Every June 19th, World Sickle Cell Day shines a spotlight on one of the most common inherited blood disorders—yet one that remains underdiagnosed, underfunded, and underrepresented in research.
Sickle cell disease (SCD) causes red blood cells to become rigid and sickle-shaped, blocking blood flow and triggering severe pain, organ damage, stroke, and other life-threatening complications. It affects approximately 100,000 people in the U.S. and millions more worldwide. Despite this prevalence, many patients face delayed diagnoses, limited access to specialized care, and few treatment options beyond managing symptoms.
Organizations like the Sickle Cell Disease Association of America (SCDAA), led by Regina Hartfield, are working to close these gaps through advocacy, research funding, and connecting the community to studies that could transform outcomes for patients. As part of this effort, we at TrialX are proud to support the Sickle Cell Disease Association of America through our global clinical trial finder—helping connect patients and caregivers to relevant sickle cell research studies.
Making Trial Discovery Simpler for the Sickle Cell Community with Clinical Trial Finder
To help address the disconnect between available research and patient awareness, the SCDAA Clinical Trial Finder, powered by TrialX, is designed to make clinical trial information easier to navigate. The finder offers two primary ways to explore studies:
Guided Search: A Step-by-Step Way to Find Relevant Clinical Trials
The guided search begins with short, easy-to-understand information about clinical trials and what participation may involve. There is also an option to learn more through a resource page that explains potential risks and benefits.
Users are then asked a few simple questions, such as location, age, and willingness to travel, along with a few additional details related to their condition. Based on these inputs, the finder shows clinical trials that may be relevant.
This approach is designed to support people who may be new to clinical research and want a clearer, more guided way to get started.
Search On Your Own: Browse Available Clinical Trials
For those who prefer to explore independently, the SCDAA clinical trial finder also offers a Search on Your Own option, giving users more control to browse and compare studies at their own pace.
Users can view available sickle cell disease studies and apply filters, including:
- Location
- Study type
- Trial phase
- Online or remote participation options
- Keywords
This option is particularly useful for patients who already have some familiarity with clinical research, or who are looking for studies related to specific SCD complications such as pain crises, acute chest syndrome, stroke, or organ damage.
Clear contact options are included throughout, allowing individuals to reach out to study teams directly if they want to learn more about a specific trial.
A Few Ongoing Sickle Cell Disease Clinical Trials Currently Recruiting
- Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Sponsor: Abdullah Kutlar, Augusta University
This Phase I interventional study is investigating the safety and tolerability of panobinostat—an HDAC inhibitor—in adults with sickle cell disease who have not responded to, or cannot tolerate, hydroxyurea therapy. HDAC inhibitors have shown promise in increasing fetal hemoglobin (HbF) levels, which are known to improve outcomes in SCD, while also reducing the inflammation that drives many of the disease’s most debilitating effects.
Researchers will assess how the body responds to escalating doses of panobinostat over a 12-week treatment period, tracking changes in HbF levels, total hemoglobin, inflammatory markers, and quality of life. The findings could help identify a new treatment pathway for SCD patients with limited options.
Location: Recruiting at Augusta University in Augusta, Georgia, United States.
Learn more about the study and see if you may be eligible to participate.
- Investigation of the Genetics of Hematologic Diseases
Sponsor: St. Jude Children’s Research Hospital
This long-term observational study at St. Jude Children’s Research Hospital aims to build a deeper understanding of the genetic roots of blood diseases, including sickle cell disease. By collecting samples—such as blood, bone marrow, saliva, and other biological materials—alongside detailed health histories, researchers hope to uncover why certain genetic blood disorders develop and why some patients experience more severe outcomes than others.
Using advanced genomic tools including whole genome sequencing, researchers will work to identify novel genetic mutations, modifier genes, and variants linked to treatment response and toxicity. For SCD specifically, the study will also investigate biomarkers for pain episodes and the long-term effects of hydroxyurea therapy. Findings from this registry could open new doors for diagnosis and treatment across a range of inherited blood conditions.
Location: Recruiting at St. Jude Children’s Research Hospital in Memphis, Tennessee, United States.
Learn more about the study and see if you may be eligible to participate.
- Virtual Reality Devices as an Adjunct to Usual Care for Patients With Sickle Cell Disease Experiencing Vaso-Occlusive Crises
Sponsor: University of Maryland, Baltimore
This pilot interventional study is exploring whether virtual reality (VR) can help reduce pain during vaso-occlusive crises in adults with sickle cell disease. Conducted in the emergency department at the University of Maryland Medical Center, the study compares two VR approaches—active immersive (where patients interact with a virtual environment) and passive immersive (where patients observe without interaction)—against a placebo control group using a blindfold and earplugs.
Researchers will track pain scores, opioid use, length of ED treatment, and patient comfort at multiple time points throughout the session. The goal is to determine whether VR can serve as a meaningful non-pharmacological tool to complement standard pain management—potentially reducing reliance on opioids during acute crises.
Location: Recruiting at the University of Maryland Medical Systems in Baltimore, Maryland, United States.
Learn more about the study and see if you may be eligible to participate.
- A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Sponsor: EpiDestiny, Inc.
This Phase I randomized controlled trial is investigating a novel oral drug combination for adults with high-risk sickle cell disease who have not responded adequately to hydroxyurea therapy. The study compares nicotinamide (Vitamin B3) alone to oral tetrahydrouridine (THU) and decitabine, and then evaluates the effects of all three drugs used together.
Each treatment phase runs for 12 weeks, with an option for participants to continue into a 24-week extension of the combination therapy. Researchers will measure changes in hemoglobin levels to assess how well the treatments work, with the goal of identifying an effective oral regimen that could offer a new path forward for patients with limited treatment options.
Location: Recruiting at the University of Illinois at Chicago College of Medicine in Chicago, Illinois, United States.
Learn more about the study and see if you may be eligible to participate.
- Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease (Restore)
Sponsor: Kamau Therapeutics
This first-in-human Phase I/II study is evaluating nula-cel, a CRISPR-Cas9 gene correction therapy, in patients aged 12 to 40 with severe sickle cell disease. Rather than adding a new gene, nula-cel works by directly correcting the sickle cell mutation in a patient’s own stem cells—converting the disease-causing HbS hemoglobin back to healthy HbA—before returning those edited cells to the patient via IV infusion.
Researchers will assess the safety of the treatment and track key outcomes including engraftment, overall survival, reduction in vaso-occlusive crises, and changes in hemoglobin levels over 24 months. If successful, this approach could represent a meaningful step toward a lasting correction for one of the most severe forms of sickle cell disease.
Locations: Recruiting at Children’s Hospital Los Angeles and Lucile Packard Children’s Hospital in California; Washington University in St. Louis, Missouri; and Nationwide Children’s Hospital in Columbus, Ohio, United States.
Learn more about the study and see if you may be eligible to participate.
Explore Ongoing Sickle Cell Disease Clinical Trials
World Sickle Cell Day is a reminder of how much work remains—and how much progress is possible when communities, researchers, and advocates come together. Increasing visibility and access to clinical trials helps move research forward and supports progress for everyone living with sickle cell disease.
If you or someone in your family has sickle cell disease and would like to learn more about ongoing studies, the SCDAA Clinical Trial Finder provides information on current research opportunities.
