Brief Title
ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia
Official Title
Multi-center Clinical Study of Immunosuppressants, Cyclophosphamide, And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia
Brief Summary
To assess whether ATG Combined With Cyclophosphamide and cord blood infusion can accelerate hematopoietic reconstruction in severe aplastic anemia patients and improve clinical curative effect and safety
Detailed Description
Aplastic Anemia( AA), is a set of bone marrow hematopoietic dysfunction caused by a variety of causes, with hyperplasia of bone marrow hematopoietic cells to reduce whole blood cells and peripheral blood at the characteristics of clinical main performance for anemia, bleeding and infection. According to the severity of the bone marrow failure and the progress of the clinical course ,it is divided into Severe Aplastic Anemia (SAA) and the Non - Severe Aplastic Anemia (NSAA).Severe Aplastic Anemia can be divided into two categories: Very Severe Aplastic Anemia (VSAA) and Severe Aplastic Anemia (SAA), with the characteristics of rapid progress, refractory, poor prognosis, high mortality .The natural course is six months or so, and most patients die in a year . Hematopoietic stem cell transplantation and immunosuppressive therapy are two main treatment . The former is by far the only possible cure. It is recommended as first-line treatments, if patients have a matched sibling donor. The recommended age limit is 40 years old. But for those who have no sibling donor or patients older than 40 years old, it is recommend the immunosuppressive therapy. The investigators have already summarized the effectiveness of rabbit antithymocyte immunoglobulin (ATG), cyclophosphamide (Cy) and cyclosporine, A (CsA) and the combination of the umbilical cord blood infusion for SAA/VSAA patients without suitable donor, with short duration, without long-term immunosuppressive therapy history. The total effectiveness rates has improved to 88%, with shorter immunosuppressive maintaining therapy , rapid hematopoietic reconstruction, fewer complications. The aim of this study is to further explore whether this solution can accelerate hematopoietic reconstruction of SAA/VSAA patients and its clinical curative effect and security. This study scheme has been approved by the Jinan military region general hospital medical ethics committee.
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
The total response rate
Secondary Outcome
Neutrophil recovery time
Condition
Aplastic Anemia
Intervention
Rabbit ATG, (Genzyme)
Study Arms / Comparison Groups
ATG, Cy and cord blood transfusion group
Description: ATG 3mg/kg/d for 5 days Cy 50mg/kg/d for 2 days CSA Started from 5mg/kg/d and adjusted to maintain trough serum concentration of 200-400ng/ml One unit of cord blood having no more than 3 HLA-A,B and DRB1 mismatches is transfused 24h after last dose of ATG administration. Intervention: Drug: Rabbit ATG, Thymoglobuline (Genzyme) plus Cyclophosphamide plus CSA Biological: Cord blood transfusion
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
130
Start Date
February 2017
Completion Date
July 2019
Primary Completion Date
December 2018
Eligibility Criteria
Inclusion Criteria: 1. Male or female ,under the age of 60. 2. Diagnosis of SAA and VSAA in accordance with theCamitta standard (see appendix 1). 3. Confirmed of heavy and very heavy aplastic anemia within 6 months. 4. No obvious abnormal liver and kidney function: ALT, AST,≤2.5 times the upper limit of normal , serum Creatinine and BUN ≤1.25 times the upper limit of normal 5. Clear understanding, voluntary to participate in the study, and signed informed consent document by the patient or the legal guardian 6. Willingness and ability to comly with the treatment plan, follow-up and laboratory tests as required Exclusion Criteria: 1. Congenital aplastic anemia 2. Pregnancy or breastfeeding 3. Participated in other clinical trials within three months 4. Presence of Any fatal disease, including respiratory failure, heart failure, liver or kidney failure, et al 5. Aplastic anemia caused by the treatment of other malignant tumor treatment 6. With severe mental illness 7. With other malignant tumor 8. Severe infection or the infection difficult to be controlled 9. Received ATG or cyclosporine A within six months 10. Severely allergic to biological agents 11. Any other situation judged by the investigator that the patients inappropriate for entry into this study
Gender
All
Ages
1 Year - 60 Years
Accepts Healthy Volunteers
No
Contacts
Fang Zhou, MD, 86-0531-51665316, [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT02838992
Organization ID
JinanMGH
Responsible Party
Principal Investigator
Study Sponsor
Jinan Military General Hospital
Collaborators
Shandong University of Traditional Chinese Medicine
Study Sponsor
Fang Zhou, MD, Study Chair, The General Hospital Of Jinan Military Command
Verification Date
January 2017