Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
Verified March 2016 by Mayo Clinic
Information provided by (Responsible Party):
Shakila Khan, Mayo Clinic
First received: December 17, 2007
Last updated: March 23, 2016
Last verified: March 2016
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
Drug: Cyclophosphamide,Campath IH and TBI
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
||Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors
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 [ Time Frame: 2 years ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Patient survival probability at 100 days, 1 year and 2 years. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
| Estimated Enrollment:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||February 2017 (Final data collection date for primary outcome measure)
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)
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.
|Ages Eligible for Study:
||up to 60 Years (Child, Adult)
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00578266
|Rochester, Minnesota, United States, 55905 |
|Contact: Shakila Khan, MD 507-284-3442 Khan.firstname.lastname@example.org |
|Principal Investigator: Shakila P Khan, M.D |
||Shakila P. Khan, M.D.
||Shakila Khan, MD, Mayo Clinic
History of Changes
|Other Study ID Numbers:
|Study First Received:
||December 17, 2007
||March 23, 2016
||United States: Institutional Review Board
Keywords provided by Mayo Clinic:
Severe Aplastic Anemia (SAA)
Transplant for Severe Aplastic Anemia (SAA)
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on September 23, 2016
Bone Marrow Diseases
Physiological Effects of Drugs
Antineoplastic Agents, Alkylating
Molecular Mechanisms of Pharmacological Action