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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
  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 :

  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 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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:

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 2016
Estimated Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
No Intervention: No Arms 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
Contact: Shakila Khan, MD     507-284-3442     Khan.shakila@mayo.edu    
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: Shakila Khan, MD, Mayo Clinic
ClinicalTrials.gov Identifier: NCT00578266     History of Changes
Other Study ID Numbers: 06-006216
Study First Received: December 17, 2007
Last Updated: February 27, 2013
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
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Cyclophosphamide
Alemtuzumab
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
 Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists

ClinicalTrials.gov processed this record on May 23, 2013