Status:
ACTIVE_NOT_RECRUITING
Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)
Lead Sponsor:
University of Illinois at Chicago
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
16-60 years
Phase:
PHASE1
PHASE2
Brief Summary
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- sup...
Detailed Description
Sickle cell disease is an inherited defect caused by a mutation in the Beta globin gene affecting red blood cells. Symptoms begin at 6 months of life and often lead to debilitating vaso-occlusive pain...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- 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
Exclusion
Key Trial Info
Start Date :
November 11 2011
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2029
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT01499888
Start Date
November 11 2011
End Date
October 1 2029
Last Update
April 11 2025
Active Locations (1)
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1
University of Illinois at Chicago
Chicago, Illinois, United States, 60612