Early Heart Complication Screening for Sickle Cell Disease

Recruiting at 1 trial location

Overseen By Keary Jane't

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Inova Health Care Services

Disqualifiers: Heart failure, Angina, Dysrhythmias, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 3

Duration: 12 months

100 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if early heart screenings and treatments can prevent heart issues in people with sickle cell disease. It focuses on detecting and managing iron overload, a condition that can lead to heart problems. The trial tests three iron-chelating drugs—Deferasirox, Deferiprone, and Deferoxamine—to assess their effectiveness in controlling iron levels and reducing hospital visits. People with sickle cell disease who experience heart-related symptoms, such as shortness of breath or leg swelling, might be suitable candidates. Participants must be willing to undergo regular heart checks, including echocardiograms and MRIs. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to significant advancements in care.

Do I need to stop my current medications for this trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research shows that the treatments being tested—deferasirox, deferiprone, and deferoxamine—are generally safe for people with sickle cell disease who have excess iron from blood transfusions.

Deferasirox has been studied in people with sickle cell disease and effectively lowers iron levels. However, patients should be monitored for side effects during treatment. Common side effects, occurring in more than 5% of patients, include stomach pain and rash.

Deferiprone is another option. Studies indicate it is safe and well-tolerated in people with sickle cell disease. It has been used for many years, and safety data supports its use for managing excess iron.

Lastly, deferoxamine has been used for a long time and helps reduce iron levels in the body. Patients usually tolerate it well, but monitoring for side effects remains important.

In summary, research supports that all three treatments are generally safe for those with sickle cell disease, though monitoring for side effects is recommended. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments for sickle cell disease because they aim to address cardiac complications through a combination of iron chelation therapies: Deferasirox, Deferiprone, and Deferoxamine. These treatments work by removing excess iron from the body, which is crucial since patients with sickle cell disease often suffer from iron overload due to frequent blood transfusions. Unlike standard care options that might not specifically target cardiac iron buildup, this approach emphasizes regular cardiac monitoring alongside iron chelation, potentially offering a more targeted protection for the heart. This comprehensive strategy might lead to better management of heart-related issues in sickle cell patients, enhancing their quality of life.

What evidence suggests that this trial's treatments could be effective for heart complications in sickle cell disease?

Research has shown that deferasirox, deferiprone, and deferoxamine effectively reduce excess iron in people with sickle cell disease. In this trial, participants will receive one of these treatments as part of the intervention for cardiac iron overload. Deferasirox lowers iron levels in the liver and reduces serum ferritin, a blood marker indicating iron levels. Deferiprone has demonstrated a continued reduction in iron over time. Deferoxamine has been the preferred treatment for excess iron for decades, proving its ability to lower iron levels effectively. Each treatment helps manage excess iron, crucial for preventing complications like heart problems in sickle cell disease. ^{1 2 5 6 7}

Who Is on the Research Team?

Sheinei Alan, MD

Principal Investigator

Inova Fairfax Hospital

Are You a Good Fit for This Trial?

This trial is for individuals with sickle cell disease who may be at risk of heart complications due to iron overload. Participants should not be pregnant and must not have conditions that could interfere with the study or pose additional health risks.

Inclusion Criteria

I am 18 years old or older.

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)

I am fully active or can carry out light work.

Exclusion Criteria

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

Pregnancy or breastfeeding

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive regular cardiac monitoring, iron chelation therapy, and guideline-directed medical therapy

12 months

Regular visits every 3 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Deferasirox
• Deferiprone
• Deferoxamine

Trial Overview The study investigates if early heart screening using Echocardiography and Electrocardiogram (ECG), followed by treatment with iron chelators like Deferasirox, Deferiprone, or Deferoxamine can prevent cardiac issues in sickle cell patients.

How Is the Trial Designed?

Group I: Intervention for Cardiac Iron Overload in Sickle Cell Patients Experimental Treatment 5 Interventions

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

Who Is Running the Clinical Trial?

Inova Health Care Services

Lead Sponsor

Trials

80

Recruited

22,700+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/21592110/

Long-term safety and efficacy of deferasirox (Exjade) for up ... To date, there is a lack of long-term safety and efficacy data for iron chelation therapy in transfusion-dependent patients with sickle cell disease (SCD).

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6489379/

Deferasirox for managing transfusional iron overload in ... For four outcomes, namely liver iron concentration, serum ferritin, creatinine increase and satisfaction with treatment, it was judged as 'moderate' quality; ...

3. sciencedirect.com sciencedirect.com/science/article/pii/S0006497121060912

Transfusional Iron Overload in Sickle Cell Patients ... We report on the utilization and outcomes in Fe overloaded SCD patients who were prescribed the oral chelating agent deferasirox.

4. ashpublications.org ashpublications.org/blood/article/116/21/845/69827/Long-Term-Safety-and-Efficacy-of-Deferasirox

Long-Term Safety and Efficacy of Deferasirox (Exjade ® ) In ... The oral iron chelator, deferasirox, effectively reduced iron burden in SCD patients aged ≥2 years during 1 year of treatment (Vichinsky et al.

5. clinicaltrials.gov clinicaltrials.gov/study/NCT01511848

Study Of Efficacy,Safety of Combined Deferasirox and ... The primary outcome measure is to assess efficacy in lowering serum ferritin level(the change in serum ferritin compared to baseline) with combining DFP and ...

6. novartis.com novartis.com/us-en/sites/novartis_us/files/jadenu.pdf

jadenu.pdf Table 1 displays adverse reactions occurring in greater than 5% of deferasirox-treated beta-thalassemia patients. (Study 1), sickle cell disease patients (Study ...

7. sciencedirect.com sciencedirect.com/science/article/pii/S0268960X08700079

Review Long-term efficacy and safety of deferasirox It is recommended that patients treated with deferasirox are monitored regularly for iron status and adverse events, to ensure that an effective and tolerable ...