Myelodysplastic SyndromesPhase 2NCT05392647
Hetrombopag for Lower-risk MDS With Thrombocytopenia: a Prospective，Open-label, Single-arm Study.
A Phase 2 study testing Hetrombopag in people with Myelodysplastic Syndromes, run by Tongji Hospital.
Trial summary
The is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in lower-risk MDS patients with thrombocytopenia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndromes
- What is being tested?
- Hetrombopag
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 28 participants
- Where
- No sites listed yet
- Study dates
- 1 Jul 2022to 31 Dec 2024
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 18 years or older；
- Confirmed diagnosis of MDS based on 2016 WHO (World Health Organization) and classification with very low, low or intermediate (score ≤ 3.5) risk according to the revised International Prognostic Scoring System (IPSS-R).
- Platelets < 30 x 10^9/L or platelets < 50 x 10^9/L with active bleeding at screening.
- Eastern Cooperative Oncology Group (ECOG) performance score ≤ 2；
- Ability to understand information material and written informed consent.
Who cannot join
- Severe active bleeding/infection or any other uncontrolled severe condition;
- History of treatment with thrombopoietin receptor agonists (eltrombopag, avatrombopag, romiplostim, etc.) or recombinant human thrombopoietin with 1 month prior;
- Patients with known active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) at screening（If hepatitis B surface antigen or hepatitis B core antibody are positive, HBV-DNA testing are needed. If positive, it suggests a viral replication and subjects will be excluded. If hepatitis C antibody are positive, HCV-RNA testing are needed. If positive, it suggests a viral replication and subjects will be excluded）;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2.5 x upper limit of normal (ULN), total bilirubin >2.5 x ULN, creatinine >1.5 x ULN;
- History of portal hypertension or cirrhosis；
- History of any types of solid tumors within 5 years prior, whether the tumors has been treated, metastasized or relapsed (except basal cell carcinoma);
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- History of congestive heart failure requiring medical management, cardiac arrhythmias, peripheral arteriovenous thrombosis within 1 years prior, or myocardial infarction or cerebral infarction within 3 months prior;
- ECOG performance score ≥ 3;
- Female subjects who are pregnant or breastfeeding, or subjects who are planning pregnancy within 6 months;
- Women of child-bearing potential and men must agree to use contraception prior to study entry and for the duration of study participation;
- Participated in other clinical trials within 3 months prior;
- Any condition which, in the investigator's opinion, deems the subject an unsuitable candidate to receive study drug.
Trial locations and contacts
No sites are listed yet.
About this study
The is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in lower-risk MDS patients with thrombocytopenia.
Read the detailed description
The is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in patients with very low, low, or intermediate risk myelodysplastic syndromes(MDS) according to the revised International Prognostic Scoring System (IPSS-R) with thrombocytopenia. The primary endpoint was proportion of patients achieving platelet response at week 24.
- Conditions
- Myelodysplastic Syndromes
- Being tested
- Hetrombopag
- Keywords
- hetrombopag, thrombocytopenia, Myelodysplastic Syndromes
- Medical subject headings
- Myelodysplastic Syndromes, Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HetrombopagDrug
Also called Hetrombopag Olamine Tablets
All eligible patients were given hetrombopag orally at an initial dose of 5 mg once daily after overnight fasting (>8 hours of fasting) and at least 2 hours before a meal . The dose of hetrombopag could be modified on the basis of the platelet count of patients after 2 weeks of administration.
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | Hetrombopag Receives: Drug: Hetrombopag |
What is being measured
The primary outcome is the main question the study is designed to answer.
Platelet response
proportion of patients achieving platelet responses at week 24
Measured: 24 weeks
Secondary outcomes (4)
Overall response rate
Patients will be assessed for response according to the IWG criteria.
Measured: 24 weeks
Proportion of patients with platelet response during treatment
Improvement in platelet counts by 24 weeks of therapy
Measured: 24 weeks
Incidence and severity of bleeding events according to the WHO Bleeding Scale
The severity of bleeding symptoms was graded according to the World Health Organization (WHO) bleeding scale (grade 0: no bleeding; grade 1: petechiae; grade 2: mild blood loss; grade 3, gross blood loss; grade 4: debilitating blood loss).
