Early heart screening and treatment for people with sickle cell disease

Early Detection and Management of Cardiac Iron Overload in Sickle Cell Disease Using Multimodal Imaging for Improved Clinical Outcomes

This study tests whether regular heart checks and early iron-removing treatment can prevent heart problems and cut hospital stays in adults with sickle cell disease who have high iron or heart symptoms.

Quick facts

• Phase: PHASE2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Inova Health Care Services (other)
• Locations: 2 sites (Fairfax, Virginia and 1 other locations)
• Trial ID: NCT07023666 on ClinicalTrials.gov

What this trial studies

Participants will have cardiac imaging (ECG, echocardiogram, cardiac MRI) and lab testing every three months for one year to look for iron-related heart problems. Those with evidence of iron overload or cardiac findings may receive iron chelation (deferoxamine, deferasirox, or deferiprone) and guideline-directed heart medications. The trial enrolls adults with sickle cell disease, including up to about 80 people with ferritin ≥ 400 ng/mL and up to 20 with lower ferritin but cardiac symptoms. Outcomes include development or progression of cardiac complications and rates of hospitalization over the 12-month period.

Who should consider this trial

Good fit: Adults (≥18 years) with any sickle cell genotype who are willing to undergo regular cardiac imaging and follow-up, with ferritin ≥ 400 ng/mL (or lower ferritin with cardiac symptoms), and ECOG 0–1 are ideal candidates.

Not a fit: People with known heart failure, unstable angina, uncontrolled arrhythmias, severe renal or liver impairment, contraindication to chelation, active severe infections, pregnancy or inability to attend follow-up are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, this approach could prevent iron-related heart damage and reduce hospitalizations for adults with sickle cell disease.

How similar studies have performed: Iron chelation and routine cardiac screening have improved cardiac outcomes in transfusion-dependent conditions like thalassemia, but their specific benefit in sickle cell disease is less well established.

Eligibility criteria

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

Where this trial is running

Fairfax, Virginia and 1 other locations

• Inova Schar Cancer — Fairfax, Virginia, United States (RECRUITING)
• Inova Health Care Service — Falls Church, Virginia, United States (RECRUITING)

Study contacts

• Principal investigator: Sheinei Alan, MD — Inova Fairfax Hospital
• Study coordinator: Keary Jane't

Conditions: Sickle Cell Disease, Iron Overload, Cardiomyopathies, Arrhythmias