Actively Recruiting

Sickle Cell Disease Clinical Trials

Find recruiting clinical trials for sickle cell disease in the UK — including HbSS, HbSC, and beta thalassemia. Gene therapy is transforming treatment. See pathways and where trials fit in.

Free to use — Live data from ClinicalTrials.gov — Updated hourly

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Sickle Cell Disease Treatment Pathway

See where clinical trials fit into your treatment journey

Standard Care

Disease-modifying therapy

Standard: Hydroxycarbamide (Hydroxyurea), transfusions, pain management

Voxelotor / Crizanlizumab

Newer targeted therapies

Standard: Voxelotor (Oxbryta) for anaemia, Crizanlizumab for pain crises

Gene Therapy

Curative intent

Standard: Gene addition (Lovo-cel) or gene editing (Casgevy/Exa-cel)

About Sickle Cell Disease

HbSS

The most severe form of sickle cell disease. Two sickle cell genes inherited. Causes frequent pain crises, anaemia, and organ damage.

HbSC

One sickle cell gene and one haemoglobin C gene. Generally milder than HbSS but can still cause serious complications.

Gene Therapy

The newest curative approach. Casgevy (Exa-cel) became the first CRISPR-based therapy approved by MHRA in 2023. Multiple gene therapy trials are recruiting.

Search Sickle Cell Disease Trials

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Frequently Asked Questions

Gene therapy aims to be a one-time curative treatment. Casgevy (Exa-cel), the first CRISPR-based therapy approved by the MHRA, has shown excellent results in clinical trials. However, long-term data is still being collected.

Eligibility varies by trial but typically includes patients with severe sickle cell disease (HbSS or HbS/beta-zero thalassemia) who experience frequent pain crises. Age requirements and other criteria differ between studies.

Trial teams provide comprehensive support including travel expenses, liaison with your regular healthcare team, and access to specialist sickle cell nurses. Cultural and community support is also available.

How to Join a Sickle Cell Disease Clinical Trial

1

Search

Use our search above to find trials that match your condition and location. Review the eligibility criteria carefully.

2

Discuss

Talk to your GP or specialist about any trials you are interested in. They can help determine if a trial is appropriate for you.

3

Contact

Reach out to the trial team directly using the contact information on the ClinicalTrials.gov listing. They will guide you through screening.

4

Enrol

If you meet the criteria and decide to participate, you will go through informed consent and begin the trial process.

Related Comparisons

🗺️ Related Pathway: Rare & Genetic Pathway