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Sirolimus Treatment for Refractory PRCA

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ClinicalTrials.gov Identifier: NCT03364764
Recruitment Status : Completed
First Posted : December 7, 2017
Last Update Posted : July 6, 2021
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 sirolimus for refractory PRCA.

Condition or disease Intervention/treatment Phase
Pure Red Cell Aplasia Drug: Sirolimus 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.

Sirolimus (rapamycin) is an agent produced by the bacterium Streptomyces hygroscopicus, inhibits the mammalian target of rapamycin (mTOR). mTOR is a serine/threonine kinase that regulates cell growth, proliferation, metabolism and survival in eukaryotic cells, and is identified as two interacting complex, mTORC1 and mTORC2. Sirolimus primarily inhibits mTORC1, has been approved for prevent organ transplant rejection, especially in renal transplantation, sirolimus also promises to treat autoimmune, degenerative and hyperproliferative disorders. Recently, sirolimus 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 sirolimus on refractory PRCA so far.

In this study, It is anticipate to evaluate the effect of sirolimus on 30 patients with refractory PRCA, the side-effects was documented and plasma concentration of sirolimus will be monitor.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 64 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Sirolimus Treatment for Refractory Pure Red Cell Aplasia, a Prospective Study
Actual Study Start Date : February 1, 2018
Actual Primary Completion Date : February 1, 2019
Actual Study Completion Date : May 1, 2021

Resource links provided by the National Library of Medicine

Drug Information available for: Sirolimus

Arm Intervention/treatment
Experimental: efficiency of sirolimus on PRCA
A prospective research of the sirolimus efficiency on refractory PRCA patients On refractory PRCA patients, sirolimus was tried. Dosage: 2mg QD for the first day, then 1 mg QD. Medication time should last at least 6 months.
Drug: Sirolimus
On refractory PRCA patients, sirolimus was tried. Dosage: 2mg QD for the first day, then 1 mg QD. Medication time should last at least 6 months.
Other Name: Sirolimus Tablets

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:   Yes

Inclusion Criteria:

  1. Refractory pure red cell aplasia.
  2. Excluding other diseases which might cause hematological abnormalities.
  3. No response or intolerant to first and second line therapies.
  4. 18-80 years old.
  5. All subjects must: agree not to donate blood or be counseled about pregnancy precautions and risks of fetal exposure.
  6. Written informed consent.

Exclusion Criteria:

  1. NOT refractory pure red cell anemia.
  2. Response and well tolerate to first or second line therapy.
  3. Patients who are under 18-year-old or over 80-year-old.
  4. Pregnant or lactating.
  5. 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): NCT03364764

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China, Beijing
Peking Union Medical College Hospital
Beijing, Beijing, China, 100730
Sponsors and Collaborators
Bing Han
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Principal Investigator: Bing B Han, PhD. 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: NCT03364764    
Other Study ID Numbers: PRCA-1
First Posted: December 7, 2017    Key Record Dates
Last Update Posted: July 6, 2021
Last Verified: June 2020
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:
refractory pure red cell aplasia
prospective study
Additional relevant MeSH terms:
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Red-Cell Aplasia, Pure
Hematologic Diseases
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs