Phase III Randomized Study of Cyclophosphamide With or Without Antithymocyte Globulin Before Bone Marrow Transplantation in Patients With Aplastic Anemia

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00004474

Enrollment

224

Registered

1999-10-19

Start date

1998-09-30

Completion date

2007-08-31

Last updated

2018-03-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Aplastic Anemia

Keywords

Hematologic Disorders, Rare Disease

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.

Detailed description

PROTOCOL OUTLINE: This is a randomized, multicenter study. Patients are randomized to receive cyclophosphamide IV over 60 minutes on days -5 to -2 with or without antithymocyte globulin IV over 4 hours. All patients then receive bone marrow over 60-120 minutes on day 0, 36 hours after the last dose of cyclophosphamide. Patients are followed at day 100, at 6 months, and at 1 year posttransplant.

Interventions

DRUGAnti-thymocyte globulin

Given via IV over 4 hours

DRUGCyclophosphamide

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

PROCEDUREBone marrow transplantation

Given on Day 0

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Eligibility

Sex/Gender

ALL

Age

No minimum to 59 Years

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

Measure	Time frame
Graft failure, graft versus host disease, and survival	Measured at Day 100, Month 6, and Year 1 post-transplant

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026