Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP)

Sponsor
Shandong University (Other)
Overall Status
Recruiting
CT.gov ID
NCT04949009
Collaborator
(none)
400
1
12
33.4

Study Details

Study Description

Brief Summary

The project was undertaking by Qilu Hospital of Shandong University and other well-known hospitals in China. Aims at evaluating effectiveness and safety of avatrombopag in the treatment of primary immune thrombocytopenia.

Condition or Disease Intervention/Treatment Phase

Detailed Description

The investigators are undertakingSingle-arm, multi-Centre, Observational study of 400 Patients with primary immune thrombocytopenia (ITP) from Qilu Hospital of Shandong University and other well-known hospitals in China. ITP patients will be given one tablet (20 mg) of avatrombopag daily.If the platelet count is higher than 150×109/L, the dose should be reduced. Prolonged dosing intervals or a combination of reduced daily dose are preferred.If the drug was taken for ≥1 week and the platelet count was still less than 30×109/L, the dosage should be increased. The maximum dose is 40 mg daily. Aplatelet count,AE, laboratory parameters, ECG, vital signs and other symptoms will be evaluated before and after treatment. Adverse events will be recorded throughout the study.

Study Design

Study Type:
Observational
Anticipated Enrollment :
400 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP): a Single-arm, Multi-centre, Observational Study
Actual Study Start Date :
Mar 1, 2021
Anticipated Primary Completion Date :
Oct 1, 2021
Anticipated Study Completion Date :
Mar 1, 2022

Arms and Interventions

Arm Intervention/Treatment
Patients with primary immune thrombocytopenia (ITP)

Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"

Drug: Avatrombopag
Avatrombopag is an orally bioavailable, small molecule thrombopoietin receptor agonist that has been developed by Dova Pharmaceuticals for the treatment of thrombocytopenic disorders.
Other Names:
  • Doptelet
  • Outcome Measures

    Primary Outcome Measures

    1. Percentage of participants achieving platelet response on day 28 of treatment [on day 28 of treatment]

      Percentage of participants achieving platelet response on day 28 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.

    Secondary Outcome Measures

    1. Percentage of participants achieving platelet response on day 8 of treatment. [on day 8 of treatment.]

      Percentage of participants achieving platelet response on day 8 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.

    2. Percentage of participants achieving platelet response on day 14 of treatment. [on day 14 of treatment.]

      Percentage of participants achieving platelet response on day 14 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy.

    3. Changes in participants' bleeding scores. [through study completion, an average of 6 months]

      Thrombocytopenia bleeding scoring system is used to evaluate the changes in participants' bleeding scores.

    4. Evaluation of adverse effects related to avatrombopag. [through study completion, an average of 6 months]

      Evaluation of adverse effects related to avatrombopag including side effect,toxic reaction,post effect,anaphylactic reaction.

    Other Outcome Measures

    1. Percentage of participants achieving platelet response (R) at the second month of treatment. [at the second month of treatment]

      Percentage of participants achieving the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy at the second month of treatment.

    2. Participants' 6-month continuous remission rate. [6 month of treatment]

      Percentage of participants achieving 6-month continuous remission including symptoms associated with bleeding and mental symptoms

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    1. Aged 18+ years, male or female;

    2. Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"

    3. At least 2 consecutive blood tests showed a decrease in platelet count; no obvious abnormality in the morphology of blood cells by peripheral blood smear microscopy.

    4. Spleen is generally not enlarged.

    5. Bone marrow examination: the morphological characteristics of bone marrow cells in ITP patients were increased or normal megakaryocytes with maturation disorders.

    6. Other secondary thrombocytopenia must be excluded: autoimmune diseases, thyroid disease, lymphoproliferative disorders, myelodysplastic syndrome (MDS), aplastic anemia (AA), various malignant hematologic diseases, tumor infiltration, Chronic liver disease, hypersplenism, common variant immunodeficiency disease (CVID), infection, vaccination causing secondary thrombocytopenia; Thrombocytopenia due to depletion; Drug induced thrombocytopenia; Alloimmune thrombocytopenia; Thrombocytopenia during pregnancy; Congenital thrombocytopenia and pseudo-thrombocytopenia;

    7. ECOG general status score ≤ 2;

    8. Platelet count < 30×109/L;platelet count ≥< 30×109/L accompanied by active bleeding; If the platelet count is around 30×10^9/L and no active bleeding, a second examination must be performed to further confirm the platelet count.

    9. Voluntarily signed the informed consent.

    10. Any other circumstances that the investigator considers appropriate for the patient to participate in the study.

    Exclusion Criteria:
    1. Patients with secondary thrombocytopenia.

    2. Currently receiving other TPO-RAs, rhIL-11 and rhTPO treatment, and subjects are unwilling to switch to avatrombopag treatment.

    3. Patients with severe insufficiency of heart, lung, liver and kidney.

    4. Pregnant or breast-feeding, or contraceptive measures cannot be taken during the trial.

    5. Subjects participated in clinical studies of other investigational drugs or devices within 30 days prior to screening.

    6. Having a history of psychotropic drug abuse and unable to quit or having mental disorders.

    7. Having significant factors that affect the absorption of oral drugs, such as inability to swallow, chronic diarrhea and intestinal obstruction, etc.

    8. Subject is allergic to avatrombopag or any of its excipients;

    9. Any other circumstances that the investigator considers inappropriate for the patient to participate in the study.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Qilu Hospital, Shandong University Jinan Shandong China

    Sponsors and Collaborators

    • Shandong University

    Investigators

    • Principal Investigator: Ming Hou, MD PhD, Shandong University Qilu Hospital

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    Responsible Party:
    Ming Hou, Proff, Shandong University
    ClinicalTrials.gov Identifier:
    NCT04949009
    Other Study ID Numbers:
    • SKX-2007
    First Posted:
    Jul 2, 2021
    Last Update Posted:
    Jul 2, 2021
    Last Verified:
    Jun 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by Ming Hou, Proff, Shandong University
    Additional relevant MeSH terms:

    Study Results

    No Results Posted as of Jul 2, 2021