Know Cancer

or
forgot password

Cyclophosphamide and Antithymocyte Globulin Conditioning Regimen for Marrow Transplantation From HLA-Matched Family Members for Severe Aplastic Anemia: Effect of Marrow Cell Dose on Chronic Graft-vs.-Host Disease: A Multi-Center Trial


Phase 2
N/A
65 Years
Not Enrolling
Both
Aplastic Anemia

Thank you

Trial Information

Cyclophosphamide and Antithymocyte Globulin Conditioning Regimen for Marrow Transplantation From HLA-Matched Family Members for Severe Aplastic Anemia: Effect of Marrow Cell Dose on Chronic Graft-vs.-Host Disease: A Multi-Center Trial


PRIMARY OBJECTIVES:

I. Minimize the incidence of chronic GVHD by restricting the transplanted marrow dose to
2.0-2.5 x 10^8 nucleated cells/kg.

SECONDARY OBJECTIVES:

I. Engraftment and overall survival.

OUTLINE:

CONDITIONING REGIMEN: Patients receive cyclophosphamide intravenously (IV) on days -5 to -2
and anti-thymocyte globulin IV over 4-10 hours on days -4 to -2.

TRANSPLANTATION: Patients undergo allogeneic bone marrow transplantation on day 0.

GVHD PROPHYLAXIS: Patients receive methotrexate IV on days 1, 3, 6, and 11 and cyclosporine
IV over 1 hour or orally (PO) twice daily on days -1 to 50, followed by a taper until 6
months after grafting.

After completion of study treatment, patients are followed up at on day 180, 1 year, 1.5
years, 2 years, 3 years, and yearly thereafter.


Inclusion Criteria:



- Any patient who has aplastic anemia with marrow failure involving 2 of the three
following criteria: granulocytes < 500/uL; a corrected reticulocyte count of < 1%;
platelet count of < 20,000/uL

- Availability of an human leukocyte antigen (HLA)-matched family member

- DONOR: Family member who is HLA-matched

- DONOR: If more than one HLA-matched family member is available, priority will be
given to a donor who is genotypically HLA-identical, of appropriate cytomegalovirus
(CMV) serology, ABO compatible, and, in case of a female donor, non-parous

Exclusion Criteria:

- Severe disease other than aplastic anemia that would severely limit the probability
of survival during the graft procedure:

- Patients who have developed clonal cytogenetic abnormalities or myelodysplastic
syndrome (preleukemia)

- Patients with Fanconi's anemia

- Aplasia secondary to radiation or cytotoxic chemotherapy

- Patients with paroxysmal nocturnal hemoglobinuria who have not developed
aplastic anemia

- Severe organ toxicities:

- Cardiac insufficiency requiring treatment or symptomatic coronary artery
disease;

- Severe hypoxemia , partial pressure of oxygen (pO2) < 70 mm Hg, with decreased
diffusion capacity of carbon monoxide (DLCO) < 70% of predicted; or mild
hypoxemia, pO2 < 80 mm Hg with severely decreased DLCO < 60% of predicted;

- Impaired renal function (creatinine > 2 times upper limit of normal or estimated
creatinine clearance < 60 ml/min)

- Fungal infections with radiological progression after receipt of amphotericin B or
active triazole for greater than 1 month

- Human immunodeficiency virus (HIV)-positive patients

- Females who are pregnant or breast-feeding

- DONOR: Donors who have increase anesthetic risk and are not able psychologically and
medically to tolerate the procedure

- DONOR: HIV-positive donors

Type of Study:

Interventional

Study Design:

Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Incidence of chronic GVHD

Outcome Description:

Analyzed using cumulative incidence estimates, treating death or rejection as competing risk events. Confidence intervals will be calculated using the method described by Marubini and Valsecchi (1995). Risk factors for the development of chronic GVHD will be evaluated using Cox regression analyses methods.

Outcome Time Frame:

2 years

Safety Issue:

No

Principal Investigator

Rainer Storb

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Authority:

United States: Institutional Review Board

Study ID:

2054.00

NCT ID:

NCT00343785

Start Date:

February 2006

Completion Date:

Related Keywords:

  • Aplastic Anemia
  • Anemia
  • Anemia, Aplastic
  • Graft vs Host Disease

Name

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumSeattle, Washington  98109
Huntsman Cancer Institute/University of UtahSalt Lake City, Utah  84112
Froedtert and the Medical College of WisconsinMilwaukee, Wisconsin  53226