Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

Tracking Information
First Submitted Date ICMJE	December 17, 2007
First Posted Date ICMJE	December 21, 2007
Last Update Posted Date	March 24, 2017
Study Start Date ICMJE	February 2007
Actual Primary Completion Date	August 16, 2016 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: December 20, 2007)	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 ]
Original Primary Outcome Measures ICMJE	Same as current
Change History	Complete list of historical versions of study NCT00578266 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: December 20, 2007)	Patient survival probability at 100 days, 1 year and 2 years. [ Time Frame: 2 years ]
Original Secondary Outcome Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
Official Title ICMJE	Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors
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.
Study Type ICMJE	Interventional
Study Phase	Phase 1
Study Design ICMJE	Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
Condition ICMJE	Anemia, Aplastic
Intervention ICMJE	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)
Study Arms	No Arms; Intervention: Drug: Cyclophosphamide,Campath IH and TBI
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: March 23, 2017)	8
Original Estimated Enrollment ICMJE; (submitted: December 20, 2007)	24
Actual Study Completion Date	August 16, 2016
Actual Primary Completion Date	August 16, 2016 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	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.
Sex/Gender	Sexes Eligible for Study: All
Ages	up to 60 Years (Child, Adult)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT00578266
Other Study ID Numbers ICMJE	06-006216
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Shakila Khan, Mayo Clinic
Study Sponsor ICMJE	Mayo Clinic
Collaborators ICMJE	Not Provided
Investigators ICMJE	Principal Investigator: Shakila P. Khan, M.D. Mayo Clinic
PRS Account	Mayo Clinic
Verification Date	March 2017
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP