Pure Red Cell Aplasia Clinical Trial
Official title:
Sirolimus Treatment for Refractory Pure Red Cell Aplasia, a Prospective Study
NCT number | NCT03364764 |
Other study ID # | PRCA-1 |
Secondary ID | |
Status | Completed |
Phase | Phase 4 |
First received | |
Last updated | |
Start date | February 1, 2018 |
Est. completion date | May 1, 2021 |
Verified date | June 2020 |
Source | Peking Union Medical College Hospital |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 64 |
Est. completion date | May 1, 2021 |
Est. primary completion date | February 1, 2019 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 18 Years to 80 Years |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
China | Peking Union Medical College Hospital | Beijing | Beijing |
Lead Sponsor | Collaborator |
---|---|
Bing Han |
China,
Bride KL, Vincent T, Smith-Whitley K, Lambert MP, Bleesing JJ, Seif AE, Manno CS, Casper J, Grupp SA, Teachey DT. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. Blood. 2016 Jan 7;12 — View Citation
Li J, Wang Z, Dai L, Cao L, Su J, Zhu M, Yu Z, Bai X, Ruan C. Effects of rapamycin combined with low dose prednisone in patients with chronic immune thrombocytopenia. Clin Dev Immunol. 2013;2013:548085. doi: 10.1155/2013/548085. Epub 2013 Dec 2. — View Citation
Means RT Jr. Pure red cell aplasia. Blood. 2016 Nov 24;128(21):2504-2509. Review. — View Citation
Teachey DT, Greiner R, Seif A, Attiyeh E, Bleesing J, Choi J, Manno C, Rappaport E, Schwabe D, Sheen C, Sullivan KE, Zhuang H, Wechsler DS, Grupp SA. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative sy — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Hemoglobin level | Hemoglobin level in g/L | 6 months | |
Secondary | Hemoglobin level | Hemoglobin level in g/L | 2 years |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT00004143 -
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
|
Phase 2 | |
Terminated |
NCT03966053 -
The Use of Trifluoperazine in Transfusion Dependent DBA
|
Phase 1/Phase 2 | |
Recruiting |
NCT06065852 -
National Registry of Rare Kidney Diseases
|
||
Terminated |
NCT00001962 -
A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure
|
Phase 2 | |
Recruiting |
NCT03918265 -
Tacrolimus Treatment for Refractory Autoimmune Cytopenia
|
Phase 4 | |
Active, not recruiting |
NCT00006055 -
Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases
|
N/A | |
Completed |
NCT01362595 -
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
|
Phase 1/Phase 2 | |
Recruiting |
NCT03540472 -
Tacrolimus Treatment for Refractory PRCA
|
Phase 4 | |
Recruiting |
NCT03214354 -
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
|
Phase 2 | |
Completed |
NCT00314795 -
Efficacy and Safety of Peginesatide (AF37702) in the Treatment of Anemia in Participants With Chronic Kidney Disease
|
Phase 2 |