Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

Sponsor

Mayo Clinic (Other)

Overall Status

Completed

CT.gov ID

NCT00578266

Collaborator

(none)

Enrollment

Location

Arm

114.5

Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief 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 :

Patient survival probability at 100 days, 1 year and 2 years.
Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
Engraftment at 6 months, 1 year and 2 years

Condition or Disease	Intervention/Treatment	Phase
Anemia, Aplastic	Drug: Cyclophosphamide,Campath IH and TBI	Phase 1

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.

Study Design

Study Type:

Interventional

Actual Enrollment :

8 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Feb 1, 2007

Actual Primary Completion Date :

Aug 16, 2016

Actual Study Completion Date :

Aug 16, 2016

Arms and Interventions

Arm	Intervention/Treatment
Other: No Arms	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)

Arm

Intervention/Treatment

Other: No Arms

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)

Outcome Measures

Primary Outcome Measures

1. Engraftment at 6 months, 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 [2 years]

Secondary Outcome Measures

Patient survival probability at 100 days, 1 year and 2 years. [2 years]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Diagnosis of SAA based on bone marrow aspirate and biopsy results. Failure to respond to immunosuppressive therapy and/or lack of an HLA identical family member.
A 10/10 or 9/10 HLA matched unrelated donor or a 9/10 matched related donor available after high resolution typing.

Exclusion Criteria:

Patients with Aplastic anemia and active infection must be treated to maximally resolve this problem before beginning the conditioning regimen.
HIV seropositive patients
Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.
Patient greater than 60 years of age.
Women who are pregnant or nursing.
Patients with active hepatitis
Patients with severe cardiac dysfunction defined as shortening fraction <25%.
Patients with severe renal dysfunction defined as creatinine clearance <40ml/mim/1.73m2.
Patient with severe pulmonary dysfunction with FEV1, FVC and DLCO 40% of predicted or 3 SD below normal.