Early Screening and Treatment of Heart Complication in Sickle Cell Disease

Inova Health Care Services

Status and phase: Enrolling; Phase 2

Conditions: Sickle Cell Disease

Treatments: Drug: Deferoxamine; Drug: Deferiprone; Device: Electrocardiogram (ECG); Drug: Deferasirox; Device: Echocardiography

Study type: Interventional

Funder types: Other

Identifiers

NCT07023666

INOVA-2023-159

Details and patient eligibility

About

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.

Full 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.

Enrollment: 100 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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100 participants in 1 patient group

Intervention for Cardiac Iron Overload in Sickle Cell Patients

Experimental group

Description:

The study intervention consists of regular cardiac monitoring, iron chelation therapy and guideline-directed medical therapy (GDMT).

Treatment:

Device: Echocardiography

Drug: Deferasirox

Device: Electrocardiogram (ECG)

Drug: Deferiprone

Drug: Deferoxamine