A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Di
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 st
| Condition(s) | Sickle Cell Disease |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| 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 |
| Who can participate | Inclusion Criteria: 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 Exclusion Criteria: * 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 |
| Ages | 8 Years to 18 Years |
| Sex | All |
| Lead sponsor | Boston Children's Hospital |
| Locations | Boston, Massachusetts, United States |
| Start date | 2019-10-25 |
| NCT ID | NCT04166526 |
| Official listing | https://clinicaltrials.gov/study/NCT04166526 |