Aplastic AnemiaAACD34NCT00186797
Purified CD34+ Hematopoietic Stem Cell Transplantation From Alternate Donors for Patients With Severe Aplastic Anemia
A study testing Allogeneic stem cell transplant and Fludarabine, Cyclophosphamide in people with Aplastic Anemia, run by St. Jude Children's Research Hospital at 1 site in United States.
Trial summary
This study is for patients with Severe Aplastic Anemia (SAA). A stem cell transplant from a genetically matched sibling donor can help or cure this disease in 85 to 100 percent of patients. Stem cells are immature blood cells that grow to become red blood cells, white blood cells or platelets. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Allogeneic stem cell transplant and Fludarabine, Cyclophosphamide
- Type of treatment
- Surgery or procedure
- How many people
- 28 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2002to May 2007
- Phase
- Interventional
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105
About this study
This study is for patients with Severe Aplastic Anemia (SAA). A stem cell transplant from a genetically matched sibling donor can help or cure this disease in 85 to 100 percent of patients. Stem cells are immature blood cells that grow to become red blood cells, white blood cells or platelets. A genetic "match" means a brother or sister has same immune type (HLA type) as the patient. Unfortunately, few patients have a matched sibling donor. The chance of negative outcomes is much higher with other types of donors.
This study will test the success of a new approach to stem cell transplant for SAA. Patients in this study will receive drugs and radiation treatment to destroy their diseased bone marrow and to prepare them for stem cell transplant. Bone marrow is the tissue inside the bones where stem cells are made.Stem cells will be harvested from the blood or bone marrow of genetically matched unrelated donors or partially matched family donors. The stem cells will be filtered using a new device that is currently under study. The patients will receive large doses of the filtered stem cells (stem cell graft). Researchers want to find out how the study treatment affects patients, the disease, and the chances for survival.
- Conditions
- Aplastic Anemia
- Being tested
- Allogeneic stem cell transplant, Fludarabine, Cyclophosphamide
- Keywords
- Aplastic Anemia
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Allogeneic stem cell transplantProcedure or surgery
Fludarabine, CyclophosphamideDrug
What is being measured
The primary outcome is the main question the study is designed to answer.
To learn the safety of stem cell transplantation in patients with unrelated donors or partially matched family donors
To study the effects (good and bad) of this treatment on the patients, the aplastic anemia, and survival
To learn how well the donor bone marrow grows in patients who receive the research treatment
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startDec 2002
- Primary completionSep 2005
- Study completionMay 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by St. Jude Children's Research HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 28 participants
Sponsor and investigators
- Lead sponsor
- St. Jude Children's Research Hospital
- Sponsor type
- University, hospital or other
- Investigators
- Paul Woodard, MD, Principal investigator, St. Jude Children's Research Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00186797
- Sponsor's protocol ID
- AACD34
- Acronym
- AACD34
- Status last verified
- Apr 2010
- First submitted
- 9 Sep 2005
- Last updated
- 9 Apr 2010
- Results posted
- No