptoms of central nervous system (CNS);
- Patients who have undergone HSCT within 60 days;
- Without adequate liver or kidney function;
- With known infection with active hepatitis B or C;
- With known infection with human immunodeficiency virus (HIV);
Show all 10Show fewer
- History of clinically significant or active cardiac disease;
- Active clinically significant infection;
- Taking known strong cytochrome P450 (CYP) 2C8 inducers or inhibitors;
- Pregnancy or breast-feeding.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Peking University People's Hospital
Beijing, Beijing Municipality
Xiaojun Huang, Professor, huangxiaojun@bjmu.edu.cn
About this study
Official title: A Phase I, Open-Label, Multicenter Study to Assess the Safety, Pharmacokinetics and Efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms With IDH1 and/or IDH2 Mutation
Phase I, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms with IDH1 and/or IDH2 Mutation.
Read the detailed description
The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms with IDH1 and/or IDH2 Mutation. The first stage of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of HMPL-306 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second stage of the study is a dose expansion phase where three cohorts of patients will receive HMPL-306 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose.
- Conditions
- Acute Myeloid Leukemia
- Being tested
- HMPL-306
- Keywords
- HMPL-306, IDH1 Mutation, IDH2 Mutation, Acute myeloid leukemia, Myelodysplastic symdrome, Chronic myelomonocytic leukemia, Myeloid Leukemia/Neoplasms
- Medical subject headings
- Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Chronic, Leukemia, Myeloid, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HMPL-306Drug
HMPL-306 administered continuously as a single agent starting at 25 mg orally every day in a 28-day cycle and dose escalation is planned up to 200mg. Subjects may continue treatment with HMPL-306 until disease progression, development of other unacceptable toxicity or hematopoietic stem cell transplant.
| Study group | Role | What they receive |
|---|---|---|
| HMPL-306 | Experimental | HMPL-306 administered continuously as a single agent orally every day in a 28-day cycle. Receives: Drug: HMPL-306 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability: Incidence of adverse events
Incidence of adverse events.
Measured: Baseline up to the last patient has completed the 24 weeks of treatment
Maximum tolerated dosage (MTD) and/or recommended phase 2 dosage (RP2D)
Measured by adverse event profile.
Measured: Baseline up to the last patient has completed the 24 weeks of treatment
Secondary outcomes (7)
Cmax (Cycle 1 Day 1) of HMPL-306
Cmax: maximum observed drug concentration in measured matrix after single dose administration.
Measured: Pre-dose, 10 minutes, 1, 1.5, 2, 3, 5, 8, 11, 24, 48, 72, 120 and 168 hours after start
AUC(0-24) (Cycle 1 Day 1) of HMPL-306
AUC: area under the concentration vs. time curve from zero to infinity after single (first) dose.
Measured: Pre-dose, 10 minutes, 1, 1.5, 2, 3, 5, 8, 11, 24, 48, 72, 120 and 168 hours after start
AUC(0-tlast) (Cycle 1 Day 1) of HMPL-306
AUC from time zero to the last data point.
Measured: Pre-dose, 10 minutes, 1, 1.5, 2, 3, 5, 8, 11, 24, 48, 72, 120 and 168 hours after start
Objective Response Rate (ORR)
proportion of patients with confirmed complete response (CR) and partial response (PR).
Measured: Baseline up to the last patient has completed the 24 weeks of treatment
Duration of response (DOR)
DOR is defined as the time from the date of first observed tumor response (Complete response (CR) or Partial response (PR)) until first subsequent disease progression or until death (if death occurs before progression is documented) due to any cause.
Measured: Baseline up to the last patient has completed the 24 weeks of treatment
Progression-free survival (PFS)
PFS is defined as the time from enrollment (i.e., date of treatment assignment) to disease progression.
Measured: Baseline up to the last patient has completed the 24 weeks of treatment
Overall survival (OS)
OS is defined as the time from enrollment (i.e., date of treatment assignment) until death from any cause or until the last date the patient is known to be alive.
Measured: Baseline up to the last patient has completed the 24 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2020
- Study start14 May 2020
- Primary completion30 Jun 2021 (estimated)
- Study completion30 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hutchison Medipharma Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Weiss Yang, Study director, Hutchison MediPharma Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04272957
- Sponsor's protocol ID
- 2018-306-00CH1
- Status last verified
- Jun 2020
- First submitted
- 13 Feb 2020
- Last updated
- 16 Jun 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Hu L, Wei X, Zhao W, Hu Y, Li J, Dong Y, Gong T, Zhang X, Xu Y, Zhang Y, Xu C, Zhang C, Cai Z, Jing H, Mi R, Wu W, He W, Wang H, Tang Q, Jiang Z, Liu H, Chen G, Sun J, Chen J, Yan S, Yan H, Wangwu J, Zhong Z, Wang L, Fan S, Shi M, Su W, Huang X. HMPL-306 in relapsed or refractory IDH1- and/or IDH2-mutated acute myeloid leukemia: A phase 1 study. Med. 2025 Jun 13;6(6):100575. doi: 10.1016/j.medj.2025.100575. Epub 2025 Jan 31.
PubMed 39892383
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label, Multicenter Study to Assess the Safety, Pharmacokinetics and Efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms With IDH1 and/or IDH2 Mutation
- ClinicalTrials.gov ID
- NCT04272957
- Acronym / sponsor ID
- 2018-306-00CH1
- Conditions
- Acute Myeloid Leukemia
- Keywords
- HMPL-306, IDH1 Mutation, IDH2 Mutation, Acute myeloid leukemia, Myelodysplastic symdrome, Chronic myelomonocytic leukemia, Myeloid Leukemia/Neoplasms
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Hutchison Medipharma LimitedIndustry (pharma, biotech, devices)
- Study start
- 14 May 2020 (actual)
- Primary completion
- 30 Jun 2021 (estimated)
- Study completion
- 30 Dec 2022 (estimated)
- First posted
- 17 Feb 2020 (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.
