NCT00578266

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Mayo Clinic · Academic / Other
• Sex: All
• Age: 60 Years
• Healthy volunteers: Not accepted

Summary

For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be : 1. Patient survival probability at 100 days, 1 year and 2 years. 2. Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years. 3. Engraftment at 6 months, 1 year and 2 years

Detailed description

The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results. For this initial study we plan to enroll up to 24 patients.

Conditions

• Anemia, Aplastic

Interventions

Type	Name	Description
DRUG	Cyclophosphamide,Campath IH and TBI	DAY 5 TREATMENT * 6 5 CYTOXAN 50 mg/kg WITH MESNA * 5 CYTOXAN 50 mg/kg WITH MESNA; * 4 CYTOXAN 50 mg/kgWITH MESNA; CAMPATH 3-10 mg * 3 CYTOXAN 50 mg/kg WITH MESNA; CAMPATH; * 2 TBI; CAMPATH; TACROLIMUS * 1 TBI (second fraction); CAMPATH (am) 0 STEM CELL INFUSION (pm)

Timeline

• Start date: 2007-02-01
• Primary completion: 2016-08-16
• Completion: 2016-08-16
• First posted: 2007-12-21
• Last updated: 2017-03-24

Locations

1 site across 1 country: United States