A Pilot Study Using MRI and Optical Techniques to Monitor Brain Oxygen and Blood Flow in Children with Sickle Cell Disease Ages 8 to 18

What this Trial Is About

Sickle Cell Disease SCD affects oxygen transport and causes damage to blood vessels, especially impacting the brain. Researchers are evaluating new ways to monitor SCD and its treatments by measuring brain blood flow, oxygen use, and blood vessel function using advanced MRI and optical techniques. This study aims to find biomarkers to guide when treatment is needed and measure how well treatments work for each patient, helping to personalize care and support future SCD therapies. Participants are grouped based on their SCD status and treatments healthy children without SCD, children with SCD not receiving treatment, children who have had gene therapy, and children on chronic transfusions. All participants undergo an MRI scan lasting about an hour combined with simultaneous optical monitoring. Those on chronic transfusions have additional MRI scans and optical tests before, during, and after transfusions to track changes. During the study, participants will have one or two MRI sessions with simultaneous optical measurements to assess brain oxygen transport and blood vessel response. Researchers will collect data on how these measurements change with treatment and compare groups with different SCD statuses. The main outcome is the accuracy of the optical device FDNIRS-DCS in measuring brain blood oxygen levels over six months. The study runs until August 2027 and involves children aged 8 to 18 years.

A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease