Sickle cell disease just entered a new era — two gene therapies, casgevy and lyfgenia, were FDA-approved in late 2023 and offer the potential for a cure. They join hydroxyurea, voxelotor, crizanlizumab, and L-glutamine as treatment options, expanding what was a very limited toolkit.
What's actually going on in research
Trials are testing improved gene therapies and gene-editing approaches, less-toxic stem cell transplants, new drugs to prevent pain crises, and treatments for chronic pain and organ damage. Researchers are also studying outcomes in adults, who historically had fewer options, and access barriers to the new gene therapies.
Gene therapy
Two FDA-approved gene therapies can essentially eliminate pain crises in many patients. Trials are now testing newer, simpler approaches that may be safer and reach more patients.
Less-toxic transplants
Newer stem cell transplant approaches use lower-intensity preparation, opening the door for adults and patients without a perfectly matched donor.
New medications
Drugs that prevent pain crises and reduce organ damage continue to advance. Trials are testing combinations and approaches to chronic pain, which is often undertreated.
What to know before you search
Eligibility often depends on genotype (HbSS, HbSC, HbS-beta thalassemia), pain crisis frequency, organ damage, age, and prior treatments.
What types of trials are currently open
- Treatment trials — Testing new drugs to reduce pain crises, anemia, and organ damage in sickle cell disease.
- Gene therapy trials — Studies of newer gene-editing and gene-addition therapies aimed at curing sickle cell disease.
- Stem cell transplant trials — Testing less-toxic transplant approaches that may reach more adults and patients without matched donors.
- Pain management trials — Testing treatments for chronic pain and acute pain crises.
- Observational studies — Following people with sickle cell to understand long-term outcomes and access to new treatments.
Recently added Sickle Cell Disease trials
A Smart Phone Application to Improve Adoption of the 2024 Kidney Disease Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) Guidelines
The goal of this study is to establish whether use of a digital intervention can improve adherence and alignment with the Kidney Disease: Improving Global Outcomes (KDIGO) Chronic Kidney Disease (CKD) 2024 Guidelines. A subset of the study will focus on whether the intervention improves outcomes for young adults living with CKD, in the context of the imminent co-location of Children's Health Ireland on the St. James's Hospital campus. Young adults with CKD transitioning to adult services are recognised as a high-risk and vulnerable cohort, with many individuals unaware of increased cardiovascular risk and mortality¹². In response, and in the context of the co-location of Children's Health Ireland on the St. James's Hospital site, a young adult nephrology clinic has been established. The KDIGO CKD 2024 Guidelines identify transition as a period of increased risk and include recommendations regarding cardiovascular risk factor targets and the use of therapies known to delay CKD progression³. Electronic communication is a preferred method for accessing health information among many young adults⁴⁵ and aligns with Sláintecare digital health strategies⁶. A recently established, award-winning St. James's Hospital renal smartphone application is currently used by over 3,000 individuals living with CKD. The study aims to determine whether use of the application improves adherence to KDIGO guideline recommendations, with the objective of delaying CKD progression and associated complications. The application will support optimisation of care by signposting opportunities for evidence-based interventions (e.g., SGLT2 inhibitors, renin-angiotensin system inhibition) to healthcare providers. The application will also provide participants with tailored recommendations, reminders, educational materials, and collection of patient-reported outcome measures. Due to the diverse population and range of specialties at St. James's Hospital, the young adult clinic serves distinct subgroups, including individuals with sickle cell anaemia and survivors of cancer and haematological malignancies. These populations will be examined in the context of KDIGO guideline implementation, contributing to a limited international evidence base. This research evaluates an intervention designed to improve care for adults living with chronic kidney disease.
Multi Disciplinary Sickle Cell Disease Obstetrics Care Program in Ghana
The goal of this observational study is to determine the feasibility and effectiveness of initiating a multidisciplinary sickle cell disease (SCD) obstetrics program for women with SCD in a non-academic hospital. The main question it aims to answer is: In a before-and-after study design, we will test the hypothesis that multidisciplinary care for pregnant women with SCD in a non-academic hospital will result in a 50% relative risk reduction in mortality compared to the mortality rate in pregnant women with SCD in the same hospital before the multidisciplinary care. Participants will be managed using the academic hospital's multidisciplinary sickle cell disease obstetrics protocol adapted for the non-academic hospital
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