A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
- Conditions
- Sickle Cell Disease
- Interventions
- Device: FDNIRS-DCSDevice: MRI
- Registration Number
- NCT04166526
- Lead Sponsor
- Boston Children's Hospital
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 8
Group 1 (healthy controls):
- Children ages 8-18 without SCD
Group 2 (SCD patients without treatment):
-
SCD patients ages 8-18 who:
- have not undergone gene therapy or a bone marrow transplant
- are not receiving chronic transfusions
- have not received a blood transfusion in the past 3 months
Group 3 (SCD patients who have undergone gene therapy):
- SCD patients ages 8-18 who have had gene therapy at least one month prior to enrollment
Group 4 (SCD patients who have chronic transfusions):
- SCD patients ages 8-18 who receive chronic transfusions
- Electrical implants such as cardiac pacemakers or perfusion pumps;
- Ferromagnetic implants such as aneurysm clips, surgical clips, prosthesis, artificial hearts, valves with steel parts, metal fragments, shrapnel, tattoos near the eye, or steel implants;
- Ferromagnetic objects such as jewelry or metal clips;
- Women of childbearing age who are seeking to become pregnant, who are breastfeeding, or who suspect they may be pregnant;
- Pre-existing medical conditions including a likelihood of developing seizures or claustrophobic reactions;
- Any greater than normal potential for cardiac arrest;
- Subjects requiring sedation for MRI
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Group 2: Children with SCD not receiving treatment MRI Participants of this group have a diagnosis of SCD, but do not receive chronic transfusions, gene therapy or bone marrow transplants. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 2: Children with SCD not receiving treatment FDNIRS-DCS Participants of this group have a diagnosis of SCD, but do not receive chronic transfusions, gene therapy or bone marrow transplants. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 1: Typically developing children FDNIRS-DCS Participants of this group will not have a diagnosis of SCD. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 4: Children with SCD who have chronic transfusions MRI Participants of this group have a diagnosis of SCD and receive chronic transfusions. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 1: Typically developing children MRI Participants of this group will not have a diagnosis of SCD. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 3: Children with SCD who have undergone gene therapy FDNIRS-DCS Participants of this group have a diagnosis of SCD and have had gene therapy at least one month prior to enrollment. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 3: Children with SCD who have undergone gene therapy MRI Participants of this group have a diagnosis of SCD and have had gene therapy at least one month prior to enrollment. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring. Group 4: Children with SCD who have chronic transfusions FDNIRS-DCS Participants of this group have a diagnosis of SCD and receive chronic transfusions. These participants will undergo an MRI, lasting approximately an hour, with simultaneous FDNIRS-DCS monitoring.
- Primary Outcome Measures
Name Time Method Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain 6 months Blood oxygenation of the brain will be measured using FDNIRS-DCS techniques and then compared with data from a simultaneous MRI scan.
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (1)
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States