Early Screening and Treatment of Heart Complication in Sickle Cell Disease

NCT07023666 | Recruiting Phase 2 DMC FDA Drug FDA Device

• 1 other identifier: INOVA-2023-159
• Study Type: interventional
• Target: 100
• Locations: 1 country
• Sites: 2

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.

Conditions

Sickle Cell Disease

Keywords

Iron Overload; Cardiomyopathies; Arrhythmias

Outcome Measures

Primary Outcomes (2)

• Incidence of Hospitalization for Sickle Cell Disease Related Complications

  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

  Up to 12 months

• Length of Stay for SCD-Related Complications

  The total number of days spent in the hospital per admission for complications related to SCD.

  Up to 12 Months

Secondary Outcomes (2)

• Incidence of Left and/or Right Heart Failure

  Up to 12 Months

• Incidence of Atrial or Ventricular Arrhythmia or Conduction Abnormalities

  Up to 12 Months

Study Arms (1)

Intervention for Cardiac Iron Overload in Sickle Cell Patients

EXPERIMENTAL

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

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

Interventions

Deferoxamine DRUG

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.

Also known as: Desferal

Intervention for Cardiac Iron Overload in Sickle Cell Patients

Deferasirox DRUG

Deferasirox is used for iron chelation therapy based on iron burden assessment throughout the study.

Also known as: Jadenu, Exjade

Intervention for Cardiac Iron Overload in Sickle Cell Patients

Deferiprone DRUG

Deferiprone is used for iron chelation therapy used throughout the study.

Also known as: Ferriprox

Intervention for Cardiac Iron Overload in Sickle Cell Patients

Echocardiography DEVICE

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.

Intervention for Cardiac Iron Overload in Sickle Cell Patients

Electrocardiogram (ECG) DEVICE

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.

Intervention for Cardiac Iron Overload in Sickle Cell Patients

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Inova Health Care Services: lead

Study Sites (2)

Inova Schar Cancer

Fairfax, Virginia, 22031, United States

RECRUITING

Inova Health Care Service

Falls Church, Virginia, 22042, United States

RECRUITING

Related Publications (7)

• Fitzhugh CD, Lauder N, Jonassaint JC, Telen MJ, Zhao X, Wright EC, Gilliam FR, De Castro LM. Cardiopulmonary complications leading to premature deaths in adult patients with sickle cell disease. Am J Hematol. 2010 Jan;85(1):36-40. doi: 10.1002/ajh.21569.

  PMID: 20029950 BACKGROUND

• Hamideh D, Alvarez O. Sickle cell disease related mortality in the United States (1999-2009). Pediatr Blood Cancer. 2013 Sep;60(9):1482-6. doi: 10.1002/pbc.24557. Epub 2013 Apr 23.

  PMID: 23637037 BACKGROUND

• Lanzkron S, Carroll CP, Haywood C Jr. Mortality rates and age at death from sickle cell disease: U.S., 1979-2005. Public Health Rep. 2013 Mar-Apr;128(2):110-6. doi: 10.1177/003335491312800206.

  PMID: 23450875 BACKGROUND

• Whipple NS, Naik RJ, Kang G, Moen J, Govindaswamy SD, Fowler JA, Dowdy J, Penkert R, Joshi VM, Hankins JS. Ventricular global longitudinal strain is altered in children with sickle cell disease. Br J Haematol. 2018 Dec;183(5):796-806. doi: 10.1111/bjh.15607. Epub 2018 Nov 19.

  PMID: 30450553 BACKGROUND

• Darbari DS, Sheehan VA, Ballas SK. The vaso-occlusive pain crisis in sickle cell disease: Definition, pathophysiology, and management. Eur J Haematol. 2020 Sep;105(3):237-246. doi: 10.1111/ejh.13430. Epub 2020 May 19.

  PMID: 32301178 BACKGROUND

• Modell CB. Haemoglobinopathies. The pathophysiology of beta-thalassaemia major. J Clin Pathol Suppl (R Coll Pathol). 1974;8:12-8. No abstract available.

  PMID: 4536357 BACKGROUND

• Taher AT, Saliba AN. Iron overload in thalassemia: different organs at different rates. Hematology Am Soc Hematol Educ Program. 2017 Dec 8;2017(1):265-271. doi: 10.1182/asheducation-2017.1.265.

  PMID: 29222265 BACKGROUND

MeSH Terms

Conditions

Anemia, Sickle Cell; Iron Overload; Cardiomyopathies; Arrhythmias, Cardiac

Interventions

Deferoxamine; Deferasirox; Deferiprone; Echocardiography; Electrocardiography

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Iron Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Heart Diseases; Cardiovascular Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Hydroxamic Acids; Hydroxylamines; Amines; Organic Chemicals; Hydroxy Acids; Carboxylic Acids; Benzoates; Acids, Carbocyclic; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Triazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Pyridones; Pyridines; Cardiac Imaging Techniques; Diagnostic Imaging; Diagnostic Techniques and Procedures; Diagnosis; Ultrasonography; Heart Function Tests; Diagnostic Techniques, Cardiovascular; Electrodiagnosis

Study Officials

• Sheinei Alan, MD

  Inova Fairfax Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Keary Jane't

CONTACT

571-472-3173; keary.jane't@inova.org

Elahe Mollapour

CONTACT

571-472-0615; elahe.mollapour@inova.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 9, 2025
• First Posted: June 17, 2025
• Study Start: October 7, 2025
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): June 30, 2027
• Last Updated: October 14, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)