Healthy, Sickle Cell Disease
Conditions
Brief summary
The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect differences in blood flow and oxygen levels in different organ systems of participants with sickle cell disease (SCD). Differences in blood flow and oxygen levels detected by these techniques will be evaluated to determine their utility as biomarkers of clinical disease pathophysiology.
Interventions
None listed
Sponsors
Bioverativ Therapeutics Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
Key Inclusion Criteria: 1. Have a diagnosis of SCD confirmed by hemoglobin analysis. 2. Be in stable clinical condition, as determined by the Investigator. Subjects enrolled in Part B must also meet the following eligibility criterion at Screening: 3. Receiving scheduled standard of care RBC exchange transfusion therapy, with ≥3 transfusions already received. 4. Be deemed healthy, as determined by the Investigator, based on the physical examination, medical history, vital signs, 12-lead electrocardiogram (ECG), and clinical laboratory measurements. Key
Exclusion criteria
1. Inability to lie still for ≥5 minutes, claustrophobia sufficient to interfere with generating reliable MRI scans, body weight exceeding 320.0 lbs., or girth exceeding the magnet bore. 2. Presence of a metal device affected by MRI (e.g., any type of electronic, mechanical or magnetic implant, cardiac pacemaker, aneurysm clips, implanted cardiac defibrillator) or potential ferromagnetic foreign body (metal slivers, metal shavings, other metal objects) which would be a contraindication for MRI. 3. Acute pain crisis requiring hospitalization, with a discharge ≤4 weeks prior to the first imaging visit, or when determined by the Investigator to not be at steady state. 4. Recent (≤3 months) treatment with hydroxyurea therapy. NOTE: Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Blood flow in the brain of adults with severe SCD (4-10 vaso-occlusive crises [VOC]/ year) compared to healthy adults without SCD as assessed by MRI-ASL (arterial spin labeling) | Up to day 18 post screening visit |
Secondary
| Measure | Time frame |
|---|---|
| Skeletal muscle blood flow rates as assessed by NIRS | Up to day 21 post screening visit |
| Skin blood flow rates as assessed by LSCI | Up to day 21 post screening visit |
| Retinal blood flow rates as assessed by OI | Up to day 21 post screening visit |
| Total oxygen levels in the brain as assessed by MRI-ASL | Up to day 21 post screening visit |
| Kidney blood flow rates as assessed by MRI-SWI (susceptibility-weighted imaging) | Up to day 21 post screening visit |
| Total oxygen levels in the muscle as assessed by NIRS | Up to day 21 post screening visit |
| Total oxygen levels in the skin as assessed by LSCI | Up to day 21 post screening visit |
| Total oxygen levels in the retina as assessed by OI | Up to day 21 post screening visit |
| Total oxygen levels in the kidney as assessed by MRI-SWI | Up to day 21 post screening visit |
Countries
United States
Outcome results
None listed