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

NCT05311930 | Unknown Phase 4

• 1 other identifier: TPO-RAs-old patients
• Study Type: interventional
• Target: 69
• Locations: 0 countries
• Sites: N/A

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

Conditions

Primary Immune Thrombocytopenia

Keywords

hetrombopag; Primary Immune Thrombocytopenia; platelets

Outcome Measures

Primary Outcomes (1)

• Treatment response

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

  28 days

Secondary Outcomes (6)

• Remission rate

  28 days

• Drug efficacy

  28 days

• Evaluation of effectiveness

  28 days

• Adverse events

  6 months from treatment

• Adverse events

  6 months from treatment

Study Arms (1)

2.5mg/d

EXPERIMENTAL

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;

Drug: 2.5mg/d Hetrombopag

Interventions

2.5mg/d Hetrombopag DRUG

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;

Also known as: no use

2.5mg/d

Eligibility Criteria

• Age: 60 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. 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.

You may not qualify if:

• \. 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.

Sponsors & Collaborators

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: lead
• Jiangsu Hengrui Pharmaceutical Co., Ltd.: collaborator

Related Publications (9)

• Frederiksen H, Schmidt K. The incidence of idiopathic thrombocytopenic purpura in adults increases with age. Blood. 1999 Aug 1;94(3):909-13.

  PMID: 10419881 BACKGROUND

• 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.

  PMID: 25305203 BACKGROUND

• 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.

  PMID: 7898229 BACKGROUND

• 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.

  PMID: 31794604 BACKGROUND

• 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.

  PMID: 1984800 BACKGROUND

• 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.

  PMID: 24942752 BACKGROUND

• 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.

  PMID: 21956157 BACKGROUND

• 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.

  PMID: 18848179 BACKGROUND

• 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.

  PMID: 22956151 BACKGROUND

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

hetrombopag

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

Study Officials

• Heng Mei, PhD

  Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Heng Mei, PhD

CONTACT

86-13886160811; hmei@hust.edu.cn

Min Xu, MD

CONTACT

86-13212794115; xu_min1015@163.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: March 28, 2022
• First Posted: April 5, 2022
• Study Start: May 1, 2022
• Primary Completion: December 30, 2024
• Study Completion: December 30, 2024
• Last Updated: April 5, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: the data will be shared after the study is completed, it will take 2 years.
• Access Criteria: Not sure yet