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Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

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Disease Information

Descriptive Information
Brief Title † Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD
Official Title † Effect of Nitric Oxide (NO) Supplementation on Neurocognitive Measures in Argininosuccinate Lyase Deficiency (ASLD)
Brief Summary This is a study involving a dietary supplement. Patients with argininosuccinate lyase deficiency (ASLD) will be randomly assigned to receive either a nitric oxide dietary supplement or placebo for 24 weeks, and then crossed-over to receive the other treatment for 24 weeks. The investigators will assess the effects of the supplement in domains of general cognition, memory, executive functioning, and fine motor functioning in individuals with ASLD.
Detailed Description Argininosuccinate lyase deficiency (ASLD; also known as argininosuccinic aciduria) is the second most common urea cycle disorder (UCD) and accounts for 15-20% of all disorders of ureagenesis. Individuals with ASLD can have unique clinical and physiologic characteristics as compared to other UCDs. Previous work from the members of the UCDC have shown that in spite of having fewer episodes of hyperammonemia as compared to those with proximal blockade of the urea cycle, individuals with ASLD can develop intellectual and learning disabilities. Neurocognitive deficits have been observed even in individuals without any documented hyperammonemia. Furthermore, hepatic abnormalities including hepatomegaly, hepatic injury, fibrosis and even frank cirrhosis, and vascular issues like hypertension are well known in the disorder. Previous work from the members of the UCDC has demonstrated a tissue- and molecular-specific role for ASL in the generation of NO. ASL is not only required for the synthesis of L-arginine, the substrate for the synthesis of NO, but is also an integral member of a complex that is critical for synthesis of NO from arginine. Loss of ASL can thus lead to systemic and tissue-specific NO deficiencies, which could potentially contribute to the complex phenotype including the neurocognitive deficits. A rational therapeutic option would hence be to use a NOS-independent NO supplement. The purpose of this study is to determine whether a dietary NO supplement, Neo-ASA, would improve general cognition, memory, executive functioning, fine motor functioning, and attention in individuals with ASLD. In this single-center trial, double-blind, randomized, placebo-controlled, crossover study, individuals with ASLD will be assigned to receive a medication containing NO dietary supplement for 24 weeks and a placebo for 24 weeks. General cognition, memory, executive functioning, and fine motor functioning will be assessed and compared at the end of treatment with placebo and Neo-ASA.
Study Phase N/A
Study Type † Interventional
Study Design †
Primary Outcome Measure † Delis-Kaplan Executive Function System - Tower subtest
Secondary Outcome Measure †
Condition † Argininosuccinate Lyase Deficiency Urea Cycle Disorder Urea Cycle Disorders, Inborn Argininosuccinic Aciduria
Intervention † Dietary SupplementNeo-ASA
Study Arms / Comparison Groups Neo-ASA During this arm the participant will receive a lozenge with nitric oxide as a dietary supplement twice daily. Placebo During this arm the participant will receive a lozenge which will not contain nitric oxide as a dietary supplement twice daily.
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Dietary Supplement
Estimated Enrollment † 16
Start Date † April 15, 2017
Completion Date December 31, 2023
Primary Completion Date January 31, 2020
Eligibility Criteria † Inclusion Criteria: 1. Age > 6 and
Gender All
Ages 6 Years - 50 Years
Accepts Healthy Volunteers No
Contacts †† Mary A Mullins, RN, 832-822-4263, mullins@bcm.edu
Location Countries † United States
Administrative Information
NCT ID † NCT03064048
Organization ID H-40143
Secondary IDs ††
Responsible Party Principal Investigator
Study Sponsor † Baylor College of Medicine
Collaborators †† Rare Diseases Clinical Research Network
Investigators † Principal Investigator: Sandesh C Nagamani, M.D., Baylor College of Medicine
Information Provided By
Verification Date February 2017
First Received Date † February 10, 2017
Last Updated Date February 21, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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