Hetrombopag for Low/Intermediate-1 Risk MDS With Thrombocytopenia

NCT05024877 | Unknown Phase 2 DMC

• 1 other identifier: HBP-MDS-001
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• overall response rate at 6 months

  Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at 6 months

  6 month

Secondary Outcomes (8)

• percentage of side effects at 12 months

  12 months

• ISTH-BAT (ISTH bleeding assessment tool)

  12 months

• change of platelet transfusion

  12 months

• onset time for overall response

  through study completion, an average of 1 year

• duration of overall response

  through study completion, an average of 1 year

Study Arms (1)

Hetrombopag treatment group

EXPERIMENTAL

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)

Drug: Hetrombopag; Drug: Stanozolol Tablets

Interventions

Hetrombopag 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)

Hetrombopag treatment group

Stanozolol Tablets DRUG

Stanozolol would be given 2mg tid.

Hetrombopag treatment group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed MDS, IPSS low / intermediate risk-1
• Patients with EPO due to anemia and G-CSF due to severe neutropenia can be included, and the dosage will not change during trial
• ECOG 0-2 points
• Able to sign informed consent

You may not qualify if:

• Pregnant or lactating
• IPSS intermediate risk-2 / high risk MDS
• More than 5% of myeloblasts in bone marrow
• Myelofibrosis
• Previous transplantation or ATG treatment within 6 months
• Previous use of TPO or other TPO receptor agonists
• Active infection or tumor
• Thromboembolic or hemorrhagic disease
• Serious heart disease, including unstable angina, congestive heart failure, arrhythmia, 1-year history of myocardial infarction
• 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

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Related Publications (1)

• 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.

  PMID: 33632264 BACKGROUND

MeSH Terms

Interventions

hetrombopag; Stanozolol

Intervention Hierarchy (Ancestors)

Androstanols; Androstanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Bing Han, Docter

  Peking Union Medical College Hospital

  STUDY DIRECTOR

Central Study Contacts

Bing Han, Docter

CONTACT

+8613601059938; hanbing_li@sina.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Docter

Study Record Dates

• First Submitted: August 19, 2021
• First Posted: August 27, 2021
• Study Start: October 1, 2021
• Primary Completion: October 1, 2023
• Study Completion: December 1, 2023
• Last Updated: September 24, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: 10 years
• Access Criteria: personal contact including emails

Locations

CN (1)