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Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia

NCT00065260 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 54

Last updated 2021-07-21

Study results available
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Summary

Severe aplastic anemia, characterized by pancytopenia and a hypocellular bone marrow, is effectively treated by immunosuppressive therapy, usually a combination of antithymocyte globulin (ATG) and cyclosporine (CsA). Survival rates following this regimen are equivalent to those achieved with allogeneic stem cells transplantation. However, approximately 1/3 of patients will not show blood count improvement after ATG/CsA. General experience and small pilot studies have suggested that such patients may benefit from further immunosuppression. Furthermore, analysis of our own clinical data suggest that patients with poor blood count responses to a single course of ATG, even when transfusion-independence is achieved, have a markedly worse prognosis than patients with robust hematologic improvement. The management of such cases is uncertain.

This study will enroll patients who are either refractory to h-ATG (continued severe pancytopenia) or who have only modest improvement in blood counts (weak hematologic responders) to receive a further immunosuppressive therapy, delivered either as rabbit ATG (Thymoglobulin, r-ATG) or a humanized monoclonal antibody to T-cells, alemtuzumab (Campath-1H ). Primary endpoint will be response rate at 3 months defined as no longer meeting criteria for severe aplastic anemia. Relapse, robustness of hematopoietic recovery at 3 months, survival and clonal evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplasia and acute leukemia will be the secondary endpoints.

Conditions

  • Aplastic Anemia

Interventions

DRUG

Campath-1H

Campath-1H IV 10 days. Adults:10mg/day (children:0.2mg/kg/day).

DRUG

r-ATG

Rabbit ATG 3.5mg/kg/day for consecutive 5 days

DRUG

CsA

CsA 10mg/kg/day orally twice daily for 6 months (15mg/kg/day for children under 12 yrs.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    lead NIH

Principal Investigators

  • Danielle M Townsley, M.D. · National Heart, Lung, and Blood Institute (NHLBI)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2003-11-06
Primary Completion
2010-12-29
Completion
2016-02-05

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00065260 on ClinicalTrials.gov