Avatrombopag in Primary Immune Thrombocytopenia (ITP) in Adults (ITP)

• ClinicalTrials.gov Identifier: NCT04890041
• Recruitment Status: Recruiting
• First Posted: May 18, 2021
• Last Update Posted: May 18, 2021
• Sponsor: Institute of Hematology & Blood Diseases Hospital
• Information provided by (Responsible Party): Zhang Lei, Institute of Hematology & Blood Diseases Hospital

Study Description

Brief Summary:

This single-center study aims to study the efficacy and safety of avatrombopag in Chinese adult ITP patients. This study will be conducted in adult ITP patients who had not responded to eltrombopag or rhTPO in the previous treatment .

Condition or disease	Intervention/treatment	Phase
Primary Immune Thrombocytopenic Purpura	Drug: Avatrombopag	Not Applicable

Detailed Description:

The primary objective of this study was to evaluate the efficacy and safety of avatrombopag in adult ITP patients who had not responded to eltrombopag or rhTPO in the previous treatment.

20 eligible subjects will be enroll ed in this study.The dose will be adjusted according to the subject platelet count during the period from week 1 to week 8.

Study Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 20 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Efficacy and Safety of Avatrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults With Poor Efficacy of Eltrombopag or rhTPO: a Single-center, Prospective, One-arm Study
• Estimated Study Start Date: May 30, 2021
• Estimated Primary Completion Date: November 30, 2021
• Estimated Study Completion Date: December 31, 2021

Arms and Interventions

Arm

Intervention/treatment

Experimental: Treatment group; 20 subjects will be enrolled with the indicated treatment dose of avatrombopag

Drug: Avatrombopag; The subjects will receive avatrombopag treatment with an initial dose once a day. Platelet counts will be obtained weekly during the first 4 weeks and then every 2 weeks until the 8th week of the study. The dose adjustment range is 20 mg~ per week to 40 mg per day to maintain the platelet level between 50×10^9/L and 150×10^9/L. If the platelet count does not reach to 30×10^9/L after taking avatrombopag 40 mg once a day for 4 consecutive weeks, the treatment will be stopped. If the platelet count is more than 400×10^9/L after taking avatrombopag 20 mg once a day for 2 consecutive weeks, the treatment will be stopped.; Other Name: TPORAs

Outcome Measures

Primary Outcome Measures :

1. Treatment response [ Time Frame: From the start of study treatment (Day 1) up to the end of week 2 ]
   Percentage of participants achieving a platelet count >=30×10^9/L and at least doubling of the baseline count at week 2 of treatment

Secondary Outcome Measures :

1. Treatment response [ Time Frame: From the start of study treatment (Day 1) up to the end of week 1, 3, 4, 6 and 8. ]
   Percentage of participants achieving a platelet count >=30×10^9/L and at least doubling of the baseline count at week 1,3,4,6 and 8 of treatment
2. Treatment response [ Time Frame: From the start of study treatment (Day 1) up to the end of week 8 ]
   Percentage of participants achieving a platelet count >=50×10^9/L at week 8 of treatment
3. Time to Response [ Time Frame: From the start of study treatment (Day 1) up to the end of week 8 ]
   Time to response is defined as time from the start of treatment to the first time of achieving a platelet count >=30×10^9/L and at least doubling of the baseline count during the whole 8 weeks
4. Duration of response [ Time Frame: From the start of study treatment (Day 1) up to the end of week 8 ]
   Total duration of time a participant had a platelet count >=30×10^9/L
5. Evaluation of effectiveness [ Time Frame: From the start of study treatment (Day 1) up to the end of week 8 ]
   Number of participants that reduced or discontinued baseline concomitant ITP medications during the whole 8 weeks.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

• Men and women greater than or equal to 18 years of age.
• Participants diagnosed with chronic primary immune thrombocytopenia (greater than or equal to 12 months duration) and an average of 2 platelet counts < 30x10^9/L at least 7 days, do not have evidence of other causes of thrombocytopenia (e.g., pseudothrombocytopenia, myeloid fibrosis). The physical exam should not suggest any other diseases which may cause thrombocytopenia other than ITP.
• Previous treatment with Eltrombopag or rhTPO was ineffective (platelet count < 30x10^9/L after treatment, or platelet count increased less than twice the baseline value, or bleeding).
• Prothrombin time/International Normalized Ratio and activated partial thromboplastin time must have been within 80% to 120% of the normal range with no history of hypercoagulable state.
• Good compliance.

Exclusion Criteria:

• Patients diagnosed with secondary immune thrombocytopenia or a history of systemic lupus erythematosus and antiphospholipid antibody syndrome.
• A history of myelodysplastic syndrome (MDS), lymphoproliferative disease, myelodysplastic tumor, and leukemia.
• A history of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within 3 months before screening, and the following 2 or more risk factors: including estrogen hormone replacement therapy or contraceptive therapy, smoking, diabetes, hypercholesterolemia, high blood pressure medications, cancer or any other family history of arterial or venous thrombosis, or patients who were taking anticoagulants or antiplatelet drugs at the beginning of the screening.
• Participants with a history of significant cardiovascular disease (e.g., congestive heart failure [CHF] New York Heart Association Grade III, arrhythmia known to increase the risk of thromboembolic events [e.g., atrial fibrillation], coronary artery stent placement, angioplasty, coronary artery bypass grafting).
• Hereditary prethrombotic syndromes (e.g., thrombin factor V Leiden mutation, prothrombin G20210A mutation, or hereditary antithrombin (ATIII) deficiency).
• Participants with concurrent malignant tumor diseases.
• Blood creatinine >1.2 times the upper limit of normal value (ULN) .
• Participants with known allergy to avatrombopag or its excipients.
• Women who are pregnant or who intend to become pregnant in the near future are excluded.

Contacts and Locations

Contacts

Contact: Wei Liu; +82223909240; liuwei1@ihcams.ac.cn

Contact: Lei Zhang; +82223909240; zhanglei1@ihcams.ac.cn

Locations

China, Tianjin

Institute of Hematology & Blood Diseases Hospital; Recruiting

Tianjin, Tianjin, China, 300020

Contact: Lei Zhang, MD +862223909240 zhanglei1@ihcams.ac.cn

Contact: Lei Zhang +82223909240 zhanglei1@ihcams.ac.cn

Sponsors and Collaborators

Institute of Hematology & Blood Diseases Hospital

Investigators

• Principal Investigator: Lei Zhang; Institute of Hematology & Blood Diseases Hospital

More Information

• Responsible Party: Zhang Lei, Professor/Vice director of Thrombosis &Hemostasis Center, Institute of Hematology & Blood Diseases Hospital
• ClinicalTrials.gov Identifier: NCT04890041
• Other Study ID Numbers: SKX-ITP-001
• First Posted: May 18, 2021
• Last Update Posted: May 18, 2021
• Last Verified: May 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Thrombocytopenia; Immune System Diseases; Purpura; Purpura, Thrombocytopenic; Purpura, Thrombocytopenic, Idiopathic; Blood Platelet Disorders; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhage; Pathologic Processes; Skin Manifestations; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases