Clinical Trial Awareness: 5 Breakthrough Studies Shaping the Future of Sickle Cell Disease Care
June 19 marks World Sickle Cell Awareness Day, a global call to action to address the critical health inequities and care gaps faced by people living with Sickle Cell Disease (SCD). Approximately affecting 70,000 to 100,000 individuals in the U.S. alone, SCD is a serious, lifelong genetic condition that causes debilitating pain crises, organ damage, and reduced life expectancy. Although recent advancements—including gene editing and novel oral therapies—have brought new hope, awareness and access remain major barriers. Many patients still struggle to find relevant clinical trials or treatment options that match their unique needs.
To help close this gap, TrialX is proud to collaborate with the Sickle Cell Disease Association of America (SCDAA) to make clinical research more discoverable, inclusive, and actionable. Together, we’ve developed the SCDAA Clinical Trial Finder, a powerful, patient-friendly platform that simplifies the search for SCD trials by location, eligibility, and treatment type. The platform offers multiple ways to explore studies, including guided search and self-directed tools, as well as a Volunteer Registry that keeps patients informed about new opportunities tailored to their profiles. Through this collaboration, we aim to bridge information gaps, raise awareness, and enhance participation in research that has the potential to transform lives.
We’re spotlighting five ongoing breakthrough clinical trials that represent the future of SCD treatment.
1. First-in-Human Gene Editing Trial for Sickle Cell Disease
Sponsor- St. Jude Children’s Research Hospital
What It Tests and Why It Matters
This groundbreaking Phase 1 trial is evaluating the safety and potential efficacy of using CRISPR/Cas9 gene editing in patients with severe sickle cell disease (SCD). The goal is to increase fetal hemoglobin (HbF) levels—an alternative form of hemoglobin that can reduce or eliminate the painful symptoms and complications of SCD.
Why It Stands Out
This is one of the first clinical trials in the world to use autologous (self-donated) CRISPR-edited stem cells to treat sickle cell disease. Instead of just managing symptoms, this approach aims to correct the underlying genetic cause of SCD with a single infusion of gene-edited cells, potentially offering a long-term or even curative solution.
What It Could Mean for Patients
For young adults with severe SCD who experience frequent pain episodes or require regular transfusions, this could represent a transformative option—one that restores healthy blood production and significantly improves quality of life.
Location- This study is actively recruiting participants at St. Jude Children’s Research Hospital, Memphis, Tennessee, United States.
2. BEACON Trial: Advancing Precision Gene Editing for Sickle Cell Disease
Sponsor- Beam Therapeutics Inc.
What It Tests and Why It Matters
The BEACON trial is a Phase 1/2 study investigating BEAM-101, a base-edited therapy developed from a patient’s own blood stem cells. The goal is to boost fetal hemoglobin (HbF) production, which can help prevent or lessen the severity of painful vaso-occlusive crises (VOCs)—a hallmark complication of sickle cell disease.
Why It Stands Out
BEAM-101 uses base editing, a cutting-edge genetic engineering technique that allows for ultra-precise DNA changes without breaking the DNA strand. This could offer greater safety and accuracy than earlier gene editing approaches, representing a new frontier in personalized, curative therapies.
What It Could Mean for Patients
This therapy has the potential to deliver lasting relief from recurring VOCs, reduce hospitalizations, and significantly ease the burden of daily disease management.
Location-The study is on-site at multiple locations across the U.S. and is currently enrolling eligible individuals living with severe SCD.
3. Etavopivat: Aiming to Reduce Sickle Cell Crises and Improve Daily Function
Sponsor-Novo Nordisk A/S
What It Tests and Why It Matters
This Phase 3 clinical trial is evaluating Etavopivat, an investigational oral therapy designed to reduce the frequency of vaso-occlusive crises (VOCs)—the painful episodes caused by blocked blood vessels in people living with sickle cell disease (SCD). In addition to fewer VOCs, the study will also explore whether Etavopivat can help protect vital organs, improve stamina, and lessen fatigue.
Participants will be randomly assigned to receive either Etavopivat or a placebo, and the trial will span approximately two years. Etavopivat is also being studied in other SCD trials to further evaluate its safety and benefits.
Many people with SCD continue to experience frequent VOCs and fatigue despite current treatments. Etavopivat could offer a convenient, once-daily oral alternative with potential benefits across multiple symptoms.
