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.
Amino Acid Supplement
L-Citrulline for Sickle Cell Disease
This trial aims to investigate if intravenous citrulline can help reduce acute pain in hospitalized patients with sickle cell disease. Researchers will compare the effects of intravenous citrulline to a
Procedure
Motixafortide + Natalizumab for Sickle Cell Disease
This trial is testing two drugs, motixafortide and natalizumab, to help patients with sickle cell disease. These drugs aim to move more stem cells from the bone marrow into the blood, making it easier to collect them for gene therapy. Natalizumab is also used to treat multiple sclerosis.
Popular Filters
Trials for SCD Patients
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 for Sickle Cell Anemia Patients
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 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.
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
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.
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.
Trials Offering Remuneration
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.
Tyrosine Kinase Inhibitor
Fostamatinib for Sickle Cell Disease
This trial tests the safety and tolerability of fostamatinib, a drug taken by mouth, in people aged 18 to 65 with stable sickle cell disease. Fostamatinib aims to reduce inflammation and abnormal blood cell behavior by inhibiting a specific protein. Participants will take the drug for several weeks and have frequent check-ups to monitor side effects and effectiveness.
Frequently Asked Questions
Introduction to sickle cell disease
What are the top hospitals conducting sickle cell disease research?
Sickle cell disease, a genetic blood disorder that affects millions of people worldwide, is an area where several top hospitals excel in conducting clinical trials. One such institution making remarkable contributions is the Children's Hospital of Philadelphia in Philadelphia. With six ongoing trials dedicated to sickle cell disease and a total of 30 conducted studies to date, this hospital has been at the forefront of research since its first recorded trial in 2003. Duke University Medical Center in Durham follows suit with equal determination, currently engaged in six active sickle cell disease trials and having completed 16 previous investigations since their initial trial in 2005.
In Memphis, St. Jude Children's Research Hospital stands out as another leading facility dedicated to combatting sickle cell disease. Their commitment is evident through their participation in six ongoing clinical trials and an impressive tally of 51 completed studies thus far; they have been involved since recording their first sickle cell disease trial back in1992. Similarly joining this league of exceptional healthcare providers is the University of Illinois at Chicago located within Chicago itself where they actively run around half-a-dozen tests for treating patients affected by Sickle Cell Disorder while being accountable for twenty-six similar prior experiments all starting from merely eighteen years ago back until2003.
Furthermore adding value to this endeavor Cincinnati Children’s Hospital Medical Center ensures inclusive representationwith about five contemporary efforts working towards better understanding,Sickle Cell Disease ;their involvement was marked just sixteen years ago when beginning their first experiment on said subject matterback tillthe year2005
These top hospitals not only strive to improve outcomes for individuals impacted by sickle cell disease but also play crucial roles as pioneers pushing boundaries and broadening horizons within medical research communities nationwide.The dedication shown by these institutions highlights how each step taken towards unraveling the complexities surrounding this condition brings us closer to novel therapies and ultimately improved quality of life for those living with sickle cell disease globally
Which are the best cities for sickle cell disease clinical trials?
When it comes to sickle cell disease clinical trials, several cities emerge as frontrunners in research and development. Atlanta, Georgia leads the pack with 31 ongoing trials focusing on treatments like Biotin Labeled Red Blood Cells, Voxelotor, and crizanlizumab. Chicago, Illinois closely follows with 29 active studies investigating Crizanlizumab, Inclacumab, and Etavopivat Tablets. Philadelphia, Pennsylvania also plays a significant role in sickle cell disease research with 24 trials exploring treatments such as Crizanlizumab and Voxelotor. New york City boasts 22 active trials studying promising candidates like CTX001 and OTQ923 while Boston offers 20 ongoing studies focused on Inclacumab, Voxelotor, and AG-946. These cities serve as hubs for cutting-edge clinical trials in sickle cell disease that provide hope for improved therapies and better quality of life for patients.
Which are the top treatments for sickle cell disease being explored in clinical trials?
Exciting advancements are being made in the exploration of clinical trials for sickle cell disease treatment. Currently, CTX001 is leading the charge with three active trials and four all-time sickle cell disease trials since its listing in 2018. Hydroxyurea follows closely behind with two ongoing trials and an impressive history of 42 all-time treatments dedicated to tackling this condition since 2000. Also showing promise are plerixafor and alemtuzumab, each participating in two active trials and contributing significantly to the fight against sickle cell disease with nine and sixteen all-time clinical trials respectively. These innovative treatments offer new hope for patients battling this challenging condition.
What are the most recent clinical trials for sickle cell disease?
Promising advancements are being made in the field of sickle cell disease, with recent clinical trials offering hope for improved treatment strategies. Among these trials is Cohort 3, which focuses on evaluating a potential therapy for sickle cell disease patients in Phase 1. Another study investigates the use of Motixafortide followed by Motixafortide + Natalizumab as a potential treatment option for this condition. Additionally, a Phase 1 trial explores Treatment Arm (1) to further enhance our understanding of effective therapies for sickle cell disease. Furthermore, ongoing research delves into the efficacy and safety profile of Nexplanon specifically tailored towards individuals with sickle cell disease in its Phase 4 trial stage. Lastly, the benefits and impact of Hydroxyurea are under scrutiny through combined Phase 1 and Phase 2 investigations aiming at finding better therapeutic options against this debilitating condition. The outcomes from these diverse clinical studies bring optimism to those affected by sickle cell disease while fostering progress within the medical community.
What sickle cell disease clinical trials were recently completed?
Multiple groundbreaking clinical trials investigating potential treatments for sickle cell disease have recently concluded, highlighting ongoing efforts to combat this challenging condition. In March 2022, Bausch Health Americas completed a trial studying the efficacy of Low Dose Rifaximin DER. Another notable trial was conducted by Global Blood Therapeutics, concluding in May 2021 and exploring the potential benefits of GBT021601 therapy. CSL Behring also finalized their study on CSL889 in May 2021. These recent advancements underscore the dedication of researchers and provide hope for individuals affected by sickle cell disease.