NCT00881933

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
5

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Nov 2008

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

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

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

April 14, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 15, 2009

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2012

Completed
Last Updated

March 26, 2012

Status Verified

March 1, 2012

Enrollment Period

3.8 years

First QC Date

April 14, 2009

Last Update Submit

March 23, 2012

Conditions

Aplastic Anemia

Keywords

Fludarabine, Cyclophosphamide, TBI, severe aplastic anemia,cord blood transplantation, pediatric

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (4)

  • 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

Study Arms (1)

Fludarabine

EXPERIMENTAL
Other: Cyclophosphosphamide, TBI

Interventions

Cyclophosphosphamide, TBIOTHER

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)

Fludarabine

Eligibility Criteria

Age1 Year - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

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%, 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.
  • +4 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Seoul National University Hospital

Seoul, 110-744, South Korea

RECRUITING

MeSH Terms

Conditions

Anemia, AplasticBrain Injuries, Traumatic

Interventions

Whole-Body Irradiation

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow DiseasesBrain InjuriesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesCraniocerebral TraumaTrauma, Nervous SystemWounds and Injuries

Intervention Hierarchy (Ancestors)

RadiotherapyTherapeuticsInvestigative Techniques

Study Officials

  • Hyo Seop Ahn, M.D, Ph.D

    The Korean Society of Pediatric Hematology Oncology

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Hyoung Jin Kang, M.D, Ph.D

CONTACT

82 2 2072 3304kanghj@snu.ac.kr

Ji Won Lee, M.D

CONTACT

82 2 2072 0177agnesjw@hanmail.net

Study Design

Study Type
interventional
Phase
phase 1
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
NETWORK

Study Record Dates

First Submitted

April 14, 2009

First Posted

April 15, 2009

Study Start

November 1, 2008

Primary Completion

September 1, 2012

Study Completion

September 1, 2012

Last Updated

March 26, 2012

Record last verified: 2012-03

Locations

KR(1)