Overview

This trial has been completed.

Conditions healthy, sickle cell disease
Sponsor Biogen
Start date June 2015
End date October 2016
Trial size 38 participants
Trial identifier NCT02447627, 996SC001

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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Observational model cohort
Time perspective prospective
Arm
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
Adults with SCD receiving chronic red blood cell exchange transfusion Part A and B can occur in parallel

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)
time frame: Up to day 18 post screening visit

Secondary Outcomes

Measure
Kidney blood flow rates as assessed by MRI-SWI (susceptibility-weighted imaging)
time frame: Up to day 21 post screening visit
Skeletal muscle blood flow rates as assessed by NIRS
time frame: Up to day 21 post screening visit
Skin blood flow rates as assessed by LSCI
time frame: Up to day 21 post screening visit
Retinal blood flow rates as assessed by OI
time frame: Up to day 21 post screening visit
Total oxygen levels in the brain as assessed by MRI-ASL
time frame: Up to day 21 post screening visit
Total oxygen levels in the kidney as assessed by MRI-SWI
time frame: Up to day 21 post screening visit
Total oxygen levels in the muscle as assessed by NIRS
time frame: Up to day 21 post screening visit
Total oxygen levels in the skin as assessed by LSCI
time frame: Up to day 21 post screening visit
Total oxygen levels in the retina as assessed by OI
time frame: Up to day 21 post screening visit

Eligibility Criteria

Male or female participants from 18 years up to 45 years old.

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.

Additional Information

Official title A Study of Methodologies to Measure Blood Flow and Oxygenation as Potential Biomarkers in Adults With Sickle Cell Disease
Trial information was received from ClinicalTrials.gov and was last updated in November 2016.
Information provided to ClinicalTrials.gov by Biogen.