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

Study Overview

• Status: Completed
• Conditions: Anemia, Aplastic
• Intervention / Treatment: Drug: Cyclophosphamide,Campath IH and TBI

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.

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: No older than 60 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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	2 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Patient survival probability at 100 days, 1 year and 2 years.	2 years

Collaborators and Investigators

• Sponsor: Mayo Clinic
• Investigators: Principal Investigator: Shakila P. Khan, M.D., Mayo Clinic

Study record dates

Study Major Dates

• Study Start: February 1, 2007
• Primary Completion (Actual): August 16, 2016
• Study Completion (Actual): August 16, 2016

Study Registration Dates

• First Submitted: December 17, 2007
• First Submitted That Met QC Criteria: December 20, 2007
• First Posted (Estimate): December 21, 2007

Study Record Updates

• Last Update Posted (Actual): March 24, 2017
• Last Update Submitted That Met QC Criteria: March 23, 2017
• Last Verified: March 1, 2017

More Information

Terms related to this study

• Keywords: Severe Aplastic Anemia (SAA); Transplant for Severe Aplastic Anemia (SAA)
• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Bone Marrow Failure Disorders; Anemia; Anemia, Aplastic; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antirheumatic Agents; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Antineoplastic Agents, Alkylating; Alkylating Agents; Myeloablative Agonists; Antineoplastic Agents, Immunological; Cyclophosphamide; Alemtuzumab
• Other Study ID Numbers: 06-006216