Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00186797
• Date of registration: 09/09/2005
• Prospective Registration: No
• Primary sponsor: St. Jude Children's Research Hospital
• Public title: Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia AACD34
• Date of first enrolment: December 2002
• Target sample size: 28
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00186797
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Paul Woodard, MD
• Affiliation: St. Jude Children's Research Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age < 21 years

- Diagnosis of severe aplastic anemia. (As defined by at least 2 of the following: ANC
< 500/µl, platelet count < 20,000/µl, and a reticulocyte count < 1% after correction
for the hematocrit. In addition, the diagnostic bone marrow biopsy must contain less
than 25% of the normal cellularity).

- Patient must have failed one or more courses of immunosuppressive therapy that
included ATG. As immunosuppression may take up to 6 months to demonstrate a
response, patients must have been observed to have failed immunosuppression for a
minimum of six months.

- Absence of suitable HLA-matched sibling donor.

- Negative serum pregnancy test for females with child bearing potential.

- Patient/parent/guardian is able to provide informed consent.

Exclusion Criteria:

- Patients with a life expectancy < 6 weeks.

- Patients with severe renal disease (creatinine clearance < 40cc/min/1.73m2)

- Patients with pre-existing severe restrictive pulmonary disease (FVC <40% of
predicted)



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

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Procedure: Allogeneic stem cell transplant

Drug: Fludarabine, Cyclophosphamide

Primary Outcome(s)

To study the effects (good and bad) of this treatment on the patients, the aplastic anemia, and survival

To learn how many patients need extra T-cells or extra stem cells from the donor to help the donor's blood stem cells grow

To learn the safety of stem cell transplantation in patients with unrelated donors or partially matched family donors

To learn how well the donor bone marrow grows in patients who receive the research treatment

Secondary ID(s)

AACD34

Source(s) of Monetary Support

Please refer to primary and secondary sponsors