Back to resultsAllogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
Primary Purpose
Anemia, Aplastic
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Cyclophosphamide,Campath IH and TBI
Sponsored by
About this trial
This is an interventional treatment trial for Anemia, Aplastic focused on measuring Severe Aplastic Anemia (SAA), Transplant for Severe Aplastic Anemia (SAA)
Eligibility Criteria
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.
Sites / Locations
- Mayo Clinic
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
No Arms
Arm Description
Outcomes
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
Secondary Outcome Measures
Patient survival probability at 100 days, 1 year and 2 years.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00578266
Brief Title
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
Official Title
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors
Study Type
Interventional
2. Study Status
Record Verification Date
March 2017
Overall Recruitment Status
Completed
Study Start Date
February 2007 (undefined)
Primary Completion Date
August 16, 2016 (Actual)
Study Completion Date
August 16, 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Mayo Clinic
4. Oversight
Data Monitoring Committee
Yes
5. 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
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Anemia, Aplastic
Keywords
Severe Aplastic Anemia (SAA), Transplant for Severe Aplastic Anemia (SAA)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
No Arms
Arm Type
Other
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide,Campath IH and TBI
Intervention Description
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)
Primary Outcome Measure Information:
Title
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
Time Frame
2 years
Secondary Outcome Measure Information:
Title
Patient survival probability at 100 days, 1 year and 2 years.
Time Frame
2 years
10. Eligibility
Sex
All
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shakila P. Khan, M.D.
Organizational Affiliation
Mayo Clinic
Official's Role
Principal Investigator
Facility Information:
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
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