Cerebrovascular Reserve and White Matter Disease in Patients with Chronic Anemia
Completed
Conditions studied: Thalassemia, Sickle Cell Disease, Healthy Controls
In brief
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.
Key facts
- Study ID
- NCT03715972
- Run by
- Children's Hospital Los Angeles
- People needed
- 165
- Starts
- 2018-07-15
- Expected to finish
- 2022-12-31
- Last updated by the study team
- 2024-10-30
Who can join
Age: 7 and older. Sex: any. Healthy volunteers: accepted.
You may not qualify if…
- Hospitalization within one month
- Contraindication to acetazolamide use (seizures)
- Severe claustrophobia.
- Pregnancy or nursing (a negative HCG (pregnancy) test must be obtained prior to MRI)
- As a result of medical review, physical examination or screening investigations, the Principal Investigator (PI) considers the subject unfit for the study
- No fixed address
- In control subjects, chronic hepatitis, diabetes, hypertension, coronary artery disease, cognitively impaired or developmental delay
Where it is running
- CHLA — Los Angeles, California, United States
Full record on ClinicalTrials.gov
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