A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease

Study point of contact

Katherine Eident, BS
617-355-2184
[email protected]
Ellen Grant, MD
857-218-5111
[email protected]

Locations

1 United States site

Age

8 to 18 Years

Study type

Observational

Gender

All

Interventions

Device

Compensation

Unknown

About the study

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.

participation requirements

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

participation restrictions

– 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

Locations

  • Boston, Massachusetts, United States, Boston Children's Hospital, 02115 [Recruiting]
Last updated 2020-07-24 Enroll Now