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Sirolimus Treatment for Refractory PRCA

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03364764
Recruitment Status : Unknown
Verified November 2017 by Bing Han, Peking Union Medical College Hospital.
Recruitment status was:  Recruiting
First Posted : December 7, 2017
Last Update Posted : December 7, 2017
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 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)
Estimated Enrollment : 30 participants
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
Estimated Study Start Date : January 2018
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : February 2020

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
Criteria

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


Contacts
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Contact: Yali Y Du, Master +8615845992396 yali_crazy@126.com
Contact: Zhangbiao Z Long, Doctor +8613011826728 longzhangbiao@163.com

Locations
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China, Beijing
Peking Union Medical College Hospital Recruiting
Beijing, Beijing, China, 100730
Contact: Yali Y Du, master    +8618910575941    yali_crazy@126.com   
Sponsors and Collaborators
Bing Han
Investigators
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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: December 7, 2017
Last Verified: November 2017
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:
sirolimus
refractory pure red cell aplasia
prospective study
Additional relevant MeSH terms:
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Red-Cell Aplasia, Pure
Anemia
Hematologic Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs