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

NCT05438875 | Completed Phase 3

• 1 other identifier: 2022-1-4082
• Study Type: interventional
• Target: 96
• Locations: 1 country
• Sites: 5

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).

Conditions

Purpura, Thrombocytopenic, Idiopathic

Keywords

Resistant/recurrent ITP; MSC-C5b-9 marker; all-trans retinoic acid; Eltrombopag; prospective clinical study

Outcome Measures

Primary Outcomes (1)

• Sustained Response Rate (SR) at 18 months

  The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 18-month follow-up

  18 months

Secondary Outcomes (8)

• Complete response rate (CR)

  18 months

• Response rate (R)

  18 months

• Inefficiency (NR)

  18 months

• Recurrence rate (relapse)

  18 months

• Early response

  1 week

Study Arms (2)

Eltrombopag

ACTIVE COMPARATOR

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.

Drug: Eltrombopag

ATRA and Eltrombopag

EXPERIMENTAL

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.

Drug: all-trans retinoic acid; Drug: Eltrombopag

Interventions

all-trans retinoic acid DRUG

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

Also known as: retinoic acid

ATRA and Eltrombopag

Eltrombopag 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.

Also known as: TPO-RA

ATRA and Eltrombopag; Eltrombopag

Eligibility Criteria

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

You may qualify if:

• \. 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.

You may not qualify if:

• \. 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.

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Beijing Friendship Hospital: collaborator
• Beijing Tongren Hospital: collaborator
• Beijing Hospital: collaborator
• Navy General Hospital, Beijing: collaborator

Study Sites (5)

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

Beijing, Beijing Municipality, 100010, China

Location

Beijing Tongren hospital

Beijing, Beijing Municipality, China

Location

Department of Hematology, Beijing Friendship Hospital, Capital Medical University

Beijing, China

Location

Department of Hematology, Beijing Hospital

Beijing, China

Location

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

Beijing, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Tretinoin; eltrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Vitamin A; Retinoids; Carotenoids; Polyenes; Alkenes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Cyclohexenes; Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Terpenes; Diterpenes; Pigments, Biological; Biological Factors

Study Officials

• Xiaohui Zhang, MD

  Study Principal Investigator Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Study Record Dates

• First Submitted: June 25, 2022
• First Posted: June 30, 2022
• Study Start: October 12, 2022
• Primary Completion: November 30, 2024
• Study Completion: November 30, 2024
• Last Updated: September 9, 2025

Record last verified: 2024-12

Locations

CN (5)