Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies

NCT04993885 | Recruiting Phase 2

• 1 other identifier: IIT2021033
• Study Type: interventional
• Target: 52
• Locations: 1 country
• Sites: 1

Brief Summary

This prospective, open-label, single-center, one-arm clinical trial aims at evaluating the efficacy and safety of avatrombopag in Chinese adult Immune Thrombocytopenia (ITP) patients with autoantibodies fail (due to intolerance or resistance) to eltrombopag or herombopag treatment.

Conditions

Immune Thrombocytopenia; Autoantibodies; Evan Syndrome; Connective Tissue Diseases

Outcome Measures

Primary Outcomes (1)

• Platelet response

  Percentage of participants achieving a platelet count \>=30×10\^9/L and at least doubling of the baseline count within 12 weeks of treatment.

  From the start of study treatment (Day 1) up to the end of week 12.

Secondary Outcomes (14)

• Platelet response

  From the start of study treatment (Day 1) up to the end of week 12.

• Platelet response

  From the start of study treatment (Day 1) up to the end of week 12.

• Duration of platelet response

  From the start of study treatment (Day 1) up to the end of week 12.

• Platelet response

  From the start of study treatment (Day 1) up to the end of week 12.

• Time to platelet response

  From the start of study treatment (Day 1) up to the end of week 12.

Other Outcomes (1)

• Incidence of Toxicity

  From the start of study treatment (Day 1) up to the end of week 12.

Study Arms (1)

Treatment group

EXPERIMENTAL

Fifty-two subjects will be enrolled with the indicated treatment dose of avatrombopag

Drug: Avatrombopag

Interventions

Avatrombopag DRUG

The subjects will receive avatrombopag treatment with an initial dose of 40mg once a day. Platelet counts will be obtained weekly during the first 4 weeks and then every 2 weeks until week 12 after treatment. The dose adjustment range is 20 mg per week to 40 mg per day to maintain the platelet level between 30×10\^9/L and 200×10\^9/L. If the platelet count does not reach to 30×10\^9/L after taking avatrombopag 40mg once a day for 4 consecutive weeks, the treatment will be stopped.

Treatment group

Eligibility Criteria

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

You may qualify if:

• The patients have provided written informed consent prior to enrollment.
• Men and women greater than or equal to 18 years of age.
• Diagnosed as ITP secondary to connective tissue diseases (including but not limited to systemic lupus erythematosus, Sjogren's syndrome and rheumatoid arthritis), primary ITP with positive antinuclear antibody but not up to the diagnostic criteria of connective tissue diseases, primary Evans syndrome, Evans syndrome secondary to connective tissue diseases, and primary ITP with positive Coomb's test but not up to the diagnostic criteria of Evans syndrome.
• Platelet count\<30 ×10\^9/L at screening.
• Patients who have previously failed to receive Eltrombopag or Herombopag \[poor efficacy (Eltrombopag 75 mg/d or Herombopag 7.5 mg/D, at least 4 weeks), or the efficacy cannot be maintained\], or who have contraindications, can not tolerate or refuse Eltrombopag or Herombopag treatment.
• Treatment for ITP (including but not limited to glucocorticoids, recombinant human thrombopoietin, and other thrombopoietin receptor agonists other than Avatrombopag) must be completed before enrollment, or the dose must be stable or in a phase of reduction within 2 weeks before enrollment.
• Effective contraceptive measures will be taken during the clinical trial.

You may not qualify if:

• Patients with active thyroid disease requiring treatment.
• Patients with any prior history of arterial or venous thrombosis within 3 months, and with any of the following risk factors: cancer, Factor V Leiden, ATIII deficiency, or patients who are using anticoagulants or antiplatelet drugs at the beginning of screening.
• Those who had received rituximab within 3 months.
• Patients who had failed to respond to the previous use of Avatrombopag (40mg once a day for more than 4 weeks).
• Subjects known to be allergic to Avatrombopag or any of its excipients.
• Patients who had received splenectomy within 3 months or have splenectomy plan within 3 months.
• Patients with lupus encephalopathy or lupus nephritis.
• Patients with cataract.
• Patients with infectious fever (including but not limited to pulmonary infection) within 1 month or with active infection during screening.
• Existing hepatitis B virus, hepatitis C virus replication or HIV infection.
• Severe liver dysfunction (alanine aminotransferase or glutamic oxaloacetic transaminase \> 3×ULN).
• Patients with severe cardiac or pulmonary dysfunction.
• Severe renal damage (creatinine clearance \< 30 ml/min).
• There are surgical planners during the study.
• History of psychiatric disorder.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic; Evans Syndrome; Connective Tissue Diseases

Interventions

avatrombopag

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; Skin and Connective Tissue Diseases

Study Officials

• Lei Zhang, M.D.

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Rongfeng Fu, M.D.

CONTACT

+862223909009; furongfeng@ihcams.ac.cn

Lei Zhang, M.D.

CONTACT

+862223909240; zhanglei1@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2021
• First Posted: August 6, 2021
• Study Start: September 1, 2021
• Primary Completion: July 31, 2024
• Study Completion: September 30, 2025
• Last Updated: February 24, 2025

Record last verified: 2025-02

Locations

CN (1)