Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP)

Primary Immune Thrombocytopenia Clinical Trial

Official title:

Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP): a Single-arm, Multi-centre, Observational Study

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04949009
• Other study ID #: SKX-2007
• Status: Recruiting
• Start date: March 1, 2021
• Est. completion date: March 2022

Study information

• Verified date: June 2021
• Source: Shandong University
• Contact: Ming Hou, MD PhD
• Phone: 86-531-82169114
• Email: houming[@]medmail.com.cn
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 400
• Est. completion date: March 2022
• Est. primary completion date: October 1, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Primary Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

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.

Locations

Country	Name	City	State
China	Qilu Hospital, Shandong University	Jinan	Shandong

Sponsors (1)

Lead Sponsor	Collaborator
Shandong University

Country where clinical trial is conducted

China, 

References & Publications (4)

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. — View 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. Review. — View 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. Review. — View Citation

Shirley M. Avatrombopag: First Global Approval. Drugs. 2018 Jul;78(11):1163-1168. doi: 10.1007/s40265-018-0949-8. Review. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Percentage of participants achieving platelet response (R) 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.	at the second month of treatment
Other	Participants' 6-month continuous remission rate.	Percentage of participants achieving 6-month continuous remission including symptoms associated with bleeding and mental symptoms	6 month of treatment
Primary	Percentage of participants achieving platelet response 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.	on day 28 of treatment
Secondary	Percentage of participants achieving platelet response 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.	on day 8 of treatment.
Secondary	Percentage of participants achieving platelet response 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.	on day 14 of treatment.
Secondary	Changes in participants' bleeding scores.	Thrombocytopenia bleeding scoring system is used to evaluate the changes in participants' bleeding scores.	through study completion, an average of 6 months
Secondary	Evaluation of adverse effects related to avatrombopag.	Evaluation of adverse effects related to avatrombopag including side effect,toxic reaction,post effect,anaphylactic reaction.	through study completion, an average of 6 months