The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9

NCT ID: NCT05438875

Last Updated: 2025-09-09

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 96 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-10-12
• Study Completion Date: 2024-11-30

Brief Summary

A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).

Detailed Description

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

Conditions

Purpura, Thrombocytopenic, Idiopathic

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Eltrombopag

The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The highest is 75mg/d, if the dose is higher than 200×109/L, the dose is reduced. When the dose is higher than 400×109/L, the drug is temporarily discontinued, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.

Group Type: ACTIVE_COMPARATOR

Eltrombopag

Intervention Type: DRUG

The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.

ATRA and Eltrombopag

ATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.

Group Type: EXPERIMENTAL

all-trans retinoic acid

Intervention Type: DRUG

ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.

Eltrombopag

Intervention Type: DRUG

The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.

Interventions

all-trans retinoic acid

ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.

Intervention Type: DRUG

Eltrombopag

The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.

Intervention Type: DRUG

Other Intervention Names

retinoic acid; TPO-RA

Eligibility Criteria

Inclusion Criteria

• 1. Isolated thrombocytopenia (platelet count <30 × 109/L); 2. age > 18 years; 3. normal white blood cells and red blood cells on bone marrow examination; 4. increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease); 5. the spleen was normal in size; 6. Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2; 7. ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy; 8. Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment.

Exclusion Criteria

• 1. Secondary ITP such as drug-related thrombocytopenia; 2. thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus); 3. severe cardiac, renal, hepatic, or respiratory insufficiency; 4. severe immunodeficiency; 5. pregnancy or lactation; 6. myelodysplasia or Myelofibrosis; 7. history of malignancy; 8. ongoing immunosuppressive therapy for other diseases; 9. patients previously treated with eltrombopag were excluded from this study.

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

Sponsors

Beijing Friendship Hospital

OTHER

Sponsor Role: collaborator

Beijing Tongren Hospital

OTHER

Sponsor Role: collaborator

Beijing Hospital

OTHER_GOV

Sponsor Role: collaborator

Navy General Hospital, Beijing

OTHER

Sponsor Role: collaborator

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiao Hui Zhang

Vice president of Peking Univeristy Institute of Hematology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Xiaohui Zhang, MD

Role: PRINCIPAL_INVESTIGATOR

Study Principal Investigator Peking University People's Hospital

Locations

Peking University Insititute of Hematology, Peking University People's Hospital

Beijing, Beijing Municipality, China

Beijing Tongren hospital

Beijing, Beijing Municipality, China

Department of Hematology, Beijing Friendship Hospital, Capital Medical University

Beijing, , China

Department of Hematology, Beijing Hospital

Beijing, , China

Department of Hematology, Senior Department of Hematology, the Fifth Medical Center of PLA General Hospital

Beijing, , China

Countries

China

Other Identifiers

2022-1-4082

Identifier Type: -

Identifier Source: org_study_id