Measured: 28 weeks
Adverse events
Adverse events were recorded and graded according to National Cancer Institute Common Terminology Criteria for Adverse Events, version 5.0
Measured: 28 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2022
- Study start1 Jul 2022 (estimated)
- Primary completion30 Jul 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromesStudies of the same condition, open and under way.
- More trials run by Tongji 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
- 28 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tongji Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jia Wei, Tongji Hospital
- Investigators
- Jia Wei, PhD, Principal investigator, Tongji Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05392647
- Sponsor's protocol ID
- OBU-HuB-MDS-II-001
- Status last verified
- May 2022
- First submitted
- 23 May 2022
- Last updated
- 26 May 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hetrombopag for Lower-risk MDS With Thrombocytopenia: a Prospective，Open-label, Single-arm Study.
- ClinicalTrials.gov ID
- NCT05392647
- Acronym / sponsor ID
- OBU-HuB-MDS-II-001
- Conditions
- Myelodysplastic Syndromes
- Keywords
- hetrombopag, thrombocytopenia, Myelodysplastic Syndromes
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 28 participants (estimated)
- Lead sponsor
- Tongji HospitalUniversity, hospital or other
- Study start
- 1 Jul 2022 (estimated)
- Primary completion
- 30 Jul 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 26 May 2022 (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 is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in lower-risk MDS patients with thrombocytopenia.
Detailed description
The is a phase 2, single-arm, open-label clinical study assessing the efficacy and safety of hetrombopag to increase platelet count in patients with very low, low, or intermediate risk myelodysplastic syndromes(MDS) according to the revised International Prognostic Scoring System (IPSS-R) with thrombocytopenia. The primary endpoint was proportion of patients achieving platelet response at week 24.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Drug: HetrombopagHetrombopag |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 18 years or older；
- Confirmed diagnosis of MDS based on 2016 WHO (World Health Organization) and classification with very low, low or intermediate (score ≤ 3.5) risk according to the revised International Prognostic Scoring System (IPSS-R).
- Platelets < 30 x 10^9/L or platelets < 50 x 10^9/L with active bleeding at screening.
- Eastern Cooperative Oncology Group (ECOG) performance score ≤ 2；
- Ability to understand information material and written informed consent.
Key exclusion criteria (12 listed)
- Severe active bleeding/infection or any other uncontrolled severe condition;
- History of treatment with thrombopoietin receptor agonists (eltrombopag, avatrombopag, romiplostim, etc.) or recombinant human thrombopoietin with 1 month prior;
- Patients with known active hepatitis B, hepatitis C, or human immunodeficiency virus (HIV) at screening（If hepatitis B surface antigen or hepatitis B core antibody are positive, HBV-DNA testing are needed. If positive, it suggests a viral replication and subjects will be excluded. If hepatitis C antibody are positive, HCV-RNA testing are needed. If positive, it suggests a viral replication and subjects will be excluded）;
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) >2.5 x upper limit of normal (ULN), total bilirubin >2.5 x ULN, creatinine >1.5 x ULN;
- History of portal hypertension or cirrhosis；
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 |
|---|---|---|
| HetrombopagAlso: Hetrombopag Olamine TabletsAll eligible patients were given hetrombopag orally at an initial dose of 5 mg once daily after overnight fasting (>8 hours of fasting) and at least 2 hours before a meal . The dose of hetrombopag could be modified on the basis of the platelet count of patients after 2 weeks of administration. | Drug | Single arm |
Primary outcome
Platelet response
Time frame: 24 weeks
proportion of patients achieving platelet responses at week 24
4 secondary outcomes
Overall response rate
Time frame: 24 weeks
Proportion of patients with platelet response during treatment
Time frame: 24 weeks
Incidence and severity of bleeding events according to the WHO Bleeding Scale
Time frame: 28 weeks
Adverse events
Time frame: 28 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2022.
- Individual participant dataSharing planned: No
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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.
Jia Wei, PhD principal investigator
Tongji Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Hetrombopag in people with Myelodysplastic Syndromes, run by Tongji Hospital.
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 5 inclusion and 12 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 26 May 2022. Status can change between updates, so confirm with the study contact before making plans.
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 Jia Wei, PhD (86-13986102084, jiawei@tjh.tjmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Platelet response (time frame: 24 weeks). 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 26 May 2022.