CompletedNot Applicable

Nitric Oxide Supplementation on Neurocognitive Functions in Patients With ASLD

United States16 participantsStarted 2017-09-15

Plain-language 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.

Who can participate

Age range6 Years – 50 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Age \> 6 and \<50 years
✓. Diagnosis of ASLD confirmed by biochemical OR enzymatic OR genetic testing
✓. Has a history of compliance with diet and treatment
✓. Negative pregnancy test and ability to use birth control method for the entire duration of the study (if the subject is of child-bearing potential)
✓. Males who enroll in the study (and their partners) should argee to use an acceptable form of birth control for the entire duration of the study

Exclusion criteria

✕. Clinical or laboratory abnormality of Grade 3 or greater according to the CTCAE (or for conditions not covered by the CTCAE, a severe or life-threatening toxicity) at enrollment which, in the view of the investigator compromises safety. (Elevated plasma levels of aspartate and alanine aminotransferases, or low serum potassium will not be considered as exclusion criteria as these are phenotypic manifestations of ASLD.)
✕. Known hypersensitivity to Neo-ASA or nitrite
✕. Individuals currently being administered other investigational agents

What they're measuring

Delis-Kaplan Executive Function System - Tower subtest

Timeframe: 24 weeks

Stanford-Binet - 4th Edition: Bead Memory and Sentence Memory subtests

Timeframe: 24 weeks

Grip Strength

Timeframe: 24 weeks

Grooved Pegboard

Timeframe: 24 weeks

Wechsler Intelligence Scale for Children OR Wechsler Adult Intelligence Scale - 4th Edition (in subjects > 16 years of age)

Timeframe: 24 weeks

Tower of London Test

Timeframe: 24 weeks

Conners Continuous Performance Test - 3rd Edition Conners Continuous Performance Test - 3rd Edition

Timeframe: 24 weeks

Trial details

NCT IDNCT03064048

SponsorBaylor College of Medicine

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2023-01-31

View on ClinicalTrials.gov More Argininosuccinate Lyase Deficiency trials

Plain-language summary

Who can participate

Age range6 Years – 50 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Age \> 6 and \<50 years
✓. Diagnosis of ASLD confirmed by biochemical OR enzymatic OR genetic testing
✓. Has a history of compliance with diet and treatment
✓. Negative pregnancy test and ability to use birth control method for the entire duration of the study (if the subject is of child-bearing potential)
✓. Males who enroll in the study (and their partners) should argee to use an acceptable form of birth control for the entire duration of the study

Exclusion criteria

✕. Clinical or laboratory abnormality of Grade 3 or greater according to the CTCAE (or for conditions not covered by the CTCAE, a severe or life-threatening toxicity) at enrollment which, in the view of the investigator compromises safety. (Elevated plasma levels of aspartate and alanine aminotransferases, or low serum potassium will not be considered as exclusion criteria as these are phenotypic manifestations of ASLD.)
✕. Known hypersensitivity to Neo-ASA or nitrite
✕. Individuals currently being administered other investigational agents

What they're measuring

Delis-Kaplan Executive Function System - Tower subtest

Timeframe: 24 weeks

Stanford-Binet - 4th Edition: Bead Memory and Sentence Memory subtests

Timeframe: 24 weeks

Grip Strength

Timeframe: 24 weeks

Grooved Pegboard

Timeframe: 24 weeks

Wechsler Intelligence Scale for Children OR Wechsler Adult Intelligence Scale - 4th Edition (in subjects > 16 years of age)

Timeframe: 24 weeks

Tower of London Test

Timeframe: 24 weeks

Conners Continuous Performance Test - 3rd Edition Conners Continuous Performance Test - 3rd Edition

Timeframe: 24 weeks