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Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
NCT00244010 · View on ClinicalTrials.gov ↗
Study Summary
Due to an overall and disease free survival of 85% to 100%, allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia (SAA). Unfortunately, only about 25% of patients have such a donor. For patients with SAA lacking a matched sibling donor, immunosuppressive therapy is the current treatment of choice. Approximately 70% of these patients have a complete or partial response to immunosuppressive therapy, achieving transfusion independence and/or growth factor independence. For the approximately 30% of patients who do not respond to immunosuppressive therapy or experience recurrence, alternative donor (matched unrelated, partially matched family member) transplantation is a treatment option. However, graft rejection and graft-versus-host-disease (GVHD) are significant barriers to success, decreasing event-free survival to 30% to 50%. This study offers stem cell transplantation using a partially matched family member (haploidentical) donor to those patients with no available HLA-matched sibling or matched unrelated donor. In an attempt to reduce GVHD and regimen-related toxicity while maintaining adequate engraftment, we plan to infuse a highly purified stem cell graft. The Miltenyi Biotec CliniMACS CD3 depletion system will be used to derive a defined allogeneic graft highly enriched for CD34+ hematopoietic cells and depleted of CD3+ T-lymphocytes from G-CSF mobilized, donor-derived peripheral blood stem cells. Patients 21 years of age and younger with refractory cytopenias are also eligible for this protocol as there are no other potentially curative therapies currently available for these conditions. The primary objective of this study is to evaluate the safety of transplantation using a haploidentical donor product engineered to targeted cell counts using the investigational CliniMACS device for patients with refractory severe aplastic anemia (SAA) or refractory c
Conditions Studied
Interventions
- DEVICE Allogeneic stem cell transplant
Study Locations (1)
Tennessee
- St. Jude Children's Research Hospital — Memphis
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 4 participants |
| Start Date | 2005-10 |
| Est. Completion | 2009-02 |
| Phase | NA |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00244010
The ClinicalTrials.gov registry entry for NCT00244010 describes a study currently listed as completed. It is categorized as NA, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is St. Jude Children's Research Hospital, which has 441 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 4 conditions, with Diamond-Blackfan Anemia appearing as the primary indexed condition, and to 1 intervention — of which Allogeneic stem cell transplant is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT00244010 reports 1 study location spanning 1 distinct geographic area — top geographies include Tennessee. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT00244010 about?
NCT00244010 is a clinical study titled "Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias". Due to an overall and disease free survival of 85% to 100%, allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia (SAA). Unfortunately, only about 25% of patients have such a donor. For patients ...
What is the current status of trial NCT00244010?
This trial is currently completed. It is a NA study. The enrollment target is 4 participants. The study started on 2005-10. Estimated completion is 2009-02.
What conditions does trial NCT00244010 study?
This clinical trial studies the following conditions: Diamond-Blackfan Anemia, Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Kostmann Syndrome. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT00244010?
The interventions under investigation include: Allogeneic stem cell transplant (DEVICE). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT00244010?
This trial is sponsored by St. Jude Children's Research Hospital, which has 441 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT00244010 being conducted?
This trial has 1 study location across Tennessee. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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