ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia
Multi-center Clinical Study of Immunosuppressants, Cyclophosphamide, And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia
1 other identifier
interventional
130
1 country
1
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_4
Started Feb 2017
Typical duration for phase_4
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 6, 2016
CompletedFirst Posted
Study publicly available on registry
July 20, 2016
CompletedStudy Start
First participant enrolled
February 1, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
July 1, 2019
CompletedFebruary 1, 2017
January 1, 2017
1.8 years
July 6, 2016
January 31, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
The total response rate
Response rate is the ratio of CR and PR patients to all evaluated patients at the time point. CR,PR,NR and relapse is evaluated according to the guidelines for the diagnosis and management of adult aplastic anaemia from British Committee for Standards in Haematology (BCSH)
Every 3 months to 24 months
Secondary Outcomes (4)
Neutrophil recovery time
From day 0 until the first of 3 consecutive days
Infection rates
1 year
Overall survival
2 years
Treatment related mortality
2 years
Study Arms (2)
ATG, Cy and cord blood transfusion group
EXPERIMENTALATG 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
ATG and CSA group
ACTIVE COMPARATORATG 3mg/kg/d for 5 days CSA started from 5mg/kg/d and adjusted to maintain trough serum concentration of 200-400ng/ml Intervention: Drug: Rabbit ATG, Thymoglobuline (Genzyme) plus CSA
Interventions
ATG is an infusion of rabbit-derived antibodies against human T cells, which is used in the prevention and treatment of acute rejection in organ transplantation and therapy of aplastic anemia
Cyclophosphamide is a medication mainly used in chemotherapy. It is an alkylating agent of the nitrogen mustard type
CsA is an immunosuppressant drug widely used in organ transplantation to prevent rejection. It reduces the activity of the immune system by interfering with the activity and growth of T cells
Cord blood is blood that remains in the placenta and in the attached umbilical cord after childbirth. Cord blood is collected because it contains stem cells, which can be used to treat hematopoietic and genetic disorders
Eligibility Criteria
You may qualify if:
- Male or female ,under the age of 60.
- Diagnosis of SAA and VSAA in accordance with the \<aplastic anemia, diagnosis and treatment expert consensus\> Camitta standard (see appendix 1).
- Confirmed of heavy and very heavy aplastic anemia within 6 months.
- 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
- Clear understanding, voluntary to participate in the study, and signed informed consent document by the patient or the legal guardian
- Willingness and ability to comly with the treatment plan, follow-up and laboratory tests as required
You may not qualify if:
- Congenital aplastic anemia
- Pregnancy or breastfeeding
- Participated in other clinical trials within three months
- Presence of Any fatal disease, including respiratory failure, heart failure, liver or kidney failure, et al
- Aplastic anemia caused by the treatment of other malignant tumor treatment
- With severe mental illness
- With other malignant tumor
- Severe infection or the infection difficult to be controlled
- Received ATG or cyclosporine A within six months
- Severely allergic to biological agents
- Any other situation judged by the investigator that the patients inappropriate for entry into this study
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Jinan Military General Hospitallead
- Shandong University of Traditional Chinese Medicinecollaborator
- Jining Medical Universitycollaborator
- Weifang Medical Universitycollaborator
- Guangzhou First People's Hospitalcollaborator
- Harbin Hematology and Oncology Institutecollaborator
- Jining First People's Hospitalcollaborator
- JIANGXI Provincal People's Hospitalcollaborator
- Jinhua Central Hospitalcollaborator
- Linyi People's Hospitalcollaborator
- Shandong Cord Blood Bankcollaborator
- Qingdao Hiser Medical Groupcollaborator
- Qingdao Universitycollaborator
- Taian City Central Hospitalcollaborator
- Yantai Yuhuangding Hospitalcollaborator
- Yishui Central Hospital of LINYIcollaborator
- Institute of Hematology & Blood Diseases Hospital, Chinacollaborator
- Shengjing Hospitalcollaborator
Study Sites (1)
The General Hospital Of Jinan Military Command
Jinan, Shandong, 250031, China
Related Publications (5)
Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, Hillmen P, Ireland R, Kulasekararaj A, Mufti G, Snowden JA, Samarasinghe S, Wood A, Marsh JC; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16. No abstract available.
PMID: 26568159BACKGROUNDZhou F, Ge L, Yu Z, Fang Y, Kong F. Clinical observations on intensive immunosuppressive therapy combined with umbilical cord blood support for the treatment of severe aplastic anemia. J Hematol Oncol. 2011 Jun 10;4:27. doi: 10.1186/1756-8722-4-27.
PMID: 21663651RESULTXie LN, Fang Y, Yu Z, Song NX, Kong FS, Liu XM, Zhou F. Increased immunosuppressive treatment combined with unrelated umbilical cord blood infusion in children with severe aplastic anemia. Cell Immunol. 2014 May-Jun;289(1-2):150-4. doi: 10.1016/j.cellimm.2014.03.014. Epub 2014 Apr 3.
PMID: 24838091RESULTYu Z, Zhou F, Ge LF, Liu XM, Fang Y, Xie LN, Kong FS, Song NX, Yu QQ. Mechanism of immunosuppressants combined with cord blood for severe aplastic anemia. Int J Clin Exp Med. 2015 Feb 15;8(2):2484-94. eCollection 2015.
PMID: 25932194RESULTXie LN, Zhou F. Unexpected unrelated umbilical cord blood stem cell engraft in two patients with severe aplastic anemia that received immunosuppressive treatment: A case report and literature review. Exp Ther Med. 2015 Oct;10(4):1563-1565. doi: 10.3892/etm.2015.2698. Epub 2015 Aug 21.
PMID: 26622526RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Fang Zhou, MD
The General Hospital Of Jinan Military Command
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Principal Investigator
Study Record Dates
First Submitted
July 6, 2016
First Posted
July 20, 2016
Study Start
February 1, 2017
Primary Completion
December 1, 2018
Study Completion
July 1, 2019
Last Updated
February 1, 2017
Record last verified: 2017-01