Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)

Phase 1

• Conditions: Aplastic Anemia
• Interventions: Other: Cyclophosphosphamide, TBI

• Registration Number: NCT00881933
• Lead Sponsor: The Korean Society of Pediatric Hematology Oncology

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2008-11
• Study First Submitted: 2009-04-14
• Study First Submitted QC: 2009-04-14
• Study First Posted: 2009-04-15
• Status Verified: 2012-03
• Last Update Submitted: 2012-03-23
• Last Update Posted: 2012-03-26
• Primary Completion: 2012-09
• Study Completion: 2012-09

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 5

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Fludarabine	Cyclophosphosphamide, TBI	-

Primary Outcome Measures

Name	Time	Method
To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.	From Oct 2008. to Sep 2011

Secondary Outcome Measures

Name	Time	Method
To evaluate the incidence and severity of toxicity and treatment related mortality	From Oct 2008. to Sep 2011
To evaluate overall and event free survival rate	From Oct 2008. to Sep 2011
To evaluate acute and chronic GVHD	From Oct 2008. to Sep 2011
To evaluate immunologic recovery after CBT	From Oct 2008. to Sep 2011

Trial Locations

Locations (1)

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of