Clinical Trials

RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood. Unfortunately, the conventional approach to allogeneic stem cell transplantation is a risky procedure. For some non-cancerous conditions, the risks of this procedure outweigh the potential benefits. This protocol is designed to test a new approach to allogeneic stem cell transplantation.

The main objective of this study is to prove the superiority of a procedure which calculates the volume of RBCs to transfuse and the time between apheresis based on this algorithm, compared to the current procedure. The primary endpoint would be the number of patients with individually achieved objectives in terms of % HbS before each apheresis (which reflects the effectiveness of the previous apheresis) over a period of 12 months.

This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use.

PRIMARY OBJECTIVE

* To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs).

SECONDARY OBJECTIVES

This pilot study will assess the effects of a brief laboratory-based guided imagery procedure on responses to pain in patients with sickle cell disease (SCD) and healthy controls.

The investigators will conduct a hybrid type 1 effectiveness implementation trial to assess the effectiveness of acupuncture and guided relaxation on 360 people with Sickle Cell Disease (SCD), while observing and gathering information on implementation in three health systems: University of Illinois Hospital \& Health Sciences System, University of Florida Health, and Duke University Health Systems.

The Sickle Cell Anemia (SCA) is a recessive genetic condition, monogenic, resulting in defects in the red cell structure. In the investigators' country, this disease affects about 3,000 children each year and is considered one of the most prevalent disorders among the group of existing hereditary diseases.

The purpose of this research is to determine whether a new stress management and mindfulness program can improve quality of life in individuals with sickle cell disease. The program is based on a number of empirically supported treatments including techniques from cognitive-behavioral therapy like overcoming motivational barriers to self-care, learning to see things as accurately as possible, practicing assertive communication, relaxation exercises like deep slow breathing, mindfulness meditation, and other stress management strategies.

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK), and pharmacodynamics (i.e., how the body absorbs, distributes, breaks down, and excretes) of GBT021601, a hemoglobin S (HbS) polymerization inhibitor, in participants with SCD, following single and multiple ascending doses.

This is a study for parents of young children with Sickle Cell Trait (SCT) identified by newborn screening who are referred and present for in person SCT education at the Institution.

The study will determine the feasibility of implementing a SCT education program (SCTaware) that is appropriate for all parents, including those with low base knowledge and low health literacy and then test if results in high and sustained SCT knowledge.

Sickle cell anemia is a global public health disorder that affects millions of people across the globe. It is an inherited autosomal recessive hemoglobin condition caused by a beta-globin gene mutation that results in an aberrant sickle cell hemoglobin molecule with glutamic acid replaced by valine at position six of the beta-globin chain.