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Efficacy and Safety of TPO Receptor Agonists in the Treatment of Elderly ITP Patients

Primary Purpose

Primary Immune Thrombocytopenia

Status
Not yet recruiting
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
2.5mg/d Hetrombopag
Sponsored by
Wuhan Union Hospital, China
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Primary Immune Thrombocytopenia focused on measuring hetrombopag, Primary Immune Thrombocytopenia, platelets

Eligibility Criteria

60 Years - 100 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 1. The patient voluntarily signed the informed consent; 2. The patient is a newly diagnosed ITP patient, aged ≥60 years old; 3. Two consecutive PLTs < 30×109/L, or two consecutive PLTs < 30×109/L≤PLT<50×109/L but with risk factors such as bleeding (such as previous bleeding history and/or anticoagulation/antiplatelet) Concomitant medication) or age > 75 years; 4. Have not received first-line treatment such as hormones, IVIG, etc.; 5. There is any hormonal contraindication (with active peptic ulcer, recent gastrointestinal anastomosis, corneal ulcer, severe hypertension (high blood pressure ≥ grade 2), diabetes with poor blood sugar control, infection that cannot be controlled by antibiotics ( Bacterial and viral infections), heart failure and adrenal hyperfunction, severe mental illnesses such as epilepsy, severe osteoporosis, rheumatoid arthritis, tuberculosis, fractures, patients with combined antithrombotic and antiplatelet drugs, etc.); 6. The following clinical biochemical indicators must be within ±20% of the upper and lower limits of normal values: creatinine, alanine aminotransferase, aspartate aminotransferase, total bilirubin and alkaline phosphatase.

Exclusion Criteria:

  • 1. Exclude immune diseases such as systemic lupus erythematosus, antiphospholipid syndrome, etc.; 2. Exclude drug-related thrombocytopenia; 3. Bone marrow-related examinations suggest the presence of other primary diseases of the blood system (such as MDS, AA, thrombotic thrombocytopenic purpura, etc.) or the presence of myelofibrosis MF≥2; 4. Participated in other clinical trials affecting platelet count and function 3 months before the trial; 5. Previously received first-line therapy such as hormones, IVIG; 6. Previous use of TPO-RAs or poor efficacy against known TPO drugs; 7. The patient has experienced severe arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), or clinical symptoms suggest thrombophilia; 8. HIV, hepatitis B or C seropositive or a history of liver cirrhosis or portal hypertension; 9. Life-threatening bleeding (WHO bleeding score 4) or the patient is expected to require salvage treatment before the first dose; 10. Has a history of malignant tumor or is accompanied by malignant tumor; 11. The investigator believes that there are any other circumstances that may cause the subjects to fail to complete the study or bring obvious risks to the subjects.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    2.5mg/d

    Arm Description

    After the subjects signed the informed consent and passed the screening, they entered the treatment period and received a starting dose of 2.5 mg/d of Hetrabopag. During the treatment process, the clinician adjusted the drug dose according to the patient's own conditions. The maximum drug dose was 7.5 mg qd, 28 d Evaluate efficacy and safety after completion;

    Outcomes

    Primary Outcome Measures

    Treatment response
    Proportion of subjects with platelet counts ≥50×10^9/L after 28 days of treatment

    Secondary Outcome Measures

    Remission rate
    Complete response, effective, ineffective proportion of testers after 28 days of treatment
    Drug efficacy
    During treatment, the proportion of subjects, which the platelet count at least once reached ≥50×109/L.
    Evaluation of effectiveness
    During treatment, the proportion of subjects, which the platelet count increased at least 2 times compared with baseline.
    Adverse events
    Evaluate the incidence and severity of bleeding based on the ITP-BAT bleeding score
    Adverse events
    During treatment, the proportion of subjects, which received at least once rescue
    Side effects of drugs
    Assessing safety through the adverse events,such as liver damage, etc.

