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 : Completed
First Posted : December 7, 2017
Last Update Posted : July 6, 2021
|Condition or disease||Intervention/treatment||Phase|
|Pure Red Cell Aplasia||Drug: Sirolimus||Phase 4|
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 :||Interventional (Clinical Trial)|
|Actual Enrollment :||64 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
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.
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
- Hemoglobin level [ Time Frame: 6 months ]Hemoglobin level in g/L
- Hemoglobin level [ Time Frame: 2 years ]Hemoglobin level in g/L
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
|Peking Union Medical College Hospital|
|Beijing, Beijing, China, 100730|
|Principal Investigator:||Bing B Han, PhD.||Peking Union Medical College Hospital|