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Trial record 2 of 554 for:    Thrombocytopenia: Clinical Trials

A Single-center Clinical Trial of Bortezomib in Management of Immune Thrombocytopenia (ITP) (ITP)

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ClinicalTrials.gov Identifier: NCT03013114
Recruitment Status : Not yet recruiting
First Posted : January 6, 2017
Last Update Posted : January 6, 2017
Sponsor:
Information provided by (Responsible Party):
Ming Hou, Shandong University

Brief Summary:

Primary immune thrombocytopenia (ITP) is a disorder caused by autoantibody-mediated platelet destruction and decreased platelet production. It has been reported that refractory ITP is closely related to long-lived plasma cells (PCs), which are resistant to glucocorticoids, conventional immunosuppressive and cytotoxic drugs, irradiation and B-cell depletion therapies.

Proteasome inhibition bortezomib is one of the most promising therapeutic approaches to target PCs, since this strategy has been shown to efficiently eliminate multiple myeloma cells, that is, transformed PCs. It also has been successfully used in SLE-like mice, experimental autoimmune MG rats and experimental hemophilia-A mice that develop anti-factor VIII antibodies in preclinical models by depleting both short-lived and long-lived PCs. Additionally, treatment with bortezomib resulted in a rapid clinical response in a patient with refractory thrombotic thrombocytopenic purpura associated with the depletion of inhibitory autoantibodies against ADAMTS13, a metalloproteinase that cleaves the von Wille-brand factor, which is produced by plasma cells. Hence, the elimination of autoreactive PCs by proteasome inhibitors might represent a new treatment strategy for autoantibody-mediated diseases.

To date, refractory ITP is lacking of effective treatments and these findings encouraged us to conduct a study of bortezomib in management of ITP with high anti-platelet antibodies level. Data from this study may provide some idea of bortezomib in the treatment of ITP.


Condition or disease Intervention/treatment Phase
Immune Thrombocytopenia Drug: Bortezomib Phase 2

Detailed Description:

The investigators are undertaking a single-center, single-arm study of 20 primary ITP adult patients from Shandong University Qilu Hospital

. All the participants are selected to receive bortezomib treatment (given intravenously at a dose of 1.3mg/m2 on days 1,4,8,11). Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 20 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Single-center Clinical Trial of Bortezomib in Management of Immune Thrombocytopenia (ITP) With High Anti-platelet Antibodies Level
Study Start Date : January 2017
Estimated Primary Completion Date : December 2017
Estimated Study Completion Date : December 2017


Arm Intervention/treatment
Experimental: Bortezomib
Bortezomib was given by intravenous bolus injection at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11, repeated every 21 days. It will be given four cycles.
Drug: Bortezomib
Bortezomib was given by intravenous bolus injection at a dose of 1.3 mg/m2 on days 1, 4, 8 and 11, repeated every 21 days. It will be given four cycles.




Primary Outcome Measures :
  1. Platelet counts [ Time Frame: an average of 3 months ]
    1. Complete response (CR): A platelet count ≥ 100 * 10^9/L measured on two occasions > 7 days apart and the absence of bleeding.
    2. Response (R): A platelet count ≥ 30 * 10^9/L and a greater than two fold increase in platelet count from baseline measured on two occasions > 7 days apart and the absence of bleeding.
    3. No response (NR): A platelet count < 30 * 10^9/L or a less than two fold increase in platelet count from baseline or the presence of bleeding. Platelet count must be measured on two occasions more than a day apart.


Secondary Outcome Measures :
  1. Numbers of Megakaryocyte Polyploidy [ Time Frame: 6 days after every treatment cycle, an average of 3 months ]
  2. Expression rate of long-lived plasma cells [ Time Frame: 6 days after every treatment cycle, an average of 3 months ]


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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • failure to achieve at least Response
  • need of treatment(s) (including, but not limited to, low dose of corticosteroids) to minimize the risk of clinically significant bleeding. Need of on-demand or adjunctive therapy alone does not qualify the patient as refractory
  • primary ITP confirmed by excluding other supervened causes of thrombocytopenia

Exclusion Criteria:

  • pregnancy
  • hypertension
  • cardiovascular disease
  • diabetes
  • liver and kidney function impairment
  • HCV, HIV, HBsAg seropositive status
  • patients with systemic lupus erythematosus and/or antiphospholipid syndrome

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03013114


Contacts
Contact: Ming Hou houming@medmail.com.cn

Locations
China, Shandong
Shandong University Qilu hospital Not yet recruiting
Jinan, Shandong, China, 250012
Contact: Hou Ming       houming@medmail.com.cn   
Sponsors and Collaborators
Shandong University
Investigators
Principal Investigator: Ming Hou Qilu hospital, Shandong University

Responsible Party: Ming Hou, Professor and Director, Shandong University
ClinicalTrials.gov Identifier: NCT03013114     History of Changes
Other Study ID Numbers: ITP-Bortezomib
First Posted: January 6, 2017    Key Record Dates
Last Update Posted: January 6, 2017
Last Verified: January 2017

Keywords provided by Ming Hou, Shandong University:
Bortezomib Immune thrombocytopenia

Additional relevant MeSH terms:
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Bortezomib
Antineoplastic Agents