A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
概览
- 阶段
- 不适用
- 干预措施
- 未指定
- 疾病 / 适应症
- Sickle Cell Disease
- 发起方
- Boston Children's Hospital
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain
- 状态
- 招募中
- 最后更新
- 4个月前
概览
简要总结
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.
研究者
Ellen Grant
Director, FNNDSC
Boston Children's Hospital
入排标准
入选标准
- •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):
排除标准
- •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
结局指标
主要结局
Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain
时间窗: 6 months
Blood oxygenation of the brain will be measured using FDNIRS-DCS techniques and then compared with data from a simultaneous MRI scan.