Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP)

• Conditions: Primary Immune Thrombocytopenia
• Interventions: Drug: Avatrombopag

• Registration Number: NCT04949009
• Lead Sponsor: Shandong University

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.

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 150×10\^9/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 30×10\^9/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 Timeline

• Study Start: 2021-03-01
• Study First Submitted: 2021-05-13
• Status Verified: 2021-06
• Study First Submitted QC: 2021-06-24
• Last Update Submitted: 2021-06-24
• Study First Posted: 2021-07-02
• Last Update Posted: 2021-07-02
• Primary Completion: 2021-10-01
• Study Completion: 2022-03

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 400

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)"

   1. 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.
   2. Spleen is generally not enlarged.
   3. Bone marrow examination: the morphological characteristics of bone marrow cells in ITP patients were increased or normal megakaryocytes with maturation disorders.
   4. 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;

3. ECOG general status score ≤ 2;

4. Platelet count < 30×10^9/L；platelet count ≥< 30×10^9/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.

5. Voluntarily signed the informed consent.

6. 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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with primary immune thrombocytopenia (ITP)	Avatrombopag	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)"

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
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.
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.
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.
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.

Trial Locations

Locations (1)

Qilu Hospital, Shandong University; 🇨🇳 Jinan, Shandong, China