Phase I, multicenter study to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms with IDH1 and/or IDH2 Mutation.
Detailed description
The purpose of this Phase I, multicenter study is to evaluate the safety, pharmacokinetics, pharmacodynamics and efficacy of HMPL-306 in Patients of Relapsed/Refractory Myeloid Leukemia/Neoplasms with IDH1 and/or IDH2 Mutation. The first stage of the study is a dose escalation phase where cohorts of patients will receive ascending oral doses of HMPL-306 to determine maximum tolerated dose (MTD) and/or the recommended Phase II dose. The second stage of the study is a dose expansion phase where three cohorts of patients will receive HMPL-306 to further evaluate the safety, tolerability, and clinical activity of the recommended Phase II dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HMPL-306 | Experimental | Drug: HMPL-306HMPL-306 administered continuously as a single agent orally every day in a 28-day cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- ≥18 years of age;
- Signed Informed Consent Form;
- Relapsed/refractory Acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia(CMML) and others myeloid neoplasm;
- IDH1 and/or IDH2 mutated disease status as assessed by local laboratory;
- Cooperative Oncology Group (ECOG) performance status of 0-2;
Key exclusion criteria (10 listed)
- Previously treated with any prior IDH1 inhibitor, IDH2 inhibitor, or IDH1/IDH2 double-targeted therapy and had disease progression during treatment;
- with known involvement or clinical symptoms of central nervous system (CNS);
- Patients who have undergone HSCT within 60 days;
- Without adequate liver or kidney function;
- With known infection with active hepatitis B or C;
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 |
|---|---|---|
| HMPL-306HMPL-306 administered continuously as a single agent starting at 25 mg orally every day in a 28-day cycle and dose escalation is planned up to 200mg. Subjects may continue treatment with HMPL-306 until disease progression, development of other unacceptable toxicity or hematopoietic stem cell transplant. | Drug | HMPL-306 |
Primary outcome
Safety and tolerability: Incidence of adverse events
Time frame: Baseline up to the last patient has completed the 24 weeks of treatment
Incidence of adverse events.
Maximum tolerated dosage (MTD) and/or recommended phase 2 dosage (RP2D)
Time frame: Baseline up to the last patient has completed the 24 weeks of treatment
Measured by adverse event profile.
7 secondary outcomes
Cmax (Cycle 1 Day 1) of HMPL-306
Time frame: Pre-dose, 10 minutes, 1, 1.5, 2, 3, 5, 8, 11, 24, 48, 72, 120 and 168 hours after start
AUC(0-24) (Cycle 1 Day 1) of HMPL-306
Time frame: Pre-dose, 10 minutes, 1, 1.5, 2, 3, 5, 8, 11, 24, 48, 72, 120 and 168 hours after start
AUC(0-tlast) (Cycle 1 Day 1) of HMPL-306
Time frame: Pre-dose, 10 minutes, 1, 1.5, 2, 3, 5, 8, 11, 24, 48, 72, 120 and 168 hours after start
Objective Response Rate (ORR)
Time frame: Baseline up to the last patient has completed the 24 weeks of treatment
Duration of response (DOR)
Time frame: Baseline up to the last patient has completed the 24 weeks of treatment
Progression-free survival (PFS)
Time frame: Baseline up to the last patient has completed the 24 weeks of treatment
Overall survival (OS)
Time frame: Baseline up to the last patient has completed the 24 weeks of treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2020.
- Derived from this study: PubMed 39892383Hu L, Wei X, Zhao W, Hu Y, Li J, Dong Y, Gong T, Zhang X, Xu Y, Zhang Y, Xu C, Zhang C, Cai Z, Jing H, Mi R, Wu W, He W, Wang H, Tang Q, Jiang Z, Liu H, Chen G, Sun J, Chen J, Yan S, Yan H, Wangwu J, Zhong Z, Wang L, Fan S, Shi M, Su W, Huang X. HMPL-306 in relapsed or refractory IDH1- and/or IDH2-mutated acute myeloid leukemia: A phase 1 study. Med. 2025 Jun 13;6(6):100575. doi: 10.1016/j.medj.2025.100575. Epub 2025 Jan 31.
- Individual participant dataSharing planned: No
Related trials for Acute Myeloid Leukemia
RecruitingPhase 1
A Study of CHM-029 in Participants With NPM1 Mutated, KMT2A or NUP98 Rearranged AML
AML (Acute Myeloid Leukemia)
RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
RecruitingPhase 1/2
Venetoclax and CLAG-M for the Treatment of Acute Myeloid Leukemia and High-Grade Myeloid Neoplasms
Acute Biphenotypic Leukemia, Acute Myeloid Leukemia
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome
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.
Weiss Yang study director
Hutchison MediPharma Ltd
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HMPL-306 in people with Acute Myeloid Leukemia, run by Hutchison Medipharma Limited 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 6 inclusion and 10 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 16 Jun 2020. 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 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.
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 Xianlin Duan (02120678852, xianlind@hmplglobal.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability: Incidence of adverse events (time frame: Baseline up to the last patient has completed the 24 weeks of treatment). The study lists 2 primary outcomes in all. 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 NCT04272957, which also lists 1 linked publication.
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 16 Jun 2020.