NCT01499888Active, not recruitingPhase 1/2
Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)
Phase I/II Study of Allogeneic Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease (SCD)
Source: ClinicalTrials.gov · Last updated 8 Jul 2026
Brief summary (registry, in English)
The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from hydroxyurea therapy. Fully HLA matched siblings will be used as donors for hematopoietic stem cells to reduce the risk of morbidity and mortality in this cohort of patients.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 45 (actual)
- First submitted
- 23 Nov 2011
- Start date
- 11 Nov 2011
- Primary completion date
- 23 Sept 2028
- Lead sponsor
- University of Illinois Chicago
- Diseases
- Stroke, Sickle cell disease
- NCT number
- NCT01499888
What you commit to
- Study type: Interventional · Phase 1/2
- Participants enrolled: 45
- From 11 Nov 2011 to the primary completion date, 23 Sept 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Who the study is looking for
- Patients with sickle cell disease, subtype Hgb SS, SC, or SB disease who are on chronic transfusion therapy for a prior stroke or those patients who were intolerant of hydroxyurea therapy or were being treated with hydroxyurea therapy and were complicated by at least one of the following:
- Stroke or central nervous system event lasting longer than 24 hours
- Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year severe enough to interfere with the patient's normal daily function or require medical attention in the clinic, emergency room, acute care center, or hospital
- Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits
- Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events
- Red-cell alloimmunization (≥ 2 antibodies) during longterm transfusion therapy
- Bilateral proliferative retinopathy with major visual impairment in at least one eye
- Osteonecrosis of 2 or more joints
- Sickle cell nephropathy
- Stage I or II sickle lung disease
- Symptoms of pulmonary hypertension and mean pulmonary artery pressure \> 25mmHg
- Age 16-60 years
- Karnofsky performance status of 70 or higher
- Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%
- Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50%
- Estimated GFR ≥ 30mL/min as calculated by the modified MDRD equation
- ALT ≤ 3x upper limit of normal
- No evidence of chronic active hepatitis or cirrhosis
- HIV-negative
- Patient is not pregnant
- History of compliance with medications and medical care
- Patient is able and willing to sign informed consent
- Patient has an HLA-identical matched related donor
Age in the registry: 16 years to 60 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| University of Illinois at Chicago Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.