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Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone for Subjects With Idiopathic Thrombocytopenic Purpura

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03830749
Recruitment Status : Recruiting
First Posted : February 5, 2019
Last Update Posted : February 17, 2020
Sponsor:
Information provided by (Responsible Party):
Humanity & Health Medical Group Limited

Brief Summary:
Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Diagnosis of ITP is established according to the American Society of Hematology guidelines. Eligible ITP subjects have platelet counts <30×109/L or counts <50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions. Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint is prolonged response rate which was defined as the proportion of enrolled subjects maintaining platelet counts >50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy.

Condition or disease Intervention/treatment Phase
Immune Thrombocytopenia Drug: Eltrombopag Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Study to Evaluate the Safety and Efficacy of Eltrombopag Plus Pulsed Dexamethasone as First Line Therapy for Subjects With Idiopathic Thrombocytopenic Purpura (ITP)
Actual Study Start Date : July 1, 2018
Estimated Primary Completion Date : December 31, 2020
Estimated Study Completion Date : March 31, 2021


Arm Intervention/treatment
Experimental: Single Arm
Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone
Drug: Eltrombopag
Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone




Primary Outcome Measures :
  1. The prolonged response rate [ Time Frame: 6 months after completion of therapy ]
    Defined as proportion of patients with platelet counts > 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy


Secondary Outcome Measures :
  1. Time to relapse [ Time Frame: 6 months after completion of therapy ]
    Defined as the interval from completion of 3 months treatment to platelet count<30,000/ul and restarting of ITP therapy including but not limited to platelet transfusion, IVIG, corticosteroids, immunosuppressive drugs, rituximab

  2. Early response rate [ Time Frame: 6 months after completion of therapy ]
    Defined as proportion of patients requiring less than 3 courses of pulsed dexamethasone to maintain platelet counts > 150,000/ul

  3. Health related quality of life [ Time Frame: 6 months after completion of therapy ]
    Assessed by SV36 HRQoL questionnaires



Information from the National Library of Medicine

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

Inclusion Criteria:

  1. Subject has signed and dated a written informed consent.
  2. Adults (≥18 years) diagnosed with ITP according to the American Society for Hematology/British Committee for Standards in Haematology (ASH/BCSH) Guidelines. In addition, the peripheral blood smear should support the diagnosis of ITP with no evidence of other disease causative of thrombocytopenia (e.g., pseudo thrombocytopenia, myelofibrosis). The physical examination should be normal or at least not show signs suggestive of any disease likely to be associated with thrombocytopenia.
  3. No prior ITP treatment except platelet transfusions
  4. Subject has no intercurrent medical event, including evidence of any thrombosis.
  5. Normal prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT), no history of hypercoagulable state.
  6. The following clinical chemistries must be within the normal reference range:

    creatinine, ALT, AST, total bilirubin, total albumin and alkaline phosphatase.

  7. Subject is practicing an acceptable method of contraception (documented in chart).
  8. Subject is able to understand and comply with protocol requirements and instructions.

Exclusion Criteria:

  1. Any clinically relevant abnormality, other than ITP, identified on the screening examination, or any other medical condition or circumstance, which in the opinion of the investigator makes the subject unsuitable for participation in the study or suggests another diagnosis.
  2. History of active malignancy or on cancer therapy. Patients with a history of malignancy in complete remission for longer than 5 years are eligible
  3. History of arterial or venous thrombosis (stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism).
  4. ≥ two of the following risk factors: Factor V Leiden, hormone replacement therapy, systemic contraception containing estrogen, smoking, diabetes, hypercholesterolemia, medications for hypertension or cancer.
  5. Pre-existing cardiac disease (including congestive heart failure, and arrhythmia requiring treatment), or clinically significant findings on resting 12-lead ECG at screening.
  6. Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic gonadotrophin (β-hCG) pregnancy test) at screening or pre-dose on Day 1.
  7. History of alcohol/drug abuse.
  8. Treatment with an investigational drug within 30 days or five half-lives (whichever is longer) preceding the first dose of study medication

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03830749


Contacts
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Contact: Gregory Cheng, MD, PhD +852-28613777 gregory.cheng@hnhmgl.com
Contact: Danny Wang, PhD +852-28613777 danny.wang@hnhmgl.com

Locations
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Hong Kong
Humanity & Health Research Centre Recruiting
Hong Kong, Hong Kong SAR, Hong Kong
Contact: Danny Wang    852-28613777    danny.wang@hnhmgl.com   
Sponsors and Collaborators
Humanity & Health Medical Group Limited
Investigators
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Principal Investigator: Gregory Cheng, PhD, MD Humanity & Health Research Centre

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Responsible Party: Humanity & Health Medical Group Limited
ClinicalTrials.gov Identifier: NCT03830749    
Other Study ID Numbers: HHRC_ITP
First Posted: February 5, 2019    Key Record Dates
Last Update Posted: February 17, 2020
Last Verified: February 2020

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Dexamethasone
Dexamethasone acetate
BB 1101
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Protease Inhibitors