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

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004474
• Other study ID #: 199/14004
• Secondary ID: RPCI-RP-9804NCI-
• Status: Completed
• Phase: Phase 3
• First received: October 18, 1999
• Last updated: March 9, 2018
• Start date: September 1998
• Est. completion date: August 2007

Study information

• Verified date: October 2008
• Source: Office of Rare Diseases (ORD)
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 224
• Est. completion date: August 2007
• Est. primary completion date: August 2007
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 59 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia

Intervention

Drug:
• Anti-thymocyte globulin
Given via IV over 4 hours
• Cyclophosphamide
Given via IV over 60 minutes on Days -5 to -2

Procedure:
• Bone marrow transplantation
Given on Day 0

Locations

Country	Name	City	State
United States	Roswell Park Cancer Institute	Buffalo	New York
United States	University of Texas - MD Anderson Cancer Center	Houston	Texas
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Midwest Children's Cancer Center	Milwaukee	Wisconsin

Sponsors (1)

Lead Sponsor	Collaborator
Roswell Park Cancer Institute

Country where clinical trial is conducted

United States, 

Outcome

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