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NCT05861453RECRUITINGIRB Ready

Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients with Sickle Cell Disease

Sponsored by Afimmune

Quick Self-Assessment

See if you qualify and express your interest

Preparing your pre-screening questions...
๐Ÿ“Š
1 of 30 participants interested
3% interest

Express Your Interest Today

Interested in learning more? Complete the form below.

Secure & Confidential

Your information is protected and will only be shared with the research team.

Why Participate?

  • No-Cost Study Care

  • Advance Medical Science

    Contribute to groundbreaking research

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel (varies by study)

  • Help Others

    Benefit future patients

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3In-person assessment if eligible
  4. 4Begin participation

About This Study

To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged โ‰ฅ18 years.

Who Can Participate

Inclusion Criteria

Patients with sickle cell disease (SCD) including:
2 sickle hemoglobin genes \[HbSS\]
HbSฮฒ0 thalassemia
HbSฮฒ+ thalassemia
Heterozygous for hemoglobin S and hemoglobin C \[HbSC\]
Male or female patients aged 18 years and older on the day of signing the informed consent form (ICF)
Patients who have had between 2 and 15 episodes of vaso-occlusive crisis (VOC) in the past year (12 months)
For patients taking hydroxyurea (HU), the dose of HU must be stable for at least 3 months prior to signing the ICD and with no anticipated need for dose adjustment during the study.
Female patients and male patients with female partners of childbearing potential must use highly effective contraceptive methods for the duration of the study.

Exclusion Criteria

Patients who are receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion), have received an RBC transfusion for any reason within

โ“ Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility during the screening process.

Study Details

๐Ÿ“‹

Study Type

INTERVENTIONAL

๐Ÿ‘ฅ

Target Enrollment

30 participants

โšง

Gender

ALL

๐Ÿข

Sponsor

Afimmune

Study Locations

Choose your preferred location or select flexible during enrollment

Unknown

Birmingham

Alabama

Location available
View Birmingham location page
Unknown

Farmington

Connecticut

Location available
View Farmington location page
Unknown

Washington D.C.

District of Columbia

Location available
View Washington D.C. location page
Unknown

Atlanta

Georgia

Location available
View Atlanta location page
Unknown

Atlanta

Georgia

Location available
View Atlanta location page
Unknown

Atlanta

Georgia

Location available
View Atlanta location page
Unknown

Chicago

Illinois

Location available
View Chicago location page
Unknown

Baltimore

Maryland

Location available
View Baltimore location page
Unknown

Bethesda

Maryland

Location available
View Bethesda location page

๐Ÿ“ And 7 more locations available

Frequently Asked Questions

Q:What is a clinical trial?

A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.

Q:Will I be compensated?

Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.

Q:Will I receive a placebo instead of treatment?

When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.

Q:Can I leave a trial if I change my mind?

Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.

Q:How long does a clinical trial last?

Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.

Still have questions? Our study coordinators are here to help.

Ready to Make a Difference?

Express your interest in this groundbreaking clinical trial

๐Ÿ”’ Secure ยท ๐Ÿฅ Expert Care

Looking for Sickle Cell Disease Treatment in Birmingham?

Join others in Alabama exploring innovative treatment options through clinical research

Sickle Cell Disease Treatment Options in Birmingham, Alabama

If you're searching for Sickle Cell Disease treatment in Birmingham, participating in a clinical research study may provide access to innovative approaches under expert medical supervision. This study is actively recruiting participants in Birmingham, Farmington, Washington D.C. and surrounding areas.

Clinical trials offer participants the opportunity to receive cutting-edge treatments while contributing to medical research that may help future patients with Sickle Cell Disease. All study-related care is provided at no cost to participants.

Local Sites
3 locations in Alabama
Now Enrolling
Up to 30 participants
Quick Start
Screening available now

Why Consider a Clinical Trial for Sickle Cell Disease?

Potential Benefits

  • โœ“Access to new treatment approaches before public availability
  • โœ“Close monitoring by experienced medical professionals
  • โœ“Study-related care provided at no cost
  • โœ“Contribute to medical research for Sickle Cell Disease

What to Expect

  • โ†’Initial screening to determine eligibility
  • โ†’Regular check-ups and monitoring visits
  • โ†’Possible compensation for time and travel
  • โ†’You can withdraw at any time

Frequently Asked Questions About This Sickle Cell Disease Study

Important Clinical Trial Information

This information is provided for educational purposes and does not constitute medical advice. Clinical trial participation involves potential risks and benefits. Eligibility requirements apply and will be assessed during the screening process.

Study identifier: NCT05861453. For complete study details, visit ClinicalTrials.gov. Always consult with your healthcare provider before making decisions about your medical care or participating in clinical research.