CompletedNot Applicable

Nitric Oxide Supplementation in Argininosuccinic Aciduria

United States12 participantsStarted 2014-09

Plain-language summary

This is a study involving a dietary supplement. Patients with argininosuccinic aciduria will be randomly assigned to receive either a nitric oxide dietary supplement or placebo for 2 weeks, and then crossed-over to receive the other treatment for two weeks. The investigators expect to see that : 1) Patients with ASA will have a decreased ability for their arteries to dilate due to nitric oxide deficiency, 2) Treatment of ASA with the nitric oxide supplement will improve the ability of their arteries to dilate, and 3) Through the testing of subjects' fibroblasts (cells in connective tissue that produce collagen and other fibers), the investigators hope to predict which patients may respond NO supplementation.

Who can participate

Age range8 Years – 64 Years

SexALL

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Inclusion Criteria: * A confirmed diagnosis of ASA based on biochemical, enzymatic, or genetic testing * Capable of completing study procedures * History of compliance with diet and treatment Exclusion Criteria: * An active infection (viral or bacterial), any condition(s) that may precipitate a metabolic decompensation * A hypersensitivity to nitrite * A serum creatinine \> 1.5 times above the normal * A clinical or laboratory abnormality of Grade 3 or greater according to the CTCAE V4.0 (except elevations of AST or ALT which are a part of the disease) which in the view of the Investigator compromises safety

What they're measuring

Vascular endothelial function as assessed by FMD of brachial artery

Timeframe: 2 weeks

Trial details

NCT IDNCT02252770

SponsorBaylor College of Medicine

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2018-05

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