    Full Information

    First Posted
    March 28, 2022
    Last Updated
    March 28, 2022
    Sponsor
    Wuhan Union Hospital, China
    Collaborators
    Jiangsu Hengrui Pharmaceutical Co., Ltd.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05311930
    Brief Title
    Efficacy and Safety of TPO Receptor Agonists in the Treatment of Elderly ITP Patients
    Official Title
    Efficacy and Safety of TPO Receptor Agonists as First-line Drugs in the Treatment of Newly Diagnosed Elderly ITP Patients in China-an Open Label Study
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2022
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    May 1, 2022 (Anticipated)
    Primary Completion Date
    December 30, 2024 (Anticipated)
    Study Completion Date
    December 30, 2024 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Wuhan Union Hospital, China
    Collaborators
    Jiangsu Hengrui Pharmaceutical Co., Ltd.

    4. Oversight

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

    5. Study Description

    Brief Summary
    Elderly ITP patients have many underlying diseases, hormone contraindications and many adverse reactions during the use of hormones. TPO-RAs are oral small-molecule non-peptide drugs. Retrospective studies have shown that they have good efficacy and high safety in elderly patients. Therefore, this study is a prospective trial to evaluate TPO-RAs as the first-choice drug for the treatment of elderly ITP patients with contraindications to hormones, aiming to improve the efficacy-risk ratio of elderly patients
    Detailed Description
    Primary immune thrombocytopenia (ITP) is an immune disorder characterized by decreased production and increased destruction of platelets. In recent years, with the in-depth exploration of its pathogenesis and the continuous influx of new drugs, the status of second-line treatment drugs, mainly TPO receptor agonists (TPO-RAs), has been continuously improved, which has brought great importance to the treatment and management of ITP patients. However, for elderly ITP patients with severe underlying diseases, poor hormone tolerance, severe adverse reactions or hormone contraindications, whether TPO receptor agonists can be used as the first-choice drug and its efficacy and safety are still lacking relevant research, and for elderly ITP patients There is a lack of uniform guidelines for the treatment and management of patients. Limited retrospective studies have shown that TPO receptor agonists have good safety and efficacy, and are expected to become the recommended drugs for the treatment of elderly patients with ITP. However, whether TPO receptor agonists can be directly used as the first-choice drug for newly diagnosed elderly ITP patients who are not suitable for first-line treatment and its efficacy are uncertain. This study will include newly diagnosed elderly ITP patients with hormonal contraindications or potential serious side effects of hormonal therapy, take the TPO-RA drug hetropoda as the first-choice treatment drug, and explore the effectiveness of hetrompopag in such patients and safety analysis. This study will provide new ideas and clinical basis for standardized and individualized treatment of elderly ITP patients, and provide practical experience for promoting the establishment of elderly ITP treatment guidelines.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Primary Immune Thrombocytopenia
    Keywords
    hetrombopag, Primary Immune Thrombocytopenia, platelets

    7. Study Design

    Primary Purpose
    Other
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Model Description
    This is a single-arm open-label trial. After enrollment, patients received an initial dose of 2.5 mg of hetropoda for 4 weeks, and blood routine monitoring was performed regularly during treatment.
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    69 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    2.5mg/d
    Arm Type
    Experimental
    Arm Description
    After the subjects signed the informed consent and passed the screening, they entered the treatment period and received a starting dose of 2.5 mg/d of Hetrabopag. During the treatment process, the clinician adjusted the drug dose according to the patient's own conditions. The maximum drug dose was 7.5 mg qd, 28 d Evaluate efficacy and safety after completion;
    Intervention Type
    Drug
    Intervention Name(s)
    2.5mg/d Hetrombopag
    Other Intervention Name(s)
    no use
    Intervention Description
    After the subjects signed the informed consent and passed the screening, they entered the treatment period and received a starting dose of 2.5 mg/d of Hytrombopag. During the treatment process, the clinician adjusted the drug dose according to the patient's own conditions. The maximum drug dose was 7.5 mg qd, 28 d Evaluate efficacy and safety after completion;
    Primary Outcome Measure Information:
    Title
    Treatment response
    Description
    Proportion of subjects with platelet counts ≥50×10^9/L after 28 days of treatment
    Time Frame
    28 days
    Secondary Outcome Measure Information:
    Title
    Remission rate
    Description
    Complete response, effective, ineffective proportion of testers after 28 days of treatment
    Time Frame
    28 days
    Title
    Drug efficacy
    Description
    During treatment, the proportion of subjects, which the platelet count at least once reached ≥50×109/L.
    Time Frame
    28 days
    Title
    Evaluation of effectiveness
    Description
    During treatment, the proportion of subjects, which the platelet count increased at least 2 times compared with baseline.
    Time Frame
    28 days
    Title
    Adverse events
    Description
    Evaluate the incidence and severity of bleeding based on the ITP-BAT bleeding score
    Time Frame
    6 months from treatment
    Title
    Adverse events
    Description
    During treatment, the proportion of subjects, which received at least once rescue
    Time Frame
    6 months from treatment
    Title
    Side effects of drugs
    Description
    Assessing safety through the adverse events,such as liver damage, etc.
    Time Frame
    1 month from treatment

