Brief Title
Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)
Official Title
Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia
Brief Summary
Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need
alternative treatment option. Cord blood transplantation (CBT) has become an alternative
treatment means in various diseases, but it has not been proved to be good for severe
aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we
reported successful double units UCBT after engraftment failure with single unit with
promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus
TBI conditioning regimen for double units cord blood transplantation was proposed for the
patient with severe aplastic anemia without HLA-matched donor.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.
Secondary Outcome
To evaluate the incidence and severity of toxicity and treatment related mortality
Condition
Aplastic Anemia
Intervention
Cyclophosphosphamide, TBI
Study Arms / Comparison Groups
Fludarabine
Description:
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
Other
Estimated Enrollment
5
Start Date
November 2008
Completion Date
September 2012
Primary Completion Date
September 2012
Eligibility Criteria
Inclusion Criteria:
- 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%, ejection fraction > 45%.
- Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of
normal.
- Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60
ml/min/1.73m2.
- Patients must lack any active viral infections or active fungal infection.
- No appropriate donor
- Appropriate cord blood is available: matched at least in 4/6 of A, B, DR loci.
- Patients (or one of parents if patients age < 19) should sign informed consent.
Exclusion Criteria:
- 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.
Gender
All
Ages
1 Year - 21 Years
Accepts Healthy Volunteers
No
Contacts
Hyo Seop Ahn, M.D, Ph.D, 82 2 2072 3304, [email protected]
Location Countries
Korea, Republic of
Location Countries
Korea, Republic of
Administrative Informations
NCT ID
NCT00881933
Organization ID
KSPHO-SCT0804
Study Sponsor
The Korean Society of Pediatric Hematology Oncology
Study Sponsor
Hyo Seop Ahn, M.D, Ph.D, Principal Investigator, The Korean Society of Pediatric Hematology Oncology
Verification Date
March 2012