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Brain Blood Flow and Oxygen Imaging in Sickle Cell Disease Patients

Uses MRI and light-based scans to study brain blood flow in sickle cell disease.

Key facts

Who runs it: Boston Children's Hospital
Where: Boston, United StatesSee the site
Trial code: NCT04166526
Registry title: A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Status: Recruiting
People taking part: 8 people, planned
Started: 25 Oct 2019
Expected to end: 1 Aug 2027
Type of study: Observational, 4 groups
Treatments tested: FDNIRS-DCS
MRI
Record updated: 12 December 2025

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for children aged 8 to 18 with sickle cell disease, in three groups by treatment, and for children without the disease as a comparison group.

Age: 8 to 18 years
Sex: All sexes can join.
Disease stage: Sickle cell disease. Groups are untreated, after gene therapy, or on chronic transfusions.
Earlier treatment: Untreated group: no gene therapy, no bone marrow transplant, no chronic transfusions, no transfusion in the past 3 months. Gene therapy group: gene therapy at least one month before joining. Transfusion group: gets chronic transfusions.

Main reasons for not taking part

Electrical implants such as pacemakers or perfusion pumps
Metal implants or metal objects that are not safe in an MRI scan
Women of childbearing age who are seeking pregnancy, breastfeeding, or may be pregnant
Likely to have seizures or claustrophobic reactions
Needing sedation for MRI

The registry criteria, word for word

Show all 12 criteriaShow less

Who can take part

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

Who cannot take part

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
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
Women of childbearing age who are seeking to become pregnant, who are breastfeeding, or who suspect they may be pregnant
Ferromagnetic objects such as jewelry or metal clips
Subjects requiring sedation for MRI

Check if you might fit

Answer for yourself. This is not a decision: only the study team can say whether you can take part.

Are you between 8 and 18 years old?

Where in United States

Boston Children's HospitalBoston, United StatesContact: Jessie SimsTaking people

About the trial

What the trial wants to find out

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).

Read full original descriptionShow less

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.

The 4 groups in the trial

Group 1: Typically developing children
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.
Treatment: FDNIRS-DCS and MRI
Group 2: Children with SCD not receiving treatment
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.
Treatment: FDNIRS-DCS and MRI
Group 3: Children with SCD who have undergone gene therapy
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.
Treatment: FDNIRS-DCS and MRI
Group 4: Children with SCD who have chronic transfusions
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.
Treatment: FDNIRS-DCS and MRI