Why It Stands Out
This is one of the few large-scale trials exploring both crisis reduction and quality-of-life improvements in SCD through a non-invasive, daily pill. If successful, it may become an important option for patients not eligible for curative therapies like gene editing or transplant.
What It Could Mean for Patients
Etavopivat could offer a more manageable and accessible treatment option for adolescents and adults who experience recurring pain episodes but are not candidates for more intensive interventions. It represents hope for reducing daily burdens and improving long-term outcomes.
Location- This trial is being conducted on-site in multiple locations.
See full study details and eligibility here.
4. Targeted Conditioning with 131I-apamistamab: A New Approach to Safer Stem Cell Transplant for SCD
Sponsor: Columbia University
What It Tests and Why It Matters
This early-phase study is evaluating the safety and effectiveness of 131I-apamistamab, a targeted radioactive antibody therapy, as part of the pre-transplant “conditioning” process for individuals with advanced sickle cell disease (SCD). Conditioning regimens are treatments given before stem cell transplants to prepare the body to accept new cells.
What makes this study unique is its goal to minimize or eliminate the need for total body irradiation (TBI)—a standard but harsh part of conventional conditioning linked to serious long-term side effects such as infertility, secondary cancers, and lung damage.
The study aims to identify the minimum effective dose (MED) of 131I-apamistamab that can be used safely to enable successful allogeneic hematopoietic stem cell transplantation (HSCT). The therapy targets CD45, a protein found on immune cells, potentially reducing toxicity by focusing radiation only where it’s needed.
While stem cell transplant remains the only curative option for many people with SCD, it often comes at the cost of aggressive pre-treatment. This study explores a less toxic, more targeted approach, which could make curative therapies safer and more accessible.
Why It Stands Out
This is the first time 131I-apamistamab is being used in patients with SCD. If successful, it could transform transplant protocols by offering a non-myeloablative conditioning option—meaning one that doesn’t completely wipe out bone marrow—thus lowering the risks associated with transplant.
What It Could Mean for Patients
By reducing conditioning toxicity, this approach could expand access to curative stem cell transplants for a broader group of SCD patients, especially those who may not be able to tolerate intensive therapy.
Location: On-site in New York
5. LIBRA Trial: Targeting Inflammation to Reduce Pain Crises in Sickle Cell Disease
Sponsor: Sanofi
What It Tests and Why It Matters
The LIBRA study is a Phase 3 clinical trial evaluating rilzabrutinib, an oral Bruton’s tyrosine kinase (BTK) inhibitor, in people living with sickle cell disease (SCD). Unlike therapies that target red blood cell health directly, rilzabrutinib works by reducing inflammation, which is believed to play a critical role in triggering vaso-occlusive crises (VOCs). The trial will assess whether rilzabrutinib can safely and effectively reduce the frequency of VOCs in both adolescents and adults. In Part A, participants are randomly assigned to receive either rilzabrutinib or a placebo for 52 weeks under double-blind conditions. Those who complete this stage may continue in an open-label extension (Part B) to monitor longer-term benefits.
Why It Stands Out
This is one of the few late-stage trials specifically focusing on the inflammatory cascade in sickle cell disease. By blocking BTK signaling, rilzabrutinib may disrupt key pathways involved in VOC formation, offering a novel approach beyond red cell–based therapies like Etavopivat or hydroxyurea.
What It Could Mean for Patients
If successful, rilzabrutinib could offer a new oral treatment option for patients who continue to experience frequent crises despite existing therapies. Its focus on inflammation may also provide relief for individuals who aren’t candidates for gene editing or stem cell transplant, expanding therapeutic possibilities.
Study Locations– This trial is actively enrolling at locations in Florida (USA) and London (UK).
View participation and eligibility here.
Join the Movement
On this World Sickle Cell Awareness Day, we encourage patients, caregivers, and advocates to explore clinical trials not just as research, but as a path to possibility. To learn more, get involved, or find a trial that matches your needs, visit the SCDAA Clinical Trial Finder.
Alongside clinical research opportunities, patients and caregivers may find valuable education, support, and community through organizations like the American Society of Hematology, Advancing Sickle Cell Advocacy Project, and Sickle Cell Warriors to navigate SCD.
