Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

This study is currently recruiting participants.

Verified February 2013 by Mayo Clinic

Sponsor:

Information provided by (Responsible Party):

Shakila Khan, Mayo Clinic

ClinicalTrials.gov Identifier:

NCT00578266

First received: December 17, 2007

Last updated: February 27, 2013

Last verified: February 2013

History of Changes

Tracking Information

First Received Date ^ICMJE

December 17, 2007

Last Updated Date

February 27, 2013

Start Date ^ICMJE

February 2007

Estimated Primary Completion Date

February 2014 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

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 ]

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

Patient survival probability at 100 days, 1 year and 2 years. [ Time Frame: 2 years ] [ Designated as safety issue: No ]

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 2
Phase 3

Study Design ^ICMJE

Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Arm (s)

No Intervention: 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

Recruiting

Estimated Enrollment ^ICMJE

Estimated Completion Date

February 2016

Estimated Primary Completion Date

February 2014 (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. Lack of an HLA identical family member.
A 6/6 or 5/6 HLA matched unrelated donor or a 5/6 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.

Gender

Both

Ages

up to 60 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact: Shakila P Khan, M.D

507-284-3442

Khan.shakila@mayo.edu

Location Countries ^ICMJE

United States

Administrative Information

NCT Number ^ICMJE

NCT00578266

Other Study ID Numbers ^ICMJE

06-006216

Has Data Monitoring Committee

Yes

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

Information Provided By

Mayo Clinic

Verification Date

February 2013

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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