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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04166526
Recruitment Status : Recruiting
First Posted : November 18, 2019
Last Update Posted : August 11, 2022
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Ellen Grant, Boston Children's Hospital

Study Description
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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.


Condition or disease Intervention/treatment
Sickle Cell Disease Device: FDNIRS-DCS Device: MRI

Study Design
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Study Type : Observational
Estimated Enrollment : 8 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
Actual Study Start Date : October 25, 2019
Estimated Primary Completion Date : May 1, 2024
Estimated Study Completion Date : August 1, 2025

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
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.
 Device: FDNIRS-DCS
FDNIRS-DCS measurements will be performed at the same time as all MRI scans. Participants in Group 4 will have additional measurements before, during, and after the their scheduled transfusion.
Other Name: Frequency-Domain Near-Infrared and Diffuse Correlation Spectroscopy

Device: MRI
Participants in Groups 1-3 will receive a single, hour-long MRI. Participants in Group 4 will receive two, one hour long MRI scans: one within a week prior to their transfusion and one within a week after their transfusion.
Other Name: Magnetic Resonance Imaging
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.
 Device: FDNIRS-DCS
FDNIRS-DCS measurements will be performed at the same time as all MRI scans. Participants in Group 4 will have additional measurements before, during, and after the their scheduled transfusion.
Other Name: Frequency-Domain Near-Infrared and Diffuse Correlation Spectroscopy

Device: MRI
Participants in Groups 1-3 will receive a single, hour-long MRI. Participants in Group 4 will receive two, one hour long MRI scans: one within a week prior to their transfusion and one within a week after their transfusion.
Other Name: Magnetic Resonance Imaging
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.
 Device: FDNIRS-DCS
FDNIRS-DCS measurements will be performed at the same time as all MRI scans. Participants in Group 4 will have additional measurements before, during, and after the their scheduled transfusion.
Other Name: Frequency-Domain Near-Infrared and Diffuse Correlation Spectroscopy

Device: MRI
Participants in Groups 1-3 will receive a single, hour-long MRI. Participants in Group 4 will receive two, one hour long MRI scans: one within a week prior to their transfusion and one within a week after their transfusion.
Other Name: Magnetic Resonance Imaging
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.
 Device: FDNIRS-DCS
FDNIRS-DCS measurements will be performed at the same time as all MRI scans. Participants in Group 4 will have additional measurements before, during, and after the their scheduled transfusion.
Other Name: Frequency-Domain Near-Infrared and Diffuse Correlation Spectroscopy

Device: MRI
Participants in Groups 1-3 will receive a single, hour-long MRI. Participants in Group 4 will receive two, one hour long MRI scans: one within a week prior to their transfusion and one within a week after their transfusion.
Other Name: Magnetic Resonance Imaging


Outcome Measures
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Primary Outcome Measures :
  1. 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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   8 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
In this pilot study, investigators plan on recruiting 8 children between the ages of 8-18 from hematology clinics.
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):

  • 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, 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04166526


Contacts
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Contact: Ellen Grant, MD 857-218-5111 Ellen.Grant@childrens.harvard.edu
Contact: Katherine Eident, BS 617-355-2184 katherine.eident@childrens.harvard.edu

Locations
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United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Jessie Sims    617-919-1309    Jessica.Sims@childrens.harvard.edu   
Sponsors and Collaborators
Boston Children's Hospital
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
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Principal Investigator: Ellen Grant, MD Boston Children's Hospital
More Information
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Responsible Party: Ellen Grant, Director, FNNDSC, Boston Children's Hospital
ClinicalTrials.gov Identifier: NCT04166526    
Other Study ID Numbers: IRB-P00033322
1OT2HL152640-01 ( U.S. NIH Grant/Contract )
First Posted: November 18, 2019    Key Record Dates
Last Update Posted: August 11, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ellen Grant, Boston Children's Hospital:
Sickle Cell
MRI
Near Infrared Spectroscopy (NIRS)
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
 Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn