Actively Recruiting
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-05-13
200
Participants Needed
1
Research Sites
688 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens. Objective: To study the benefits of treatments that are intended to cure SCD. Eligibility: People aged 18 and older with SCD who are either receiving curative therapy in the next 3 months or don t have any plans to receive a curative therapy in the next 2 years. Design: At their first visit, participants will be screened with their medical history and a physical exam. Participants will then have a baseline visit. This will take about a week to complete and will include: Blood and heart tests MRI of the brain, heart, and lungs. Participants will lie on a bed that will move into the MRI scanner. Special padding may be placed around their head to keep it still. Interactive games. Participants will complete computer games that test memory, attention, problem solving, language, spatial orientation, processing speed, and emotion. Questionnaire rating quality of life Iothalamate test. An IV catheter will be placed into a vein. A contrast agent will be injected through the IV. Blood will then be collected at different time points. Lung function tests and a 6-minute walk test Vibration controlled transient elastography. A probe placed on the abdomen will measure liver scarring. DOS test. A light attached to the finger or toe will measure blood oxygen. Participants will have an end-of-study visit about 2 years after their baseline visit. This will include repeats of the baseline visit tests.
CONDITIONS
Official Title
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Willingness to follow all study procedures and be available for the full study duration
- Male or female aged 18 years or older
- Diagnosis of any type of sickle cell disease (including HbSS, HbSC, HbSbeta0-thal, HbSbeta+-thal) with plans to receive allogeneic hematopoietic cell transplant or gene therapy, or currently receiving non-curative treatment without plans for curative therapy
- Ability to travel to the NIH Clinical Center
- Ability to understand and provide informed consent
- At least one of the following: history of stroke or abnormal transcranial doppler exam (≥ 200 m/s); sickle cell-related kidney problems (creatinine ≥1.3 mg/dL with biopsy or nephrotic syndrome or creatinine clearance < 50 mL/min); tricuspid regurgitant velocity ≥ 2.5 m/s; recurrent priapism with at least 2 episodes lasting over 4 hours; sickle cell-related liver disease (ferritin > 1000 mcg/L or direct bilirubin > 0.4 mg/dL); more than 1 hospitalization per year for vaso-occlusive crises while on hydroxyurea; any acute chest syndrome while on hydroxyurea; osteonecrosis of 2 or more joints; or red cell alloimmunization
You will not qualify if you...
- Previous transplantation, including hematopoietic stem cell transplant or kidney transplant
- Pregnant or breastfeeding
- Allergy to iodine or iodinated contrast agents (excluded only from certain kidney tests)
- Presence of implanted metal objects incompatible with MRI (e.g., cerebral aneurysm clip, cochlear implant, pacemaker)
- Patients with a pacemaker or cardioverter defibrillator incompatible with certain liver tests
- Need for peritoneal or hemodialysis
- Uncontrolled infection or acute illness
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
J
Julia M Varga, R.N.
CONTACT
C
Courtney F Joseph, M.D.
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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