Monitoring brain health in children with Sickle Cell Disease

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

This study is trying to see how Sickle Cell Disease affects brain health in children aged 8-18 by using special imaging techniques to check blood flow and oxygen levels in the brain.

Quick facts

• Study type: Observational
• Enrollment: 8 (estimated)
• Ages: 8 Years to 18 Years
• Sex: All
• Sponsor: Boston Children's Hospital (Academic / other)
• Locations: 1 site (Boston, Massachusetts)
• Trial ID: NCT04166526 on ClinicalTrials.gov

What this trial studies

This observational study aims to monitor the effects of Sickle Cell Disease (SCD) on cerebral hemodynamics in children aged 8-18 using advanced imaging techniques. It will utilize quantitative magnetic resonance imaging (MRI) and near-infrared spectroscopy (NIRS) to assess cerebral blood flow, oxygen extraction, and endothelial function. By identifying biomarkers for when intervention is necessary, the study seeks to improve treatment outcomes for SCD patients. The study includes healthy controls and various groups of SCD patients based on their treatment history.

Who should consider this trial

Good fit: Ideal candidates include children aged 8-18 with Sickle Cell Disease who have not undergone gene therapy or bone marrow transplant, as well as those who have received chronic transfusions or gene therapy.

Not a fit: Patients with electrical or ferromagnetic implants, or those seeking pregnancy, may not benefit from this study due to safety concerns with the imaging techniques used.

Why it matters

Potential benefit: If successful, this study could provide critical insights into the timing and effectiveness of interventions for children with Sickle Cell Disease.

How similar studies have performed: While the use of advanced imaging techniques in SCD is promising, this specific approach is novel and has not been extensively tested in prior studies.

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):

* 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

Where this trial is running

Boston, Massachusetts

• Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)

Study contacts

• Principal investigator: Ellen Grant, MD — Boston Children's Hospital
• Study coordinator: Ellen Grant, MD

Conditions Sickle Cell Disease; Sickle Cell; MRI; Near Infrared Spectroscopy