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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
Sponsor:
Information provided by (Responsible Party):
Bing Han, Peking Union Medical College Hospital

Tracking Information
First Submitted Date  ICMJE November 21, 2017
First Posted Date  ICMJE December 7, 2017
Last Update Posted Date July 6, 2021
Actual Study Start Date  ICMJE February 1, 2018
Actual Primary Completion Date February 1, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 1, 2017)
Hemoglobin level [ Time Frame: 6 months ]
Hemoglobin level in g/L
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 1, 2017)
Hemoglobin level [ Time Frame: 2 years ]
Hemoglobin level in g/L
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Sirolimus Treatment for Refractory PRCA
Official Title  ICMJE Sirolimus Treatment for Refractory Pure Red Cell Aplasia, a Prospective Study
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.
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Pure Red Cell Aplasia
Intervention  ICMJE 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
Study Arms  ICMJE 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.
Intervention: Drug: Sirolimus
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 1, 2021)
64
Original Estimated Enrollment  ICMJE
 (submitted: December 1, 2017)
30
Actual Study Completion Date  ICMJE May 1, 2021
Actual Primary Completion Date February 1, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE China
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03364764
Other Study ID Numbers  ICMJE PRCA-1
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Bing Han, Peking Union Medical College Hospital
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Bing Han
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Bing B Han, PhD. Peking Union Medical College Hospital
PRS Account Peking Union Medical College Hospital
Verification Date June 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP