NCT00737685

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 GVHD and rejection of organ transplants. As the fludarabine based conditioning regimens without total body irradiation have been reported to be promising for BMT/PBSCT from alternative donors in SAA, thymoglobulin was added to fludarabine and cyclophosphamide conditioning to reduce GVHD and to allow good engraftment in UBMT/UPBSCT.

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
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jan 2006

Longer than P75 for phase_2

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

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2006

Completed
2.6 years until next milestone

First Submitted

Initial submission to the registry

August 18, 2008

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 19, 2008

Completed
4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2012

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2012

Completed
Last Updated

March 26, 2012

Status Verified

March 1, 2012

Enrollment Period

6.6 years

First QC Date

August 18, 2008

Last Update Submit

March 23, 2012

Conditions

Anemia, Aplastic

Keywords

PediatricUBMTSevere

Outcome Measures

Primary Outcomes (2)

  • To evaluate the engraftment potential, incidence and severity of acute graft versus host disease,toxicity of conditioning regimen for UBMT in SAA.

    From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.

  • To evaluate overall and EFS follow-up of 1 year after UBMT/PBSCT.

    From Jan. 1. 2006 to Dec. 31. 2008. For 3 years

Secondary Outcomes (1)

  • To evaluate chronic GVHD and immunologic recovery after UBMT/PBSCT. and the efficacy of UBMT/PBSCT before immuno-suppressive therapy with anti-thymocyte globulin in severe aplastic anemia and long term toxicity of non-TBI based conditioning

    From Jan. 1. 2006 to Dec. 31. 2008. For 3 years.

Study Arms (1)

Fludarabine

EXPERIMENTAL
Drug: cyclophosphamide, fludarabine , thymoglobulin

Interventions

cyclophosphamide, fludarabine , thymoglobulinDRUG

cyclophosphamide (50 mg/kg once daily i.v. on days -9, -8, -7 \& -6) fludarabine (30 mg/m2 once daily i.v. on days -5, -4, -3 \& -2) thymoglobulin (2.5 mg/kg once daily i.v. on days -3, -2 \& -1)

Fludarabine

Eligibility Criteria

AgeUp to 25 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.
  • +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.
  • Manipulated bone marrow.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Seoul National University Hospital

Seoul, 110-744, South Korea

Location

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

Anemia, AplasticLymphoma, Follicular

Interventions

Cyclophosphamidefludarabinethymoglobulin

Condition Hierarchy (Ancestors)

AnemiaHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow DiseasesLymphoma, Non-HodgkinLymphomaNeoplasms by Histologic TypeNeoplasmsLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Study Officials

  • Hyo Seop Ahn, M.D, Ph.D

    The Korean Society of Pediatric Hematology Oncology

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NETWORK

Study Record Dates

First Submitted

August 18, 2008

First Posted

August 19, 2008

Study Start

January 1, 2006

Primary Completion

August 1, 2012

Study Completion

December 1, 2012

Last Updated

March 26, 2012

Record last verified: 2012-03

Locations

KR(1)