Hetrombopag for Low/Intermediate-1 Risk MDS With Thrombocytopenia

NCT ID: NCT05024877

Last Updated: 2021-09-24

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-01
• Study Completion Date: 2023-12-01

Brief Summary

Myelodysplastic syndrome (MDS) is a kind of clonal myeloid tumor. The major manifestation is decrease of tri-lineages of blood due to ineffective and abnormal hematopoiesis, some of which can progress to acute myeloid leukemia. According to the international prognosis scoring system (IPSS) of MDS, about 10% low/intermediate risk-1 MDS patients have severe thrombocytopenia (PLT < 30 × 109/ L). These patients have both decreased platelet count and platelet dysfunction, resulting in a high risk of bleeding. In the new prognostic score, such as IPSS-r, the degree of thrombocytopenia is regarded as a poor prognostic factor. Platelet transfusion is mainly used in the treatment of this kind of patients. The indications of transfusion include bleeding events or severe platelet count reduction (< 10 × 109 / L). However, platelet transfusion can only lead to short-term platelet elevation, while repeated transfusion increases the possibility of infection and ineffective platelet transfusion. TPO is a newly discovered hematopoietic promoting factor, which can specifically bind to the TPO receptor on the cell and participate in the regulation of proliferation, differentiation, maturation and division of megakaryocyte to form functional platelet. The efficacy and safety of the TPO receptor agonists eltrombopag and romiplostim in the treatment of thrombocytopenia in low/intermediate risk-1 MDS patients have been successfully confirmed in foreign studies. Hetrombopag is a new kind of a TPO receptor agonists which is highly specific platelet stimulating factor. At present, there is no large report on the application of Hetrombopag in such patients. The purpose of this study is to explore the short-term and long-term therapeutic effect and safety of Hetrombopag on low/intermediate risk-1 MDS patients.

Conditions

MDS

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Hetrombopag treatment group

stanozolol 2mg tid + Hetrombopag (started with 5mg/day and increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L, the maximum dosage is 15mg/day)

Group Type: EXPERIMENTAL

Hetrombopag

Intervention Type: DRUG

Hetrombopag would be given started with 5mg/day and increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L, the maximum dosage is 15mg/day)

Stanozolol Tablets

Intervention Type: DRUG

Stanozolol would be given 2mg tid.

Interventions

Hetrombopag

Hetrombopag would be given started with 5mg/day and increased by 2.5mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L, the maximum dosage is 15mg/day)

Intervention Type: DRUG

Stanozolol Tablets

Stanozolol would be given 2mg tid.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Confirmed MDS, IPSS low / intermediate risk-1

2. In the 4 weeks before inclusion, the average value of platelets was ≤ 30 × 10e9 / L, or < 50 × 10e9 / L with bleeding events

3. Patients with EPO due to anemia and G-CSF due to severe neutropenia can be included, and the dosage will not change during trial

4. ECOG 0-2 points

5. Able to sign informed consent

Exclusion Criteria

1. Pregnant or lactating

2. IPSS intermediate risk-2 / high risk MDS

3. More than 5% of myeloblasts in bone marrow

4. Myelofibrosis

5. Previous transplantation or ATG treatment within 6 months

6. Previous use of TPO or other TPO receptor agonists

7. Active infection or tumor

8. Thromboembolic or hemorrhagic disease

9. Serious heart disease, including unstable angina, congestive heart failure, arrhythmia, 1-year history of myocardial infarction

10. Baseline liver and kidney function: ALT / ASL over than 3 times normal upper limit, TBIL over than 2 times normal upper limit, and creatinine over than 2 times normal upper limit

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking Union Medical College Hospital

OTHER

Sponsor Role: lead

Responsible Party

Bing Han

Docter

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Bing Han, Docter

Role: STUDY_DIRECTOR

Peking Union Medical College Hospital

Locations

Peking Union Medical College Hospital

Beijing, , China

Countries

China

Central Contacts

Bing Han, Docter

Role: CONTACT

hanbing_li@sina.com

+8613601059938

References

Mei H, Liu X, Li Y, Zhou H, Feng Y, Gao G, Cheng P, Huang R, Yang L, Hu J, Hou M, Yao Y, Liu L, Wang Y, Wu D, Zhang L, Zheng C, Shen X, Hu Q, Liu J, Jin J, Luo J, Zeng Y, Gao S, Zhang X, Zhou X, Shi Q, Xia R, Xie X, Jiang Z, Gao L, Bai Y, Li Y, Xiong J, Li R, Zou J, Niu T, Yang R, Hu Y. A multicenter, randomized phase III trial of hetrombopag: a novel thrombopoietin receptor agonist for the treatment of immune thrombocytopenia. J Hematol Oncol. 2021 Feb 25;14(1):37. doi: 10.1186/s13045-021-01047-9.

Reference Type: BACKGROUND

PMID: 33632264 (View on PubMed)

Other Identifiers

HBP-MDS-001

Identifier Type: -

Identifier Source: org_study_id