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Fairfax, Virginia Clinical Trials

A listing of Fairfax, Virginia clinical trials actively recruiting patient volunteers.

RESULTS

Found (382) clinical trials

A Global Study to Assess the Drug Dynamics Efficacy and Safety of GZ/SAR402671 in Parkinson's Disease Patients Carrying a Glucocerebrosidase (GBA) Gene Mutation

The total study duration per subject in Part 2 will be approximately 168 weeks that will consist of 6.5 weeks of screening period, 52 weeks of treatment period, 104 weeks of follow-up period, and 6 weeks of post-treatment observation period. Part 1 maximal duration will be up to 48 weeks ...

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Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies neoGAA and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease

The duration of the study per patient will be up to 3 years that will consist of a 14- day screening period (may be extended up to 8 weeks in pre-specified situations), a 49-week blinded treatment period, a 96-week open-label treatment period, and a 4-week post-treatment observation period.

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Study of Immune Globulin Intravenous (Human) GC5107 in Subjects With Primary Humoral Immunodeficiency

This will be a prospective, open-label, single-arm, historically controlled, multicenter phase 3 study measuring the safety, efficacy and pharmacokinetics and tolerability of GC5107 in subjects with Primary Humoral Immunodeficiency disease (PHID). Subjects will receive intravenous infusions of the investigational product at the same dose and interval as used for their ...

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Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

This is a randomized, double blind, active control study of PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function. Patients treated for approximately 1 year with agalsidase beta and on a stable dose for at least 6 months will be screened and then randomized to continue treatment with ...

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Study of Efficacy and Safety of QAW039 in Patients With Severe Asthma Inadequately Controlled With Standard of Care Asthma Treatment.

A double-blind, placebo-controlled study to evaluate the efficacy and safety of two doses (Dose 1 and Dose 2) of QAW039 compared with placebo over a 52-week treatment period in in each of the groups (patients with severe asthma and high eosinophil counts and all patients with severe asthma). Efficacy will ...

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Lysosomal Acid Lipase (LAL) Deficiency Registry

Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lysosomal storage disorder (LSD) that is caused by a marked decrease of lysosomal acid lipase (LAL), the enzyme that breaks down cholesteryl esters and triglycerides in the lysosomes. Lysosomal Acid Lipase Deficiency presenting in infants (historically called Wolman Disease) is ...

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Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

The lysosome is a specialized part of the cell that functions to degrade metabolic wastes in the cell. Defects in the functioning of the lysosome result in accumulation and subsequent storage of such metabolic wastes. These defects lead to conditions known as lysosomal storage diseases (LSD). LSDs are caused by ...

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Fabry Disease Registry

The Fabry Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact: In Asia-Pacific - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com In Europe - +31-35-699-1232, europe@FabryRegistry.com In Latin America - +617-591-5500, help@FabryRegistry.com In North America - +617-591-5500, help@FabryRegistry.com

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Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

The primary objective of this prospective, multicenter, double-blind, randomized, placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily collection of patient-reported outcomes with an electronic diary.

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