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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 19 October 2017
Main ID:  NCT00578903
Date of registration: 19/12/2007
Prospective Registration: No
Primary sponsor: Baylor College of Medicine
Public title: Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia
Scientific title: Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD)
Date of first enrolment: February 2002
Target sample size: 22
Recruitment status: Terminated
URL:  https://clinicaltrials.gov/show/NCT00578903
Study type:  Interventional
Study design:   
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Kathryn Leung, MD
Address: 
Telephone:
Email:
Affiliation:  Baylor College of Medicine
Key inclusion & exclusion criteria

Inclusion Criteria:

1. Diagnosis of Severe Aplastic Anemia (SAA) based on bone marrow aspirate and biopsy
results.

2. Failure to respond to immunosuppressive therapy.

3. Lack of an Human Leukocyte Antigen (HLA) identical family member.

4. A 6/6 or 5/6 HLA matched unrelated donor or a 5/6 matched related donor available
after high resolution HLA typing.

5. Age from birth to 60 years.

Exclusion Criteria:

1. Severe disease other than aplastic anemia that would limit the probability of survival
during the graft procedure. Patients who present with active infection must be treated
to maximally resolve this problem before beginning the conditioning regimen.

2. Human immunodeficiency virus (HIV) seropositive patients

3. Patients who have clonal cytogenetic abnormalities or a myelodysplastic syndrome.

4. Patient greater than 60 years of age.

5. Women who are pregnant or nursing.

6. Patients with active hepatitis

7. Patients with severe cardiac dysfunction defined as shortening fraction < 25%.

8. Patients with severe renal dysfunction defined as creatinine clearance < 40
ml/mim/1.73m2.

9. Patient with severe pulmonary dysfunction with forced expiratory volume in the first
second (FEV1), forced vital capacity (FVC) and diffusing capacity of the lung for
carbon monoxide (DLCO) 40% of predicted or 3 standard deviations (SD) below normal.



Age minimum: N/A
Age maximum: 60 Years
Gender: All
Health Condition(s) or Problem(s) studied
Aplastic Anemia
Intervention(s)
Drug: Campath
Drug: Cytoxan
Drug: FK-506
Drug: Methotrexate
Procedure: Stem cell infusion
Radiation: Total Body Irradiation (TBI)
Primary Outcome(s)
Number of Subjects Alive at 100 Days Post Transplant [Time Frame: 100 days]
Secondary Outcome(s)
Number of Patients With Acute GVHD at 100 Days Post Transplant [Time Frame: 100 days]
Number of Patients With Chronic GVHD at 2 Years Post Transplant [Time Frame: 2 years]
Number of Patients With Engraftment Rate at 100 Days Post Transplant [Time Frame: 100 days post transplant]
Number of Subjects Alive at 1 Year Post Transplant [Time Frame: 1 year]
Number of Subjects Alive at 2 Years Post Transplant [Time Frame: 2 years]
Secondary ID(s)
10915-SAA MUD
SAA MUD
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Center for Cell and Gene Therapy, Baylor College of Medicine
Texas Children's Hospital
The Methodist Hospital System
Ethics review
Results
Results available: Yes
Date Posted: 11/10/2013
Date Completed:
URL: https://clinicaltrials.gov/ct2/show/results/NCT00578903
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