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Clinical Trials/NCT04166526
NCT04166526
Recruiting
Not Applicable

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

Boston Children's Hospital1 site in 1 country8 target enrollmentOctober 25, 2019

Overview

Phase
Not Applicable
Intervention
Not specified
Conditions
Sickle Cell Disease
Sponsor
Boston Children's Hospital
Enrollment
8
Locations
1
Primary Endpoint
Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain
Status
Recruiting
Last Updated
4 months ago

Overview

Brief 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), 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.

Registry
clinicaltrials.gov
Start Date
October 25, 2019
End Date
August 1, 2027
Last Updated
4 months ago
Study Type
Observational
Sex
All

Investigators

Responsible Party
Principal Investigator
Principal Investigator

Ellen Grant

Director, FNNDSC

Boston Children's Hospital

Eligibility Criteria

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):

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, 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

Outcomes

Primary Outcomes

Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain

Time Frame: 6 months

Blood oxygenation of the brain will be measured using FDNIRS-DCS techniques and then compared with data from a simultaneous MRI scan.

Study Sites (1)

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