Avatrombopag in the Treatment of Primary Immune Thrombocytopenia(ITP)

NCT04949009 | Unknown DMC FDA Drug

• 1 other identifier: SKX-2007
• Study Type: observational
• Target: 400
• Locations: 1 country
• Sites: 1

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.

Conditions

Primary Immune Thrombocytopenia

Keywords

Avatrombopag,Primary Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (4)

• Percentage of participants achieving platelet response on day 8 of treatment.

  on day 8 of treatment.

• Percentage of participants achieving platelet response on day 14 of treatment.

  on day 14 of treatment.

• Changes in participants' bleeding scores.

  through study completion, an average of 6 months

• Evaluation of adverse effects related to avatrombopag.

  through study completion, an average of 6 months

Other Outcomes (2)

• Percentage of participants achieving platelet response (R) at the second month of treatment.

  at the second month of treatment

• Participants' 6-month continuous remission rate.

  6 month of treatment

Study Arms (1)

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

Interventions

Avatrombopag DRUG

Avatrombopag is an orally bioavailable, small molecule thrombopoietin receptor agonist that has been developed by Dova Pharmaceuticals for the treatment of thrombocytopenic disorders.

Also known as: Doptelet

Patients with primary immune thrombocytopenia (ITP)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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)"

You may qualify if:

• Aged 18+ years, male or female;
• 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)"
• 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.
• Spleen is generally not enlarged.
• Bone marrow examination: the morphological characteristics of bone marrow cells in ITP patients were increased or normal megakaryocytes with maturation disorders.
• 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;
• ECOG general status score ≤ 2;
• 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.
• Voluntarily signed the informed consent.
• Any other circumstances that the investigator considers appropriate for the patient to participate in the study.

You may not qualify if:

• Patients with secondary thrombocytopenia.
• Currently receiving other TPO-RAs, rhIL-11 and rhTPO treatment, and subjects are unwilling to switch to avatrombopag treatment.
• Patients with severe insufficiency of heart, lung, liver and kidney.
• Pregnant or breast-feeding, or contraceptive measures cannot be taken during the trial.
• Subjects participated in clinical studies of other investigational drugs or devices within 30 days prior to screening.
• Having a history of psychotropic drug abuse and unable to quit or having mental disorders.
• Having significant factors that affect the absorption of oral drugs, such as inability to swallow, chronic diarrhea and intestinal obstruction, etc.
• Subject is allergic to avatrombopag or any of its excipients;
• Any other circumstances that the investigator considers inappropriate for the patient to participate in the study.

Sponsors & Collaborators

• Shandong University: lead

Study Sites (1)

Qilu Hospital, Shandong University

Jinan, Shandong, China

RECRUITING

Related Publications (4)

• 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.

  PMID: 30799645 BACKGROUND

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

  PMID: 29995177 BACKGROUND

• 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.

  PMID: 31565009 BACKGROUND

• 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.

  PMID: 24802775 BACKGROUND

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Ming Hou, MD PhD

  Shandong University Qilu Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ming Hou, MD PhD

CONTACT

86-531-82169114; houming@medmail.com.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Proff

Study Record Dates

• First Submitted: May 13, 2021
• First Posted: July 2, 2021
• Study Start: March 1, 2021
• Primary Completion: October 1, 2021
• Study Completion: March 1, 2022
• Last Updated: July 2, 2021

Record last verified: 2021-06

Locations

CN (1)