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    60 Years
    Maximum Age & Unit of Time
    100 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: 1. The patient voluntarily signed the informed consent; 2. The patient is a newly diagnosed ITP patient, aged ≥60 years old; 3. Two consecutive PLTs < 30×109/L, or two consecutive PLTs < 30×109/L≤PLT<50×109/L but with risk factors such as bleeding (such as previous bleeding history and/or anticoagulation/antiplatelet) Concomitant medication) or age > 75 years; 4. Have not received first-line treatment such as hormones, IVIG, etc.; 5. There is any hormonal contraindication (with active peptic ulcer, recent gastrointestinal anastomosis, corneal ulcer, severe hypertension (high blood pressure ≥ grade 2), diabetes with poor blood sugar control, infection that cannot be controlled by antibiotics ( Bacterial and viral infections), heart failure and adrenal hyperfunction, severe mental illnesses such as epilepsy, severe osteoporosis, rheumatoid arthritis, tuberculosis, fractures, patients with combined antithrombotic and antiplatelet drugs, etc.); 6. The following clinical biochemical indicators must be within ±20% of the upper and lower limits of normal values: creatinine, alanine aminotransferase, aspartate aminotransferase, total bilirubin and alkaline phosphatase. Exclusion Criteria: 1. Exclude immune diseases such as systemic lupus erythematosus, antiphospholipid syndrome, etc.; 2. Exclude drug-related thrombocytopenia; 3. Bone marrow-related examinations suggest the presence of other primary diseases of the blood system (such as MDS, AA, thrombotic thrombocytopenic purpura, etc.) or the presence of myelofibrosis MF≥2; 4. Participated in other clinical trials affecting platelet count and function 3 months before the trial; 5. Previously received first-line therapy such as hormones, IVIG; 6. Previous use of TPO-RAs or poor efficacy against known TPO drugs; 7. The patient has experienced severe arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism), or clinical symptoms suggest thrombophilia; 8. HIV, hepatitis B or C seropositive or a history of liver cirrhosis or portal hypertension; 9. Life-threatening bleeding (WHO bleeding score 4) or the patient is expected to require salvage treatment before the first dose; 10. Has a history of malignant tumor or is accompanied by malignant tumor; 11. The investigator believes that there are any other circumstances that may cause the subjects to fail to complete the study or bring obvious risks to the subjects.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Heng Mei, PhD
    Phone
    86-13886160811
    Ext
    8613986183871
    Email
    hmei@hust.edu.cn
    First Name & Middle Initial & Last Name or Official Title & Degree
    Min Xu, MD
    Phone
    86-13212794115
    Ext
    8613986183871
    Email
    xu_min1015@163.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Heng Mei, PhD
    Organizational Affiliation
    Wuhan Union Hospital, China
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    Yes
    IPD Sharing Plan Description
    the data will share to others if they not involve patient privacy
    IPD Sharing Time Frame
    the data will be shared after the study is completed, it will take 2 years.
    IPD Sharing Access Criteria
    Not sure yet
    Citations:
    PubMed Identifier
    10419881
