Treatment of Sickle Cell Anemia With Stem Cell Transplant
Public ClinicalTrials.gov record NCT01350232. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Anemia From HLA Matched or Partially-Matched Related Donors
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. It is believed this regimen is sufficiently efficacious and sufficiently gentle to apply to patients with sickle cell anemia and related disorders. It is proposed to characterize the efficacy and toxicity of this regimen in high risk patients with sickle cell anemia using criteria for patient selection that have been accepted in prior BMT trials in patients with sickle cell disease, specifically only the subset of patients whose prior clinical behavior indicates that they are at high risk for serious morbidity and early mortality. In addition, it is proposed to characterize the pathophysiology of a consistent febrile response seen in the haploidentical BMT regimen the investigators have developed at Thomas Jefferson University (TJU). The primary goal of this study is to determine the response rate to a reduced intensity conditioning regimen which consists of fludarabine, cytarabine, low dose total body irradiation and cyclophosphamide in patients with severe sickle cell anemia.
Study identification
- NCT ID
- NCT01350232
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Not applicable
- Enrollment
- 2 participants
Conditions and interventions
Interventions
- Fludarabine Drug
- Cytarabine Drug
- Cellular Infusions Device
- Total Body Irradiation Radiation
- Cyclophosphamide Drug
- Bortezomib Drug
- Rituximab Drug
- Plasmapheresis Procedure
Drug · Device · Radiation + 1 more
Eligibility (public fields only)
- Age range
- 18 Years to 45 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 2009 (Type not available)
- Primary completion
- Aug 2011 (Actual)
- Completion
- Aug 2011 (Actual)
- Last update posted
- May 16, 2025
2009 – 2011
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Thomas Jefferson University | Philadelphia | Pennsylvania | 19107 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01350232, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted May 16, 2025 · Synced Oct 20, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT01350232 live on ClinicalTrials.gov.