An Open Label Pilot Study to Evaluate the Effect of KB195, a Novel Prebiotic Gluco-Oligosaccharide Mixture, in the Diet of Patients With Urea Cycle Disorders on Gut Nitrogen Metabolism
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 4
- Locations
- 2
- Primary Endpoint
- Change in labelled (15N-urea) and total urea excretion in urine
Study Overview
Brief Summary
This clinical food study aims to explore the effect of KB195, a novel mixture of oligosaccharides, on the metabolism of nitrogen by the microbiome in patients with urea cycle disorders (UCDs). This will be done using a stable isotope to assess nitrogen metabolism in the blood, urine, and stool. The study will also assess the safety and tolerability of KB195 in patients with UCDs.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Basic Science
- Masking
- None
Eligibility Criteria
- Ages
- 14 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Confirmed UCD patient at any age ≥ 14 years
- •Parental/legal guardian permission or patient's written informed consent or assent, as applicable
- •Be willing and able to comply with study requirements
Exclusion Criteria
- •Any medical condition unrelated to the sequelae of UCD
- •Patient has N-acetylglutamate synthase (NAGS) deficiency
- •Recent hospitalization or risk for metabolic decompensation
- •Liver transplantation
- •Systemic antibiotics, probiotics or prebiotics that do not align with study guidances
- •Change in dose or frequency of any drug or other compound to modulate GI motility
- •Contraindications or known allergy/sensitivity to the use of the study products
- •Use of an investigational drug, product or device within 30 days prior to Screening Visit, or current enrolment in another investigational drug, product, or device study
- •Considered to be at risk for noncompliance or unlikely for any reason to be able to comply with the study procedures
Outcomes
Primary Outcomes
Change in labelled (15N-urea) and total urea excretion in urine
Time Frame: Baseline to Day 25
Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-urea) and total urea excretion in urine
Change in labelled (15N-ammonia) and total ammonia excretion in urine
Time Frame: Baseline to Day 25
Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-ammonia) and total ammonia excretion in urine
Change in labelled (15N-nitrogen) and total nitrogen excretion in urine
Time Frame: Baseline to Day 25
Evaluate the effect of KB195 on nitrogen metabolism in the gut of patients with UCD by analyzing the change in labelled (15N-nitrogen) and total nitrogen excretion in urine
Secondary Outcomes
- Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires(Baseline to Day 32)
- Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires(Baseline to Day 32)