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Tacrolimus Treatment for Refractory PRCA

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ClinicalTrials.gov Identifier: NCT03540472
Recruitment Status : Unknown
Verified May 2018 by Bing Han, Peking Union Medical College Hospital.
Recruitment status was:  Recruiting
First Posted : May 30, 2018
Last Update Posted : May 30, 2018
Sponsor:
Information provided by (Responsible Party):
Bing Han, Peking Union Medical College Hospital

Brief Summary:
Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of tacrolimus for refractory PRCA.

Condition or disease Intervention/treatment Phase
Pure Red Cell Aplasia Drug: tacrolimus Phase 4

Detailed Description:
Pure red cell aplasia (PRCA) is a rare normocytic normochromic anemia with reticulocytopenia, characterized by a reduction of erythroid precursors from the bone marrow, could be divided into congenital and acquired PRCA according to pathogenesis. Congenital PRCA, also known as Diamond-Blackfan syndrome, has been associated with pathogenic variant in GATA1 and TSR2 and gene encode ribosomal proteins. Acquired PRCA can be a primary disease which is usually mediated by immunology, or secondary to other diseases, such as lymphoproliferative diseases, autoimmune diseases, thymoma, infection, or drugs. The first line therapy of acquired PRCA is Cyclosporine A and steroids, the second line therapy are anti-CD20, ATG, immunosuppressive drugs like cyclophosphamide, bone marrow transplantation. Unfortunately, some patients did not response or tolerate the above treatments. Tacrolimus, also known as FK506, is an agent mainly used after allogeneic organ transplant to lower risk of organ rejection. Tacrolimus could inhibit the production the production of IL-2, and also used in the therapy of other T cell mediated diseases. Tacrolimus primarily has been approved for prevent organ transplant rejection, especially in renal transplantation, tacrolimus also promises to treat autoimmune, degenerative and hyperproliferative disorders. Recently, tacrolimus has been reported to be effective and well tolerated for many immune-mediated cytopenias, such as autoimmune lymphoproliferative syndrome, immune thrombocytopenia, EVANS syndrome, etc. However, due to the rare occurrence of PRCA and good response rate to cyclosporine, there are very few studies of tacrolimus on refractory PRCA so far. In this study, it is anticipate to evaluate the effect of tacrolimus on 30 patients with refractory PRCA, the side-effects was documented and plasma concentration of tacrolimus will be monitor.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Tacrolimus Treatment for Refractory Pure Red Cell Aplasia, a Prospective Study
Estimated Study Start Date : June 10, 2018
Estimated Primary Completion Date : December 10, 2018
Estimated Study Completion Date : June 10, 2020

Resource links provided by the National Library of Medicine

Drug Information available for: Tacrolimus

Arm Intervention/treatment
Experimental: efficiency of tacrolimus on PRCA

A prospective research of the tacrolimus efficiency on refractory PRCA patients On refractory PRCA patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study.

Medication time should last at least 6 months.

Drug: tacrolimus
On refractory PRCA patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study
Other Name: tacrolimus capsule




Primary Outcome Measures :
  1. Hemoglobin level [ Time Frame: 6 months ]
    Hemoglobin level in g/L


Secondary Outcome Measures :
  1. Hemoglobin level [ Time Frame: 2 years ]
    Hemoglobin level in g/L



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Refractory pure red cell aplasia. 18-80 years old. No response or intolerant to first and second line therapies. Written informed consent.

Exclusion Criteria:

Other diseases which might cause hematological abnormalities. Response and well tolerate to first or second line therapy. Patients who are under 18-year-old or over 80-year-old. Pregnant or lactating. Patients unwilling to or unable to comply with the protocol.


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): NCT03540472


Contacts
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Contact: Zhangbiao Long, M.D. +86 13011826728 longzhangbiao@163.com
Contact: Hongmin Li, M.D. +86 15811152096 hongmin_li@163.com

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Zhangbiao Long, M.D.    +86 13011826728    longzhangbiao@163.com   
Sponsors and Collaborators
Peking Union Medical College Hospital
Publications of Results:
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Responsible Party: Bing Han, Professor, Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03540472    
Other Study ID Numbers: tacrolimus-001
First Posted: May 30, 2018    Key Record Dates
Last Update Posted: May 30, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Bing Han, Peking Union Medical College Hospital:
tacrolimus
refractory pure red cell aplasia
prospective study
Additional relevant MeSH terms:
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Red-Cell Aplasia, Pure
Anemia
Hematologic Diseases
Tacrolimus
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Calcineurin Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action