Sickle cell disease clinical trials, in English

This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.

NCT00081523 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body.

NCT00542230 · Official ClinicalTrials.gov record · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.

NCT03685721 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

Background: Sickle cell disease can often be treated with blood stem cell transplants. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body.

NCT04008368 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.

NCT04099966 · Official ClinicalTrials.gov record · Mitchell Cairo

This is an open label, non-randomly assigned, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will measure the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

NCT04774536 · Official ClinicalTrials.gov record · Mark Walters, MD

Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant.

NCT05213572 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ("sickled") red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.

NCT05904093 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT.

NCT06071377 · Official ClinicalTrials.gov record · National Alliance for Sickle Cell Centers

Cell-free fetal DNA (cffDNA) is present in the maternal blood from the early first trimester of gestation and makes up 5%-20% of the total circulating cell-free DNA (cfDNA) in maternal plasma. Its presence in maternal plasma has allowed development of noninvasive prenatal diagnosis for single-gene disorders (SGD-NIPD).

NCT06147414 · Official ClinicalTrials.gov record · Assistance Publique - Hôpitaux de Paris

The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also seeks to understand the role of personalized peer support in enhancing engagement and outcomes of digital CBT interventions.

NCT06374238 · Official ClinicalTrials.gov record · University of Pittsburgh

A phase III, multi-center, randomly assigned, compared with a dummy treatment, neither you nor the study doctors know who gets the real treatment study to assess how well it works and safety of crizanlizumab (5 mg/kg) versus dummy treatment (placebo), with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

NCT06439082 · Official ClinicalTrials.gov record · Novartis Pharmaceuticals

This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.

NCT06506461 · Official ClinicalTrials.gov record · St. Jude Children's Research Hospital

This study wants to measure the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

NCT06546670 · Official ClinicalTrials.gov record · Novartis Pharmaceuticals

This research is being conducted to see if using an injectable contraception, Depot Medroxyprogesterone Acetate (Depo-Provera), can reduce the pain experienced by women with sickle cell disease. Participants in this study will be adult women with sickle cell disease who regularly experience sickle cell pain.

NCT06665997 · Official ClinicalTrials.gov record · University of Pennsylvania

This is a phase 2, randomly assigned, multiple-dose, compared with a dummy treatment study designed to measure the safety, how well it works, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to measure the whether it is safe and how hard side effects are to live with of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.

NCT06699849 · Official ClinicalTrials.gov record · CSL Behring

This is a multicenter, randomly assigned, neither you nor the study doctors know who gets the real treatment, compared with a dummy treatment, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the how well it works, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week neither you nor the study doctors know who gets the real treatment period (Part A), followed by an open-label LTE period (Part B).

NCT06975865 · Official ClinicalTrials.gov record · Sanofi

The goal of this study is to measure the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease. * The primary objective is to determine the feasibility of using a tAN device to treat pain in pediatric participants with SCD by assessing the completion rate, stimulation tolerability, and acceptability of the intervention measures.

NCT07090668 · Official ClinicalTrials.gov record · Emory University

Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons. The HOPE program is expanded access program to serve these patients by providing our bone marrow product

NCT07145411 · Official ClinicalTrials.gov record · Ossium Health, Inc.

This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

NCT07187973 · Official ClinicalTrials.gov record · Disc Medicine, Inc

Sickle cell disease (SCD) is a prevalent inherited blood disorder characterized by vaso-occlusive crises (VOCs), which lead to severe pain and complications. Despite hydration being a cornerstone of VOC management, the optimal fluid strategy remains uncertain.

NCT07188766 · Official ClinicalTrials.gov record · Imam Abdulrahman Bin Faisal University

INTEGRA aims at enabling personalized medicine for RADs patients by the establishment of an integrative diagnostic approach based on deep phenotypic and genetic characterization through combining new generation methodologies.

NCT07206095 · Official ClinicalTrials.gov record · Hospital Universitari Vall d'Hebron Research Institute

This is a phase 2a, global, multicenter, randomly assigned, neither you nor the study doctors know who gets the real treatment, compared with a dummy treatment study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD.

NCT07224360 · Official ClinicalTrials.gov record · CSL Behring

SIL-8301 (senicapoc) is being developed for the chronic treatment of patients with sickle cell disease in both adults and children. this study wants to compare the effects of senicapoc to dummy treatment (placebo) in patients with sickle cell disease that have had fewer than 2 acute sickle-related painful crises per year over the preceding 2 years, and have a predominantly hemolytic phenotype, defined as evidence of ongoing hemolysis and a baseline Hb of 9.5 g/dL or less, despite receiving hydroxyurea (an oral drug used for treatment of sickle cell disease) as standard of care.

NCT07282210 · Official ClinicalTrials.gov record · Biossil Inc.

The goal of this clinical trial is to learn if a multifaceted intervention composed of Gazelle-Multispectral sickle cell disease (SCD) point of care testing (POCT) and early initiation of comprehensive SCD care in children with SCD disease aged 0 - 6 months can improve their clinical outcomes. The main questions it aims to answer are: * Does the intervention lower the number of SCD-related illnesses?

NCT07488520 · Official ClinicalTrials.gov record · Swiss Tropical & Public Health Institute

The wide interindividual variability in clinical response to hydroxyurea therapy in the management of sickle cell disease has limited its use. These variabilities have been linked to differences in pharmacodynamics, pharmacokinetics, and pharmacogenetics.

NCT07543289 · Official ClinicalTrials.gov record · Ochuko Orherhe

Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.

NCT07566494 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens.

NCT07599176 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

The primary objective of this study is to determine the effect of mitapivat versus dummy treatment (placebo) on the need for transfusions in subjects with SCD.

NCT07656415 · Official ClinicalTrials.gov record · Agios Pharmaceuticals, Inc.

This randomly assigned pilot study will measure the feasibility, tolerability, and preliminary analgesic effect of headset-based virtual reality (VR) interventions for adults with sickle cell disease (SCD) experiencing vaso-occlusive crisis (VOC) treated in an infusion center. Up to 50 you would be enrolled, provide informed consent, and undergo randomization during routine outpatient sickle cell clinic visits when they are clinically stable and not experiencing an acute VOC.

NCT07674277 · Official ClinicalTrials.gov record · University of Maryland, Baltimore

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his or her own disease.

NCT07708350 · Official ClinicalTrials.gov record · Daniel Bauer

The overarching goal of this study is to measure the feasibility of a new methodology that combines three multi-level implementation strategies to optimize the population-level uptake of essential evidence-based, standard of care treatments for infants with sickle cell disease (SCD) in low-resource settings.

NCT07719972 · Official ClinicalTrials.gov record · St. Jude Children's Research Hospital