Clinical trial NCT02447627: Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease
Official title: A Study of Methodologies to Measure Blood Flow and Oxygenation as Potential Biomarkers in Adults With Sickle Cell Disease
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Healthy
Sickle Cell Disease
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2015-05-19
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Participants with mild to severe VOC

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.


Eligibility 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/Exclusion criteria may apply.

Primary outcomes:
measure: 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)
timeFrame: Up to day 18 post screening visit
Secondary outcomes:
measure: Kidney blood flow rates as assessed by MRI-SWI (susceptibility-weighted imaging)
timeFrame: Up to day 21 post screening visit
measure: Skeletal muscle blood flow rates as assessed by NIRS
timeFrame: Up to day 21 post screening visit
measure: Skin blood flow rates as assessed by LSCI
timeFrame: Up to day 21 post screening visit
measure: Retinal blood flow rates as assessed by OI
timeFrame: Up to day 21 post screening visit
measure: Total oxygen levels in the brain as assessed by MRI-ASL
timeFrame: Up to day 21 post screening visit
measure: Total oxygen levels in the kidney as assessed by MRI-SWI
timeFrame: Up to day 21 post screening visit
measure: Total oxygen levels in the muscle as assessed by NIRS
timeFrame: Up to day 21 post screening visit
measure: Total oxygen levels in the skin as assessed by LSCI
timeFrame: Up to day 21 post screening visit
measure: Total oxygen levels in the retina as assessed by OI
timeFrame: Up to day 21 post screening visit
Other outcomes:
Locations:
city: Detroit
country: United States
facility: Research Site
state: Michigan
Study officials:
affiliation: Bioverativ Therapeutics Inc.
name: Medical Director
role: STUDY_DIRECTOR
References: