Clinical trial NCT04949009: Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP)
Official title: Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP): a Single-arm, Multi-centre, Observational Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Primary Immune Thrombocytopenia
Keywords: Avatrombopag,Primary Immune Thrombocytopenia
Lead sponsor: Shandong University
Sponsor class: OTHER
First posted: 2021-07-02
Enrollment: 400
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: 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)"

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of participants achieving platelet response on day 28 of treatment
timeFrame: on day 28 of treatment
Secondary outcomes:
measure: Percentage of participants achieving platelet response on day 8 of treatment.
timeFrame: on day 8 of treatment.
measure: Percentage of participants achieving platelet response on day 14 of treatment.
timeFrame: on day 14 of treatment.
measure: Changes in participants' bleeding scores.
timeFrame: through study completion, an average of 6 months
measure: Evaluation of adverse effects related to avatrombopag.
timeFrame: through study completion, an average of 6 months
Other outcomes:
measure: Percentage of participants achieving platelet response (R) at the second month of treatment.
timeFrame: at the second month of treatment
measure: Participants' 6-month continuous remission rate.
timeFrame: 6 month of treatment
Locations:
city: Jinan
country: China
facility: Qilu Hospital, Shandong University
state: Shandong
Study officials:
affiliation: Shandong University Qilu Hospital
name: Ming Hou, MD PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Dlugosz-Danecka M, Zdziarska J, Jurczak W. Avatrombopag for the treatment of immune thrombocytopenia. Expert Rev Clin Immunol. 2019 Apr;15(4):327-339. doi: 10.1080/1744666X.2019.1587294. Epub 2019 Mar 8.
citation: Shirley M. Avatrombopag: First Global Approval. Drugs. 2018 Jul;78(11):1163-1168. doi: 10.1007/s40265-018-0949-8.
citation: Cheloff AZ, Al-Samkari H. Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. J Blood Med. 2019 Sep 5;10:313-321. doi: 10.2147/JBM.S191790. eCollection 2019.
citation: Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. doi: 10.1182/blood-2013-07-514398. Epub 2014 May 6.