NCT04166526Recruiting
A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Source: ClinicalTrials.gov · Last updated 12 Dec 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 8 (estimated)
- First submitted
- 15 Oct 2019
- Start date
- 25 Oct 2019
- Primary completion date
- 1 May 2027
- Lead sponsor
- Boston Children's Hospital
- Diseases
- Sickle cell disease
- NCT number
- NCT04166526
What you commit to
- Study type: Observational
- Planned participants: 8
- From 25 Oct 2019 to the primary completion date, 1 May 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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;
Other illness
- Electrical implants such as cardiac pacemakers or perfusion pumps;
- Pre-existing medical conditions including a likelihood of developing seizures or claustrophobic reactions;
- Any greater than normal potential for cardiac arrest;
Age
- Women of childbearing age who are seeking to become pregnant, who are breastfeeding, or who suspect they may be pregnant;
Other
- Ferromagnetic objects such as jewelry or metal clips;
- Subjects requiring sedation for MRI
Who the study is looking for
- 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
Age in the registry: 8 years to 18 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Boston Children's Hospital Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.