Sickle Cell Disease
Conditions
Keywords
Iron Overload, Cardiomyopathies, Arrhythmias
Brief summary
This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.
Detailed description
The goal of the study is to detect iron-related heart problems in subjects with sickle cell disease (SCD) through regular cardiac imaging (electrocardiogram, echocardiogram, cardiac MRI) and lab testing every 3 months during the 12 months period. The treatment will be with iron chelation and guideline-directed heart medications.
Interventions
DRUGDeferoxamine
Deferoxamine is used to reduce excess iron accumulation after monitoring iron levels. Adjustment to therapy will be based on iron burden assessments throughout the study duration.
DRUGDeferasirox
Deferasirox is used for iron chelation therapy based on iron burden assessment throughout the study.
DRUGDeferiprone
Deferiprone is used for iron chelation therapy used throughout the study.
DEVICEEchocardiography
This device uses ultrasound waves to create images of heart to help evaluate the heart's structure and function. This allows the detection of abnormalities of heart due to iron overload through out the study.
DEVICEElectrocardiogram (ECG)
The Electrocardiogram (ECG) device records the electrical activity of the heart. It is crucial for identifying arrhythmias and conduction abnormalities, which can be exacerbated by iron accumulation in the heart.
Sponsors
Inova Health Care Services
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* At least 18 years of age * Diagnosis of sickle cell disease (any genotype) * Serum ferritin levels ≥ 400 ng/mL (up to 80 patients), or less than 400 ng/mL in those who have cardiac symptoms including shortness of breath and lower extremity edema (up to 20 patients) * Willingness to undergo regular imaging (echocardiograms, ECG, cardiac MRI) * ECOG performance status of 0-1 * Able to read, understand and provide written informed consent * Deemed appropriate for participation by the treating physician
Exclusion criteria
* Unable to schedule and required follow-up visits * Medical comorbidities including: * Known heart failure * Unstable angina * Uncontrolled dysrhythmias * Acute pulmonary embolism * Active infection or severe comorbid conditions that in the view of the investigator would limit participation * History of hypersensitivity or contraindication to chelation therapy * Severe renal or hepatic impairment * Pregnancy of breastfeeding
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Hospitalization for Sickle Cell Disease Related Complications | Up to 12 months | Total number of participants hospitalized for SCD related complications including vaso-occlusive crises, acute chest syndrome and infection detect the impact of early intervention of cardiac iron overload in patients with Sickle Cell Disease |
| Length of Stay for SCD-Related Complications | Up to 12 Months | The total number of days spent in the hospital per admission for complications related to SCD. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Left and/or Right Heart Failure | Up to 12 Months | Total number of participants who develop left and/or right heart failure is estimated using descriptive statistics with 95% confidence intervals. |
| Incidence of Atrial or Ventricular Arrhythmia or Conduction Abnormalities | Up to 12 Months | The number of participants with conditions such as atrial fibrillation, ventricular tachycardia, or conduction system disease. Reported with 95% confidence intervals. |
Countries
United States
Contacts
Primary ContactKeary Jane't
Backup ContactElahe Mollapour
Outcome results
None listed