Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00427336
• Date of registration: 25/01/2007
• Prospective Registration: No
• Primary sponsor: M.D. Anderson Cancer Center
• Public title: Purine Analog-Based Conditioning in Patients With Severe Aplastic Anemia
• Scientific title: Purine Analog-Based Conditioning for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia
• Date of first enrolment: December 2000
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00427336
• Study type: Interventional
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Paolo Anderlini, MD
• Affiliation: M.D. Anderson Cancer Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- Patients up to 70 years of age with a diagnosis of severe AA (Camitta et al., 1979)
and a matched unrelated donor who are unresponsive to IS or who have relapsed after an
initial response to IS. Patients with a diagnosis of SAA and an human leukocyte
antigen (HLA) - compatible sibling donor are eligible only if they are 40 years of age
or older (up to age 70) and regardless whether they have received IS or not. Patients
with primary or secondary graft failure following autologous or allogeneic stem cell
transplant are eligible.

- Patients must have a serum bilirubin of 2 mg/dl or less, serum creatinine < 2.0 mg/dl,
no symptomatic cardiac or pulmonary disease and a PS of no more than 2. Life
expectancy not severely limited by concomitant illness (> 12 weeks). Left ventricular
ejection fraction > 40%, no uncontrolled arrhythmia or symptomatic cardiac disease.
Forced Expiratory Volume in 1 Second (FEV1), Forced Vital Capacity (FVC) and Carbon
Monoxide Diffusing Capacity (DLCO) > 40%. No symptomatic pulmonary disease. Negative
pregnancy test.

- Patients must have an HLA-compatible related or unrelated donor willing to donate
marrow or rhG-CSF-mobilized peripheral blood stem cells. In the event of transplants
from matched unrelated donors, a high-resolution allele match for HLA-A, -B, -C, -DRB1
and DQB1 ("10 of 10 match") is preferred. However, a one-antigen mismatch
("micromismatch") is also considered acceptable matching ("9 of 10 match").

- Patients must sign informed consent. In the event of a pediatric patient (i.e., a
minor), consent will be provided by their guardian/parent.

- Lack of clonal cytogenetic abnormalities associated with acute myeloid leukemia (AML),
myelodysplastic syndrome (MDS) or other hematologic malignancies.

Exclusion Criteria:

- Life expectancy of less than 8 weeks. Inability to provide informed consent.



Age minimum: N/A
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: Cyclophosphamide

Drug: Antithymocyte Globulin

Drug: Fludarabine

Primary Outcome(s)

Number of Patients With Engraftment Response [Time Frame: First 100 days post transplant.]

Secondary ID(s)

IDP00-266

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 27/10/2011
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00427336