Cerebrovascular Reserve and White Matter Disease in Patients With Chronic Anemia

About the study

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.

Study point of contact

Candice Moulder, MPh, CCRP
3233611646
[email protected]
Conley Kelley, MS
323.361.6699
[email protected]

Locations

1 United States site

Age

> 7 Years

Study type

Observational

Gender

All

Interventions

Drug

Compensation

Unknown

Gender Description

for healthy controls we will try to match gender to those that have been enrolled under the anemia cohort

participation requirements

1. Diagnosis of sickle cell disease (genotype SS, SC, or SB0), thalassemia
(transfusion dependent or transfusion independent), or normal control subject
that are ≥18yrs, ethnicity, and sex matched to the sickle cell disease
population.

2. Ability to tolerate a one hour MRI examination.

3. Age equal to or greater than 7 years old for Anemia groups.

4. Agreeable to use an approved method of contraception for the entire duration of
hydroxyurea usage if accepted onto the hydroxyurea substudy (male or female of
childbearing potential)

participation restrictions

1. Hospitalization within one month

2. Contraindication to acetazolamide use (seizures)

3. Severe claustrophobia.

4. Pregnancy or nursing (a negative HCG (pregnancy) test must be obtained prior to MRI)

5. As a result of medical review, physical examination or screening investigations, the
Principal Investigator (PI) considers the subject unfit for the study

6. No fixed address

7. In control subjects, chronic hepatitis, diabetes, hypertension, coronary artery
disease, cognitively impaired or developmental delay

Locations

  • Los Angeles, California, United States, CHLA, 90027 [Recruiting]
Last updated 2021-05-07