Nitric Oxide Supplementation in Argininosuccinic Aciduria

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-09-30

Study Completion Date

2018-05-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

L-arginine to Reduce Sympathetic Nerve Activity in CKD Patients

NCT03982160

Chronic Kidney Disease

RECRUITING PHASE4

L-Citrulline in Peripheral Artery Disease

NCT02521220

Peripheral Arterial Disease

COMPLETED NA

Effect of Sublingually Generated S-nitroso-N-acetylcysteine on Systemic Blood Pressure.

NCT05798481

Hypertension Cardiovascular Diseases

COMPLETED EARLY_PHASE1

Protective Effects of Oral L-Arginine CI-AKI

NCT02455960

Contrast Induced AKI

UNKNOWN PHASE4

L-citrulline Supplementation During Sepsis

NCT00628381

Sepsis Multiple Organ Failure

UNKNOWN PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

The investigators will perform a double blind, randomized, placebo-controlled, crossover study of NO supplementation in ASA patients assessing endothelial function and blood pressure as primary endpoints, and markers of nitric oxide production as exploratory endpoints.

Subjects will receive a nitrite-restricted diet prior to the start and during the trial period. The dietary protein prescriptions will not be altered. After screening, informed consent, subjects will undergo study procedures at the Clinical Research Center (CRC) at Texas Children's Hospital, Houston, TX.

Safety laboratories will be performed. Fibroblast and lymphoblast cultures will be established for subsequent studies.A baseline assessment of the vascular endothelial NO production and function will be assessed using FMD of brachial artery using standard techniques (1). Subjects will be randomized to receive either dietary nitric oxide supplement or placebo for 2 weeks, and then crossed-over to receive the other treatment for two weeks. The Investigational Pharmacy Services at Texas Children's Hospital will assign the initial treatment group randomly in a 1:1 ratio using established randomization procedures. Following randomization, subjects will be receive either Neo40® 40 mg/kg/day in two divided doses) or placebo for 14 days. This dose is the dietary supplemental dose and was well-tolerated in the investigators proof of concept treatment subject as well as other clinical studies in cardiovascular disease. Subjects will return on day 14 to the CRC where they will undergo assessment of FMD of the brachial artery, repeated BP measurements, and assay of NO status. The subjects will then be crossed over to receive the alternative treatment and the study procedures will be repeated in identical fashion to the initial arm.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Argininosuccinic Aciduria Argininosuccinate Lyase Deficiency Urea Cycle Disorders

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Nitric oxide supplement arm

During this arm, subjects will receive a lozenge with nitric oxide supplement

Group Type ACTIVE_COMPARATOR

Nitric oxide supplement

Intervention Type DIETARY_SUPPLEMENT

Each active comparator supplement is an all natural nitric oxide supplement. Subjects will take one lozenge twice a day.

Placebo Arm

During this arm, subjects will receive placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DIETARY_SUPPLEMENT

Placebo will not contain nitric oxide supplement.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Nitric oxide supplement

Each active comparator supplement is an all natural nitric oxide supplement. Subjects will take one lozenge twice a day.

Intervention Type DIETARY_SUPPLEMENT

Placebo

Placebo will not contain nitric oxide supplement.

Intervention Type DIETARY_SUPPLEMENT

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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

Minimum Eligible Age

8 Years

Maximum Eligible Age

64 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No