Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease

Completed

• Conditions: Healthy; Sickle Cell Disease

• Registration Number: NCT02447627
• Lead Sponsor: Bioverativ Therapeutics Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-04-16
• Study First Submitted QC: 2015-05-14
• Study First Posted: 2015-05-19
• Study Start: 2015-06
• Primary Completion: 2016-10
• Study Completion: 2016-10
• Status Verified: 2017-08
• Last Update Submitted: 2020-12-16
• Last Update Posted: 2020-12-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 38

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
Skin blood flow rates as assessed by LSCI	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
Skeletal muscle blood flow rates as assessed by NIRS	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 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 muscle as assessed by NIRS	Up to day 21 post screening visit

Trial Locations

Locations (1)

Research Site; 🇺🇸 Detroit, Michigan, United States