Boston, MANCT04166526Now EnrollingIRB Ready

Sickle Cell Disease Clinical Trial in Boston, MA

Access cutting-edge sickle cell disease treatment through this clinical trial at a research site in Boston. Study-provided care at no cost to qualified participants.

Sponsored by Boston Children's Hospital

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Expert Care in Boston

Access sickle cell disease specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related sickle cell disease treatment provided free

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Check if you qualify for this sickle cell disease clinical trial in Boston, MA

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Why Participate?

  • No-Cost Study Care

  • Local to Boston

    Convenient for MA residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Boston site if eligible
  4. 4Begin participation

About This Sickle Cell Disease Study in Boston

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.

Sponsor: Boston Children's Hospital

Who Can Participate

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

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Boston?

Yes, this clinical trial (NCT04166526) has an active research site in Boston, MA that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Sickle Cell Disease Treatment Options in Boston, MA

If you're searching for sickle cell disease treatment options in Boston, MA, this clinical trial (NCT04166526) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Boston research site is actively enrolling participants for this clinical trial. You'll receive care from experienced sickle cell disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all sickle cell disease clinical trials near you to find additional studies recruiting in your area.

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