Vitamin Supplement
Vitamin D for Sickle Cell Anemia
This trial tests if vitamin D3 can lower lung issues in kids with sickle cell disease. It compares different dosing schedules to see which works better. The goal is to find a simple, low-cost way to help these children stay healthier. Vitamin D has been studied for its potential to reduce respiratory complications in children with sickle cell disease due to its ability to fight infections and support the immune system.
Histone Deacetylase Inhibitor
Panobinostat for Sickle Cell Disease
This trial is testing a drug called panobinostat to see if it is safe and effective in treating adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor, which has been shown to increase hemoglobin F induction and inhibit cell-specific inflammation.
Anticoagulant
Defibrotide Prophylaxis + Stem Cell Transplant for Sickle Cell Disease
This trial tests if Defibrotide can safely and effectively prevent liver damage in high-risk sickle cell or beta thalassemia patients undergoing a special stem cell transplant. The medication works by improving blood flow in the liver to prevent blockages.
Popular Filters
Trials for Sickle Cell Disease Patients
Small Molecule
Etavopivat for Sickle Cell Disease
This trial is testing a new oral medicine called etavopivat in patients with sickle cell disease or thalassemia. The medicine helps red blood cells produce energy more efficiently. The goal is to reduce the need for blood transfusions and increase hemoglobin levels.
Trials for SCD Patients
Small Molecule
Etavopivat for Sickle Cell Disease
This trial is testing a new oral medicine called etavopivat in patients with sickle cell disease or thalassemia. The medicine helps red blood cells produce energy more efficiently. The goal is to reduce the need for blood transfusions and increase hemoglobin levels.
Trials for HbSS Positive Patients
Hematopoietic Stem Cell Transplantation
Stem Cell Transplant for Sickle Cell Disease
This trial is testing a new treatment for sickle cell disease which involves transplanting stem cells. The goal is to evaluate the safety and effectiveness of the treatment.
Trials for HbSC Positive Patients
Allosteric Activator
Mitapivat for Sickle Cell Disease
This trial will test how well mitapivat works compared to placebo in treating sickle cell disease by increasing hemoglobin levels and reducing or preventing sickle cell pain crises. The long-term effect of mitapivat will also be explored.
Corticosteroid
Immune Suppression Treatment for Sickle Cell Disease
This trial is studying a new way of doing an HCT: using drugs to lower the activity of the body's immune system before transplanting healthy stem cells to replace diseased or damaged cells.
Phase 3 Trials
Gene Therapy
CTX001 for Sickle Cell Disease
This trial is testing a possible new treatment for sickle cell disease using CRISPR-Cas9 technology to modify patients' own stem cells. The goal is to improve safety and efficacy compared to current treatments.
Small Molecule
Etavopivat for Sickle Cell Disease
This trial is testing a drug called etavopivat to see if it can help people with sickle cell disease. The drug aims to improve blood health and reduce pain by making red blood cells work better. The study includes patients aged 12 to 65.
Gene Therapy
Gene Therapy for Sickle Cell Disease
This trial is studying a gene therapy for sickle cell disease. The FDA has put a partial clinical hold on the trial for patients less than 18 years of age, which means that no new enrollment, cell collection, conditioning, or drug product infusion can be initiated in pediatric patients. The study is still open to patients 18 years of age and older.
Trials With No Placebo
Small Molecule
Etavopivat for Sickle Cell Disease
This trial is testing a new oral medicine called etavopivat in patients with sickle cell disease or thalassemia. The medicine helps red blood cells produce energy more efficiently. The goal is to reduce the need for blood transfusions and increase hemoglobin levels.
Carbon Dioxide for Sickle Cell Anemia
This trial is looking at how blood flow and metabolism affects brain development in early life. SCA participants and healthy controls will have their blood flow and metabolism examined using MRI, and will also undergo cognitive assessments and brief questionnaires.
Trials Offering Remuneration
Tyrosine Kinase Inhibitor
Imatinib for Sickle Cell Anemia
This trial tests a pill used for other diseases on patients with sickle cell disease. The goal is to see if it can lower harmful proteins and particles in their blood. Researchers hope this will reduce pain crises and other complications over several months.
Radiation
TMLI + Alemtuzumab for Sickle Cell Disease
This trial is testing a combination of a special type of radiation and a drug to prepare patients with sickle cell disease for a procedure that replaces their bone marrow. The treatment helps clear out the old bone marrow and prevents complications, making it easier for new cells to grow. The goal is to ensure the new cells are accepted by the body and reduce the risk of rejection and other issues. The drug has been used successfully in treating certain types of blood cancers.
Hematopoietic Cell Transplantation
Stem Cell Transplant for Sickle Cell Disease
This trial will evaluate a transplant regimen for reversing sickle cell disease, measuring success rate, survival, GVHD, viral reactivation, organ function, and gene therapy research.
