Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01129323
• Date of registration: 21/04/2010
• Prospective Registration: No
• Primary sponsor: City of Hope Medical Center
• Public title: Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia
• Scientific title: Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia
• Date of first enrolment: November 2009
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: http://clinicaltrials.gov/show/NCT01129323
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Anna Pawlowska, MD
• Affiliation: City of Hope Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- 21 years old or younger

- Male or female recipients must have histopathologically confirmed diagnosis of
severe aplastic anemia. Diagnostic Criteria for Server Aplastic Anemia will be based
on the definitions set forth by the international Aplastic Anemia Study Group

- At least two of the following:

Absolute neutrophil count <0.5 x 109/L Platelet count <20 x 109 /L Anemia with corrected
reticulocyte count <1%

AND

- Bone marrow cellularity <25%, or bone marrow cellularity <50% with fewer than 30%
hematopoietic cell

- Availability of an HLA identical sibling

Exclusion Criteria:

- Active and uncontrolled infection

- HIV-1 infection

- Pregnancy or breastfeeding.

- DLCO <40% predicted

- Left Ventricular Ejection Fraction < 40%

- Performance scale Karnofsky <=40% or Lansky<=40% for patients <16 years old



Age minimum: N/A
Age maximum: 21 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Severe Aplastic Anemia

Intervention(s)

Drug: Cyclophosphamide, Fludarabine, Rabbit ATG

Primary Outcome(s)

Engraftment rate [Time Frame: Day 42 after allogeneic transplant]

Secondary Outcome(s)

Development of Graft vs. Host Disease [Time Frame: Days 180, 365 and yearly post transplant]

Evaluation of toxicities [Time Frame: Days 180, 365 and yearly post transplant]

Relapse [Time Frame: Days 180, 365 and yearly post transplant]

Evaluation of the occurrence of secondary malignancies [Time Frame: Days 180, 365 and yearly post transplant]

Evaluation of treatment feasibility [Time Frame: Days 180, 365 and yearly post transplant]

Overall survival [Time Frame: Days 180, 365 and yearly post transplant]

Secondary ID(s)

07081

Source(s) of Monetary Support

Please refer to primary and secondary sponsors