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
About this trial
This is an interventional other trial for Primary Immune Thrombocytopenia focused on measuring hetrombopag, Primary Immune Thrombocytopenia, platelets
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.
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
NCT ID
NCT05311930
First Posted
March 28, 2022
Last Updated
March 28, 2022
Sponsor
Wuhan Union Hospital, China
Collaborators
Jiangsu Hengrui Pharmaceutical Co., Ltd.
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
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Efficacy and Safety of TPO Receptor Agonists in the Treatment of Elderly ITP Patients
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