Frequently Asked Questions
Introduction to sickle cell
What are the top hospitals conducting sickle cell research?
In the ongoing battle against sickle cell disease, several hospitals are leading the way in innovative clinical trials. Among them is the Children's Hospital of Philadelphia, nestled in the historic city of Philadelphia. With six active trials and an impressive 30 completed studies on sickle cell disease throughout its history, this hospital has been at the forefront of research since recording its first trial in 2003. Across the Midwest, the University of Illinois at Chicago is making significant strides with their own six ongoing sickle cell trials and a commendable track record of 26 previously conducted studies dating back to their inaugural trial also in 2003.
The Cincinnati Children's Hospital Medical Center joins these esteemed institutions with five active clinical trials focused on sickle cell disease. Since undertaking their initial trial in 2005, they have accumulated a total of 23 completed studies thus far. Further south lies St. Jude Children's Research Hospital located in Memphis - renowned for its dedication to pediatric care and cutting-edge research initiatives. As pioneers in this field, they recorded their first sickle cell trial back in 1992 and have since contributed significantly with five current trials underway alongside an impressive tally of 51 historical investigations.
On the West Coast, UCSF Benioff Children's Hospital Oakland plays a vital role by actively engaging in five ongoing clinical trials for sickle cell patients while contributing to scientific progress through fourteen previous investigations; theirs can be traced back to as recently as2015.The efforts made by these top-tier hospitals demonstrate not only their commitment towards advancing treatments for individuals living with sickle cell disease but also highlight how collaborative research endeavors drive medical breakthroughs across regions. Together, these healthcare powerhouses bring hope to those affected by this debilitating condition — proving that every study conducted brings us closer to improved therapies and brighter futures for countless patients around the globe
Which are the best cities for sickle cell clinical trials?
When it comes to sickle cell clinical trials, several cities offer a wealth of research opportunities. Atlanta, Georgia leads the way with 35 active trials focusing on treatments like Voxelotor, Biotin Labeled Red Blood Cells, and Arginine Hydrochloride. Chicago, Illinois follows closely behind with 30 ongoing studies examining Crizanlizumab, OTQ923, and Voxelotor among other interventions. New york City and Philadelphia tie at 22 active trials each that explore various treatments such as CTX001 and EDIT-301 for sickle cell disease. Lastly, Boston houses 20 active trials investigating therapies like Inclacumab and AG-946 in pursuit of improved outcomes for individuals living with sickle cell disease. These cities serve as vital hubs for cutting-edge research that aims to revolutionize care for those affected by this condition.
Which are the top treatments for sickle cell being explored in clinical trials?
Sickle cell disease is a complex condition, but advancements in clinical trials offer hope for improved treatments. Voxelotor takes the lead with four ongoing trials and a total of 10 sickle cell trials since its introduction in 2016. CTX001, a promising therapy, follows closely behind with three active trials and four all-time sickle cell studies since being listed in 2018. Hydroxyurea has also shown promise, participating in two current trials among its impressive track record of 42 previous sickle cell studies since 2000. A newcomer to the scene is inclacumab, currently undergoing investigation through two ongoing clinical trials after first appearing on the list just this year. With continued research and progress across these diverse treatment options, there is renewed optimism for patients living with sickle cell disease.
What are the most recent clinical trials for sickle cell?
Exciting advancements in the field of sickle cell research are bringing renewed hope to patients. The most recent clinical trials include a Phase 1 study, Cohort 3, which became available on 8/26/2023. Additionally, there is a promising trial focusing on Motixafortide followed by Motixafortide + Natalizumab for sickle cell disease that began on 7/7/2023. Another noteworthy trial, Treatment arm (1), initiated on 7/1/2023 and aims to further our understanding of potential treatment options. Furthermore, a Phase 4 trial investigating the use of Nexplanon in sickle cell patients commenced on 6/8//2023. Lastly, Hydroxyurea has been under investigation in both Phase 1 and Phase 2 trials since its availability from May onwards this year. These cutting-edge studies pave the way towards improved therapeutic approaches for individuals living with sickle cell disease.
What sickle cell clinical trials were recently completed?
Recent advancements in sickle cell clinical research have yielded valuable insights and potential treatment options. Notable completed trials include the Low Dose Rifaximin DER study by Bausch Health Americas, Inc., finalized in March 2022. Global Blood Therapeutics also achieved a significant milestone with their GBT021601 trial completion in May 2021. Similarly, CSL Behring's CSL889 trial concluded during the same period, showcasing promising results for sickle cell patients. These studies reflect the tireless efforts of researchers to combat this debilitating disease and offer hope for improved therapeutic interventions moving forward.