NCT00004474

Brief Summary

OBJECTIVES: I. Compare outcome, including graft failure, graft versus host disease, and survival of HLA-identical sibling bone marrow transplants for aplastic anemia using cyclophosphamide with or without antithymocyte globulin as a conditioning regimen.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
224

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Sep 1998

Longer than P75 for phase_3

Geographic Reach
1 country

4 active sites

Status
completed

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

September 1, 1998

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
7.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 1, 2007

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 1, 2007

Completed
Last Updated

March 12, 2018

Status Verified

October 1, 2008

Enrollment Period

8.9 years

First QC Date

October 18, 1999

Last Update Submit

March 9, 2018

Conditions

Aplastic Anemia

Keywords

Hematologic DisordersRare Disease

Outcome Measures

Primary Outcomes (1)

  • Graft failure, graft versus host disease, and survival

    Measured at Day 100, Month 6, and Year 1 post-transplant

Study Arms (2)

1

EXPERIMENTAL

Participants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2 with antithymocyte globulin IV over 4 hours; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0.

Drug: Anti-thymocyte globulinDrug: CyclophosphamideProcedure: Bone marrow transplantation

2

EXPERIMENTAL

Participants will receive cyclophosphamide IV over 60 minutes on Days -5 to -2; then, after the last does cycophosphamide, participants will receive a bone marrow transplant over 60 to 120 minutes on Day 0.

Drug: CyclophosphamideProcedure: Bone marrow transplantation

Interventions

Anti-thymocyte globulinDRUG

Given via IV over 4 hours

1
CyclophosphamideDRUG

Given via IV over 60 minutes on Days -5 to -2

12
Bone marrow transplantationPROCEDURE

Given on Day 0

12

Eligibility Criteria

AgeUp to 59 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Severe aplastic anemia with the following criteria: Hypocellular bone marrow with cellularity less than 20% At least 2 of the following hematologic abnormalities: * Neutrophil count no greater than 500/mm3 * Platelet count no greater than 20,000/mm3 * Reticulocyte count no greater than 50,000/mm3 HLA-identical sibling donor available No clonal cytogenetic abnormalities, paroxysmal nocturnal hemoglobinuria, or myelodysplastic syndrome within 3 months of diagnosis of aplastic anemia No congenital or constitutional aplastic anemia or Fanconi anemia --Patient Characteristics-- Hepatic: Bilirubin less than 3 times upper limit of normal (ULN) Renal: Creatinine less than 2 times ULN Cardiovascular: Normal cardiac function Other: * No uncontrolled infection * No severe concurrent disease * HIV negative * Fertile patients must use effective contraception

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (4)

Roswell Park Cancer Institute

Buffalo, New York, 14263-0001, United States

Location

University of Texas - MD Anderson Cancer Center

Houston, Texas, 77030-4009, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Midwest Children's Cancer Center

Milwaukee, Wisconsin, 53226, United States

Location

MeSH Terms

Conditions

Anemia, AplasticHematologic DiseasesRare Diseases

Interventions

Antilymphocyte SerumCyclophosphamideBone Marrow Transplantation

Condition Hierarchy (Ancestors)

AnemiaHemic and Lymphatic DiseasesBone Marrow Failure DisordersBone Marrow DiseasesDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Immune SeraAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsBiological ProductsComplex MixturesPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus CompoundsTissue TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

September 1, 1998

Primary Completion

August 1, 2007

Study Completion

August 1, 2007

Last Updated

March 12, 2018

Record last verified: 2008-10

Locations

US(4)