Clinical Trials
Clinical trials are medical research studies that test how safe and effective new treatments, medications, or procedures are for people living with sickle cell disease (SCD). They are an essential part of scientific progress—without them, new therapies (like medications, gene therapies, or bone marrow transplant approaches) cannot be approved for wider use.
How Clinical Trials Work
Clinical trials typically progress in different phases, each with a specific purpose:
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Phase 1 tests safety and whether a treatment causes any major side effects.
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Phase 2 begins to assess whether the treatment may work and the best dose.
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Phase 3 compares the new treatment to standard care in a larger group of people.
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Phase 4 occurs after a treatment is approved to explore new uses or long-term effects.
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Throughout every phase, researchers must follow strict safety rules and regulators (like the FDA and independent review boards) monitor the study closely.
Who Can Participate?
Eligibility varies by study.
Most trials have specific requirements based on:
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Age
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SCD type
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Treatment history
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Overall health
A research team will review your health history and walk you through what participation would involve before you decide.
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Participation is voluntary—you always have the right to ask questions, withdraw, or change your mind at any point.
Important Things to Remember
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Participation is a personal choice—you don’t have to take part if you aren’t comfortable.
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Ask questions and share your concerns with your care team before enrolling.
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Not all trials are suitable for everyone, and eligibility varies from study to study.
Clinical trials are one of the most powerful ways patients can contribute to new therapies and help shape the future of sickle cell care