ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia

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 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 the  Camitta 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

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