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Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Shakila Khan, Mayo Clinic
ClinicalTrials.gov Identifier:
NCT00578266
First received: December 17, 2007
Last updated: March 23, 2017
Last verified: March 2017
  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 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
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 Shakila Khan, 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 ]

Secondary Outcome Measures:
  • Patient survival probability at 100 days, 1 year and 2 years. [ Time Frame: 2 years ]

Enrollment: 8
Study Start Date: February 2007
Study Completion Date: August 16, 2016
Primary Completion Date: August 16, 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of SAA based on bone marrow aspirate and biopsy results. Failure to respond to immunosuppressive therapy and/or lack of an HLA identical family member.
  • A 10/10 or 9/10 HLA matched unrelated donor or a 9/10 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 ClinicalTrials.gov identifier: NCT00578266

Locations
United States, Minnesota
Mayo Clinic
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Mayo Clinic
Investigators
Principal Investigator: Shakila P. Khan, M.D. Mayo Clinic
  More Information

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: March 23, 2017

Keywords provided by Shakila Khan, 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
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists

ClinicalTrials.gov processed this record on July 19, 2017