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The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease. SCD-101 inhibits sickling of red blood cells containing Hb S (sickle hemoglobin) under low oxygen conditions, the underlying cause of sickle cell disease. SCD-101 may inhibit red blood cell sickling in patients with sickle cell disease and reduce symptoms and slow disease progression.
This study will evaluate the safety and antisickling activity of SCD-101 in up to 24 adults with sickle cell disease.
This study is an open-label, non-randomized, non-placebo-controlled, single site, dose- escalation study of SCD-101 in participants with homozygous sickle cell disease (S/S) or S/beta 0 thalassemia. All participants will be monitored for safety, tolerability, and dose-limiting toxicities.
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Sickle Cell Disease
King's County Hospital
Published on BioPortfolio: 2015-03-26T05:53:22-0400
To determine the retinal and choroidal thickness in patients with sickle cell disease compared to age, race matched population without sickle cell disease to allow a better understanding o...
To continue studies on the two major neurological complications of sickle cell disease (SCD): namely, stroke and chronic encephalopathy.
Sickle cell disease is a genetic red blood cell disorder that can result in blocking of the small blood vessels from sickle shaped red blood cells. This causes pain, the main feature of si...
Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately. The sickle cell dis...
The purpose of this study is to determine whether treatment of sickle cell patients with docosahexaenoic omega-3 acid (DHA) is effective in prevention of acute sickle cell crisis.
A pseudoxanthoma elasticum (PXE)-like phenotype develops in a subset of patients with inherited hemoglobinopathies. Although PXE tissue changes are thought to develop in the absence of ABCC6 mutations...
Oxidative stress contributes to the complex pathophysiology of sickle cell disease. Oral therapy with pharmaceutical-grade l-glutamine (USAN, glutamine) has been shown to increase the proportion of th...
Sickle cell disease is a genetic disease commonly affecting people of African, Indian, and Mediterranean descent. Patients with this chronic disease often require lifelong red blood cell transfusions....
Salmonella infections are a common bacterial cause of invasive disease in people with sickle cell disease especially children, and are associated with high morbidity and mortality rates. Although avai...
Pneumococcal carriage is the precursor for development of pneumococcal disease, and is also responsible for transmission of the organism from person-to-person. Individuals with Sickle Cell Disease (SC...
One of the sickle cell disorders characterized by the presence of both hemoglobin S and hemoglobin C. It is similar to, but less severe than sickle cell anemia.
An abnormal hemoglobin resulting from the substitution of valine for glutamic acid at position 6 of the beta chain of the globin moiety. The heterozygous state results in sickle cell trait, the homozygous in sickle cell anemia.
An acute purulent infection of the meninges and subarachnoid space caused by Streptococcus pneumoniae, most prevalent in children and adults over the age of 60. This illness may be associated with OTITIS MEDIA; MASTOIDITIS; SINUSITIS; RESPIRATORY TRACT INFECTIONS; sickle cell disease (ANEMIA, SICKLE CELL); skull fractures; and other disorders. Clinical manifestations include FEVER; HEADACHE; neck stiffness; and somnolence followed by SEIZURES; focal neurologic deficits (notably DEAFNESS); and COMA. (From Miller et al., Merritt's Textbook of Neurology, 9th ed, p111)
A disease characterized by chronic hemolytic anemia, episodic painful crises, and pathologic involvement of many organs. It is the clinical expression of homozygosity for hemoglobin S.
The condition of being heterozygous for hemoglobin S.
Blood is a specialized bodily fluid that delivers necessary substances to the body's cells (in animals) – such as nutrients and oxygen – and transports waste products away from those same cells. In vertebrates, it is composed of blo...