Actively Recruiting
A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Led by Boston Children's Hospital · Updated on 2025-12-12
8
Participants Needed
1
Research Sites
405 weeks
Total Duration
On this page
Sponsors
B
Boston Children's Hospital
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individual patients. This study proposes to monitor SCD and its treatment through their impact on cerebral hemodynamics, as the brain is one of the most vulnerable and consequential targets of the disease. Specifically, this study will optimize quantitative magnetic resonance imaging (MRI) and advanced optical spectroscopy techniques such as frequency-domain near-infrared and diffuse correlation spectroscopies (FDNIRS-DCS) to monitor 1) cerebral oxygen transport with measures of cerebral blood flow (CBF), cerebral oxygen extraction fraction (OEF) and cerebral metabolic rate of oxygen consumption (CMRO2) and 2) endothelial function with cerebrovascular reactivity (CVR). Additionally, this study aims to monitor baseline cerebral oxygen transport and CVR, as well as changes that occur with treatment (transfusion or genetic therapy to induce fetal hemoglobin) and assess hemoglobinopathy patients with known genotypes and phenotypes. The ultimate goal is to demonstrate the potential of this monitoring approach to select individual SCD subjects for interventions and evaluate individual responses to treatment. Success will help justify inclusion of these modalities in ongoing and future clinical trials of novel SCD therapies.
CONDITIONS
Official Title
A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Children ages 8 to 18 without sickle cell disease (healthy controls)
- Sickle cell disease patients ages 8 to 18 who have not had gene therapy or bone marrow transplant, are not on chronic transfusions, and have not received a blood transfusion in the past 3 months
- Sickle cell disease patients ages 8 to 18 who had gene therapy at least one month before enrollment
- Sickle cell disease patients ages 8 to 18 who receive chronic transfusions
You will not qualify if you...
- Having electrical implants such as cardiac pacemakers or perfusion pumps
- Having ferromagnetic implants like aneurysm clips, surgical clips, prostheses, artificial hearts, valves with steel parts, metal fragments, shrapnel, tattoos near the eye, or steel implants
- Having ferromagnetic objects including jewelry or metal clips
- Women of childbearing age who are seeking pregnancy, breastfeeding, or suspect pregnancy
- Pre-existing conditions that increase risk of seizures or claustrophobia
- Any condition with greater than normal risk of cardiac arrest
- Need for sedation during MRI
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
Research Team
E
Ellen Grant, MD
CONTACT
K
Katherine Eident, BS
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
4
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