    Citation
    Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood. 1999 Aug 1;94(3):909-13.
    Results Reference
    background
    PubMed Identifier
    25305203
    Citation
    Moulis G, Palmaro A, Montastruc JL, Godeau B, Lapeyre-Mestre M, Sailler L. Epidemiology of incident immune thrombocytopenia: a nationwide population-based study in France. Blood. 2014 Nov 20;124(22):3308-15. doi: 10.1182/blood-2014-05-578336. Epub 2014 Oct 10.
    Results Reference
    background
    PubMed Identifier
    7898229
    Citation
    Glynn RJ, Field TS, Rosner B, Hebert PR, Taylor JO, Hennekens CH. Evidence for a positive linear relation between blood pressure and mortality in elderly people. Lancet. 1995 Apr 1;345(8953):825-9. doi: 10.1016/s0140-6736(95)92964-9.
    Results Reference
    background
    PubMed Identifier
    31794604
    Citation
    Neunert C, Terrell DR, Arnold DM, Buchanan G, Cines DB, Cooper N, Cuker A, Despotovic JM, George JN, Grace RF, Kuhne T, Kuter DJ, Lim W, McCrae KR, Pruitt B, Shimanek H, Vesely SK. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv. 2019 Dec 10;3(23):3829-3866. doi: 10.1182/bloodadvances.2019000966. Erratum In: Blood Adv. 2020 Jan 28;4(2):252.
    Results Reference
    background
    PubMed Identifier
    1984800
    Citation
    Cortelazzo S, Finazzi G, Buelli M, Molteni A, Viero P, Barbui T. High risk of severe bleeding in aged patients with chronic idiopathic thrombocytopenic purpura. Blood. 1991 Jan 1;77(1):31-3.
    Results Reference
    background
    PubMed Identifier
    24942752
    Citation
    Ruggeri M, Tosetto A, Palandri F, Polverelli N, Mazzucconi MG, Santoro C, Gaidano G, Lunghi M, Zaja F, De Stefano V, Sartori R, Fazi P, Rodeghiero F; Gruppo Italiano Malattie EMatologiche dell'Adulto (GIMEMA) Anemia and Thrombocytopenias Working Party. GIMEMA Study ITP0311. Thrombotic risk in patients with primary immune thrombocytopenia is only mildly increased and explained by personal and treatment-related risk factors. J Thromb Haemost. 2014 Aug;12(8):1266-73. doi: 10.1111/jth.12636. Epub 2014 Jul 16.
    Results Reference
    background
    PubMed Identifier
    21956157
    Citation
    Michel M, Rauzy OB, Thoraval FR, Languille L, Khellaf M, Bierling P, Godeau B. Characteristics and outcome of immune thrombocytopenia in elderly: results from a single center case-controlled study. Am J Hematol. 2011 Dec;86(12):980-4. doi: 10.1002/ajh.22170. Epub 2011 Sep 28.
    Results Reference
    background
    PubMed Identifier
    18848179
    Citation
    Daou S, Federici L, Zimmer J, Maloisel F, Serraj K, Andres E. Idiopathic thrombocytopenic purpura in elderly patients: a study of 47 cases from a single reference center. Eur J Intern Med. 2008 Oct;19(6):447-51. doi: 10.1016/j.ejim.2007.07.006. Epub 2008 Feb 20.
    Results Reference
    background
    PubMed Identifier
    22956151
    Citation
    Zhou H, Fu R, Wang H, Zhou F, Li H, Zhou Z, Zhang L, Yang R. Immune thrombocytopenia in the elderly: clinical course in 525 patients from a single center in China. Ann Hematol. 2013 Jan;92(1):79-87. doi: 10.1007/s00277-012-1567-2. Epub 2012 Sep 6.
    Results Reference
    background

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    Efficacy and Safety of TPO Receptor Agonists in the Treatment of Elderly ITP Patients

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