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

Primary Purpose

Aplastic Anemia

Status
Unknown status
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Cyclophosphosphamide, TBI
Sponsored by
The Korean Society of Pediatric Hematology Oncology
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Aplastic Anemia focused on measuring Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,, cord blood transplantation, pediatric

Eligibility Criteria

1 Year - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Seoul National University HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Fludarabine

Arm Description

Outcomes

Primary Outcome Measures

To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.

Secondary Outcome Measures

To evaluate the incidence and severity of toxicity and treatment related mortality
To evaluate overall and event free survival rate
To evaluate acute and chronic GVHD
To evaluate immunologic recovery after CBT

Full Information

First Posted
April 14, 2009
Last Updated
March 23, 2012
Sponsor
The Korean Society of Pediatric Hematology Oncology
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1. Study Identification

Unique Protocol Identification Number
NCT00881933
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2012
Overall Recruitment Status
Unknown status
Study Start Date
November 2008 (undefined)
Primary Completion Date
September 2012 (Anticipated)
Study Completion Date
September 2012 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor
The Korean Society of Pediatric Hematology Oncology

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Aplastic Anemia
Keywords
Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,, cord blood transplantation, pediatric

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Enrollment
5 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Fludarabine
Arm Type
Experimental
Intervention Type
Other
Intervention Name(s)
Cyclophosphosphamide, TBI
Intervention Description
fludarabine (30 mg/m2 once daily i.v. on days -7, -6, -5, -4, -3, & -2) (if body weight < 10 kg or if age < 1 year: 1.33 mg/kg) cyclophosphamide (60 mg/kg once daily i.v. on days -9, & -8) total body irradiation (3G on days -1)
Primary Outcome Measure Information:
Title
To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.
Time Frame
From Oct 2008. to Sep 2011
Secondary Outcome Measure Information:
Title
To evaluate the incidence and severity of toxicity and treatment related mortality
Time Frame
From Oct 2008. to Sep 2011
Title
To evaluate overall and event free survival rate
Time Frame
From Oct 2008. to Sep 2011
Title
To evaluate acute and chronic GVHD
Time Frame
From Oct 2008. to Sep 2011
Title
To evaluate immunologic recovery after CBT
Time Frame
From Oct 2008. to Sep 2011

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Hyoung Jin Kang, M.D, Ph.D
Phone
82 2 2072 3304
Email
kanghj@snu.ac.kr
First Name & Middle Initial & Last Name or Official Title & Degree
Ji Won Lee, M.D
Phone
82 2 2072 0177
Email
agnesjw@hanmail.net
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hyo Seop Ahn, M.D, Ph.D
Organizational Affiliation
The Korean Society of Pediatric Hematology Oncology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hyo Seop Ahn, M.D, Ph.D
Phone
82 2 2072 3625
Email
hsahn@snu.ac.kr

12. IPD Sharing Statement

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

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