Clinical Trials

The purpose of this pilot study is to investigate the effects of a 6-session music therapy protocol on the pain, mood, quality of life, coping skills, and self-efficacy of adult patients with sickle cell disease (SCD) as compared to adult patients with SCD who receive standard care alone. The investigators will also determine the feasibility (delivery, acceptability, and usefulness) of the music therapy intervention for pain management and quality of life.

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. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease.

This was a retrospective cohort study using secondary data from member sites of the National Alliance of Sickle Cell Centers (NASCC) with patients who had initiated crizanlizumab between 2019 and 2022.

The project will test a tailored web and smartphone-based application (iCanCope with SCD) to improve pain self-management and functioning in youth (aged 12-18) with sickle cell disease. The program will include goal setting, peer-based social support, and pain self-management training. The investigators will determine initial program effectiveness through a pilot three-site randomized controlled trial in 160 youth randomized to treatment compared to attention control.

The purpose of this study is to assess the tolerance and efficacy of mobilizing hematopoietic stem cells after a single injection of plerixafor (0.24mg/kg) in 3 adult patients (or 5, if results of the first 3 patients are not reproducible) affected by sickle cell disease.

The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD) patients.

This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.

The purpose of this study is to evaluate the effect of Virtual Reality induced Hypnosis on patients' pain scores, anxiety and the use of analgesics during a vaso-occlusive crisis

Pediatric patients with sickle cell disease are at greater risk for exhibiting nocturnal enuresis (bedwetting) compared to the general population. This increased risk has been attributed to a decreased ability to concentrate urine caused by sickling-induced nephropathy. The sociodemographic, psychosocial, and medical factors associated with nocturnal enuresis are not well defined. In addition, the impact of these behaviors on emotional and behavioral functioning, along with health-related quality of life are not clear.

The BABY HUG Treatment Study was designed to see if treatment with the drug hydroxyurea (also called HU) in children with sickle cell disease could prevent organ damage, especially in the spleen and kidneys. There was also a chance that treatment could prevent painful crises, lung disease, stroke, and blood infection.