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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A | Cohort 1- Participants with severe SCD (4-10 VOC/year) Cohort 2- Participants with milder SCD (<4-10 VOC/year) Cohort 3- Healthy volunteers Part A and B can occur in parallel | ||
| Part B | Adults with SCD receiving chronic red blood cell exchange transfusion Part A and B can occur in parallel |
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| Measure | Description | 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Kidney blood flow rates as assessed by MRI-SWI (susceptibility-weighted imaging) | Up to day 21 post screening visit | |
| Skeletal muscle blood flow rates as assessed by NIRS | Up to day 21 post screening visit |
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Key Inclusion Criteria:
Have a diagnosis of SCD confirmed by hemoglobin analysis.
Be in stable clinical condition, as determined by the Investigator.
Subjects enrolled in Part B must also meet the following eligibility criterion at Screening:
Receiving scheduled standard of care RBC exchange transfusion therapy, with ≥3 transfusions already received.
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:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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Participants with mild to severe VOC
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Bioverativ Therapeutics Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Detroit | Michigan | 48201 | United States |
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| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
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Blood samples will be collected for hematology, blood chemistry, and venous blood gas analysis. Blood samples will also be used for exploratory biomarker development specific to SCD.
| 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 |
| Total oxygen levels in the kidney as assessed by MRI-SWI | 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 |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |