Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia - Full Text View

Sponsor:	Mayo Clinic
Information provided by:	Mayo Clinic
ClinicalTrials.gov Identifier:	NCT00578266

Purpose

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	Intervention	Phase
Anemia, Aplastic	Drug: Cyclophosphamide,Campath IH and TBI	Phase II Phase III

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Historical Control, Single Group Assignment, Safety/Efficacy Study
Official Title:	Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors

Resource links provided by NLM:

MedlinePlus related topics: Anemia

Drug Information available for: Cyclophosphamide Campath Alemtuzumab

U.S. FDA Resources

Further study details as provided by Mayo Clinic:

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:	24
Study Start Date:	February 2007
Estimated Study Completion Date:	February 2014
Estimated Primary Completion Date:	February 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
No Arms: No Intervention	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)

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.

Eligibility

Ages Eligible for Study:	up to 60 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00578266

Contacts

Contact: Shakila P Khan, M.D

507-284-3442

Khan.shakila@mayo.edu

Locations

United States, Minnesota
Mayo Clinic	Recruiting
Rochester, Minnesota, United States, 55905
Principal Investigator: Shakila P Khan, M.D

Sponsors and Collaborators

Mayo Clinic

Investigators

Principal Investigator:

Shakila P. Khan, M.D.

Mayo Clinic

More Information

No publications provided

Responsible Party:	Mayo Clinic ( Shakila Khan M.D. )
Study ID Numbers:	06-006216
Study First Received:	December 17, 2007
Last Updated:	July 20, 2009
ClinicalTrials.gov Identifier:	NCT00578266 History of Changes
Health Authority:	United States: Institutional Review Board

Keywords provided by Mayo Clinic:

Severe Aplastic Anemia (SAA)
Transplant for Severe Aplastic Anemia (SAA)

Additional relevant MeSH terms:

Molecular Mechanisms of Pharmacological Action
Immunologic Factors
Antineoplastic Agents
Hematologic Diseases
Physiological Effects of Drugs
Anemia
Cyclophosphamide
Immunosuppressive Agents
Pharmacologic Actions

Alemtuzumab
Therapeutic Uses
Myeloablative Agonists
Anemia, Aplastic
Antineoplastic Agents, Alkylating
Bone Marrow Diseases
Antirheumatic Agents
Alkylating Agents

ClinicalTrials.gov processed this record on February 08, 2010