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Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified October 2014 by Mayo Clinic
Information provided by (Responsible Party):
Shakila Khan, Mayo Clinic Identifier:
First received: December 17, 2007
Last updated: October 22, 2014
Last verified: October 2014

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 :

  1. Patient survival probability at 100 days, 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.
  3. Engraftment at 6 months, 1 year and 2 years

Condition Intervention Phase
Anemia, Aplastic
Drug: Cyclophosphamide,Campath IH and TBI
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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:

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 2016
Estimated Primary Completion Date: February 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: No Arms Drug: Cyclophosphamide,Campath IH and TBI


  • 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
  • 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.


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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00578266

Contact: Shakila P Khan, M.D 507-284-3442

United States, Minnesota
Mayo Clinic Recruiting
Rochester, Minnesota, United States, 55905
Contact: Shakila Khan, MD    507-284-3442   
Principal Investigator: Shakila P Khan, M.D         
Sponsors and Collaborators
Mayo Clinic
Principal Investigator: Shakila P. Khan, M.D. Mayo Clinic
  More Information

No publications provided

Responsible Party: Shakila Khan, MD, Mayo Clinic Identifier: NCT00578266     History of Changes
Other Study ID Numbers: 06-006216
Study First Received: December 17, 2007
Last Updated: October 22, 2014
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:
Anemia, Aplastic
Bone Marrow Diseases
Hematologic Diseases processed this record on February 27, 2015