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Completed

NCT Number: NCT00578266

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

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

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Key information

Conditions

Anemia, Aplastic Anemia Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

Up to 60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Mayo Clinic

Rochester, Minnesota, 55905, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Cyclophosphamide,Campath IH and TBI

Drug

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)

Primary outcomes

  1. 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 outcomes

  1. Patient survival probability at 100 days, 1 year and 2 years.

    Time frame: 2 years

Sponsors and collaborators

Lead sponsor

Mayo Clinic

Other

Registry information

Official study title

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors

Important dates

Study start
2007
Primary completion
2016
Study completion
2016
First posted
Dec 21, 2007
Registry last updated
Mar 24, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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