Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

This study is currently recruiting participants.
Verified January 2014 by Mayo Clinic
Sponsor:
Information provided by (Responsible Party):
Shakila Khan, Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00578266
First received: December 17, 2007
Last updated: January 27, 2014
Last verified: January 2014

December 17, 2007
January 27, 2014
February 2007
February 2014   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT00578266 on ClinicalTrials.gov Archive Site
Patient survival probability at 100 days, 1 year and 2 years. [ Time Frame: 2 years ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors

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

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.

Interventional
Phase 2
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Anemia, Aplastic
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)
No Intervention: No Arms
Intervention: Drug: Cyclophosphamide,Campath IH and TBI
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
24
February 2016
February 2014   (final data collection date for primary outcome measure)

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.
Both
up to 60 Years
No
Contact: Shakila P Khan, M.D 507-284-3442 Khan.shakila@mayo.edu
United States
 
NCT00578266
06-006216
Yes
Shakila Khan, Mayo Clinic
Mayo Clinic
Not Provided
Principal Investigator: Shakila P. Khan, M.D. Mayo Clinic
Mayo Clinic
January 2014

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