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Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia

NCT03715972 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 165

Last updated 2024-10-30

No results posted yet for this study

Summary

This is primarily an observational trial in patients with chronic anemia syndromes (sickle cell disease and thalassemia) and control subjects. The key purpose is to understand how brain blood flow reserve (the ability of the brain to increase its flow in response to stress) is altered in patients with chronic anemia. Since this parameter may depend on anemia severity, we will perform the MRI monitoring prior to and following clinically indicated transfusions in a subset of patients. Most patients will already be prescribed hydroxyurea as part of their standard of care. Since hydroxyurea could impact brain blood flow, there is also a small pilot study (20 patients, nonrandomized, open label) where MRI imaging will be performed prior to and following administration of hydroxyurea up to maximum tolerated dose. The study will enroll 90 adult subjects with transfusion independent sickle cell disease (70 SS, 10 SC, 10 Sβ0) and 60 patients with transfusion-dependent sickle cell disease. It will also include 10 transfusion independent thalassemia patients and 20 transfusion dependent thalassemia patients as well as 40 control subjects recruited from first degree relatives of the sickle cell disease population. All eligible subjects will be asked to provide informed consent before participating in the study.

Conditions

Interventions

DRUG

Hydroxyurea

info included in arm group

DRUG

Acetazolamide

Acetazolamide will not be considered a treatment; however, it will be used as a tool to help measure cerebralvascular reserve. A dose of 16 mg/kg ACZ will be administered with a maximum of 1400 mg.

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

    collaborator OTHER

  • Vanderbilt University Medical Center

    collaborator OTHER

  • Children's Hospital Los Angeles

    lead OTHER

Principal Investigators

  • John C Wood, M.D., PhD · CHLA/USC

Eligibility

Min Age
7 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2018-07-15
Primary Completion
2022-12-31
Completion
2022-12-31

Countries

  • United States

Study Locations

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Diseases
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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03715972 on ClinicalTrials.gov