Reduced Toxicity Fludarabine (Flu) + Cyclophosphamide (CPM) + Rabbit Antithymocyte Globulin (rATG) Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia (SAA)
Reduced Toxicity Fludarabine, Cyclophosphamide Plus Thymoglobulin Conditioning Regimen for Unrelated Donor Transplantation in Severe Aplastic Anemia
1 other identifier
interventional
33
1 country
1
Brief Summary
Anti-thymocyte globulin (ATG) has been used in severe aplastic anemia as a part of the conditioning regimen. Among the many kinds of ATG preparations, thymoglobulin had been found to be more effective in preventing graft versus host disease (GVHD) and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for transplantation from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in unrelated donor transplantation. Our previous phase II study of fludarabine, cyclophosphamide plus thymoglobulin conditioning resulted in good engraftment (100%) and survival rate (74%). But grade III/IV toxicities occurred in 25% of patients and all events were treatment related mortalities. As cyclophosphamide is more toxic agent than fludarabine, we plan a new phase II study re; 'reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated donor transplantation in severe aplastic anemia' by reducing dosage of cyclophosphamide and increasing dosage of fludarabine.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Nov 2008
Typical duration for phase_2
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
Study Start
First participant enrolled
November 1, 2008
CompletedFirst Submitted
Initial submission to the registry
April 15, 2009
CompletedFirst Posted
Study publicly available on registry
April 16, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
October 1, 2012
CompletedMarch 26, 2012
March 1, 2012
3.9 years
April 15, 2009
March 23, 2012
Conditions
Outcome Measures
Primary Outcomes (1)
To evaluate engraftment potential of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for unrelated bone marrow transplantation in severe aplastic anemia.
From Nov. 2008 to Oct. 2012
Secondary Outcomes (3)
To evaluate toxicities of reduced toxicity fludarabine, cyclophosphamide plus thymoglobulin conditioning regimen for UBMT/PBSCT in SAA.
From Nov. 2008 to Oct. 2012
To evaluate overall and EFS rate after UBMT/PBSCT.
From Nov. 2008 to Oct. 2012
To evaluate GVHD and immunologic recovery after UBMT/PBSCT.
From Nov. 2008 to Oct. 2012
Study Arms (1)
Fludarabine
EXPERIMENTALInterventions
cyclophosphamide (60 mg/kg once daily i.v. on days -8, -7) fludarabine (40 mg/m2 once daily i.v. on days -6, -5, -4, -3, -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -4, -3, -2)
Eligibility Criteria
You may qualify if:
- Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.
- Peripheral blood
- Neutrophils \< 0.5 x 109/l
- Platelets \< 20 x 109/l
- Corrected reticulocytes \< 1%
- Bone marrow
- Severe hypocellularity (\< 25%)
- Moderate hypocellularity (25-30%) with hematopoietic cells representing \< 30% of residual cells
- No prior hematopoietic stem cell transplantation.
- Age: no limits.
- Performance status: ECOG 0-2.
- Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases:
- Heart: a shortening fraction \> 30% and ejection fraction \> 45%.
- Liver: total bilirubin \< 2 × upper limit of normal; ALT \< 3 × upper
- Kidney: creatinine \<2 × normal or a creatinine clearance (GFR) \> 60 ml/min/1.73m2.
- +3 more criteria
You may not qualify if:
- Pregnant or nursing women.
- Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.
- Psychiatric disorder that would preclude compliance.
- Congenital aplastic anemia including Fanconi anemia.
- Manipulated bone marrow.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Seoul National University Hospital
Seoul, 110-744, South Korea
Related Publications (1)
Kang HJ, Shin HY, Park JE, Chung NG, Cho B, Kim HK, Kim SY, Lee YH, Lim YT, Yoo KH, Sung KW, Koo HH, Im HJ, Seo JJ, Park SK, Ahn HS; Korean Society of Pediatric Hematology-Oncology. Successful engraftment with fludarabine, cyclophosphamide, and thymoglobulin conditioning regimen in unrelated transplantation for severe aplastic anemia: A phase II prospective multicenter study. Biol Blood Marrow Transplant. 2010 Nov;16(11):1582-8. doi: 10.1016/j.bbmt.2010.05.010. Epub 2010 May 26.
PMID: 20685256DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Hyo seop Ahn, M.D, Ph. D
The Korean Society of Pediatric Hematology Oncology
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- NETWORK
Study Record Dates
First Submitted
April 15, 2009
First Posted
April 16, 2009
Study Start
November 1, 2008
Primary Completion
October 1, 2012
Study Completion
October 1, 2012
Last Updated
March 26, 2012
Record last verified: 2012-03