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NCT00244010 · ClinicalTrials.gov registry record · NA
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
A NA study of Diamond-Blackfan Anemia and Anemia, Aplastic, sponsored by St. Jude Children's Research Hospital.
- Completed
- Registry status
- NA
- Development phase
- 4
- Enrollment target
- 1
- Study location
NCT00244010 is a NA study of Diamond-Blackfan Anemia and Anemia, Aplastic that has completed, run by St. Jude Children's Research Hospital. The registered enrollment target is 4 participants, below the 708-participant average among 11 other Diamond-Blackfan Anemia trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00244010, a NA study of Diamond-Blackfan Anemia and Anemia, Aplastic, has completed, sponsored by St. Jude Children's Research Hospital.
- COMPLETED
- Registry status
- NA
- Development phase
- 4 participants
- Enrollment target
- 1
- Study location
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, categorized as NA. The registered enrollment target is 4 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 708-participant average among 11 other Diamond-Blackfan Anemia trials with a reported enrollment target (99% lower). The listed sponsor is St. Jude Children's Research Hospital, which has 410 total studies on file at ClinicalTrials.gov.
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.
NCT00244010 reports 1 study location spanning 1 distinct geographic area - top geographies include Tennessee.
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.
What interventions are being tested in trial NCT00244010?
The interventions under investigation include: Allogeneic stem cell transplant (DEVICE).
Who is sponsoring clinical trial NCT00244010?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
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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