Sickle Cell DiseaseNCT04166526
A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
An observational study of Sickle Cell Disease, run by Boston Children's Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 to 18 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- FDNIRS-DCS and MRI
- Type of treatment
- Medical device
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Oct 2019to 1 Aug 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: In this pilot study, investigators plan on recruiting 8 children between the ages of 8-18 from hematology clinics.
Who may be able to join
- 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
Show all 11Show fewer
- 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 join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Boston Children's Hospital
Boston, Massachusetts, 02115
Jessie Sims, 617-919-1309, Jessica.Sims@childrens.harvard.edu
About this 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.
- Conditions
- Sickle Cell Disease
- Being tested
- FDNIRS-DCS, MRI
- Keywords
- Sickle Cell, MRI, Near Infrared Spectroscopy (NIRS)
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FDNIRS-DCSDevice
Also called Frequency-Domain Near-Infrared and Diffuse Correlation Spectroscopy
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.
MRIDevice
Also called Magnetic Resonance Imaging
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Device: FDNIRS-DCS; Device: 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. Receives: Device: FDNIRS-DCS; Device: 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. Receives: Device: FDNIRS-DCS; Device: 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. Receives: Device: FDNIRS-DCS; Device: MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain
Blood oxygenation of the brain will be measured using FDNIRS-DCS techniques and then compared with data from a simultaneous MRI scan.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Nov 2019
- Study start25 Oct 2019
- Primary completion1 May 2027 (estimated)
- Study completion1 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 8 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Principal investigator, Ellen Grant, Boston Children's Hospital
- Investigators
- Ellen Grant, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04166526
- Sponsor's protocol ID
- IRB-P00033322
- Other IDs
- 1OT2HL152640-01
- Status last verified
- Dec 2025
- First submitted
- 15 Oct 2019
- Last updated
- 12 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT04166526
- Acronym / sponsor ID
- IRB-P00033322
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle Cell, MRI, Near Infrared Spectroscopy (NIRS)
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 8 participants (estimated)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 25 Oct 2019 (actual)
- Primary completion
- 1 May 2027 (estimated)
- Study completion
- 1 Aug 2027 (estimated)
- First posted
- 18 Nov 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Group 1: Typically developing children | – | Device: FDNIRS-DCS, Device: MRIParticipants 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. |
| Group 2: Children with SCD not receiving treatment | – | Device: FDNIRS-DCS, Device: MRIParticipants 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. |
| Group 3: Children with SCD who have undergone gene therapy | – | Device: FDNIRS-DCS, Device: MRIParticipants 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. |
| Group 4: Children with SCD who have chronic transfusions | – | Device: FDNIRS-DCS, Device: MRIParticipants 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. |
Who may qualify
- Ages
- 8 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- In this pilot study, investigators plan on recruiting 8 children between the ages of 8-18 from hematology clinics.
Key inclusion criteria (11 listed)
- Children ages 8-18 without SCD
- SCD patients ages 8-18 who:
- SCD patients ages 8-18 who have had gene therapy at least one month prior to enrollment
- SCD patients ages 8-18 who receive chronic transfusions
Key exclusion criteria (7 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FDNIRS-DCSAlso: Frequency-Domain Near-Infrared and Diffuse Correlation SpectroscopyFDNIRS-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. | Device | Group 1: Typically developing children, Group 2: Children with SCD not receiving treatment, Group 3: Children with SCD who have undergone gene therapy, Group 4: Children with SCD who have chronic transfusions |
| MRIAlso: Magnetic Resonance ImagingParticipants 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. | Device | Group 1: Typically developing children, Group 2: Children with SCD not receiving treatment, Group 3: Children with SCD who have undergone gene therapy, Group 4: Children with SCD who have chronic transfusions |
Primary outcome
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ellen Grant, MD principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Sickle Cell Disease, run by Boston Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 12 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Ellen Grant, MD (857-218-5111, Ellen.Grant@childrens.harvard.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Accuracy of FDNIRS-DCS in measuring blood oxygenation of the brain (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2025.