Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Urea Cycle Disorders

Intervention / Treatment
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* Other: KB195 (a novel mixture of oligosaccharides)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 14 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Basic Science
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: KB195 Arm<br> | Other: KB195 (a novel mixture of oligosaccharides)<br>* KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in labelled (15N-nitrogen) and total nitrogen excretion in urine | 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 | Baseline to Day 25 | 
| Change in labelled (15N-urea) and total urea excretion in urine | 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 | Baseline to Day 25 | 
| Change in labelled (15N-ammonia) and total ammonia excretion in urine | 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 | Baseline to Day 25 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires | Evaluate the effect of KB195 on self-report questionnaires including the Gastrointestinal Tolerability Questionnaire, an assessment of the frequency and severity of GI symptoms, e.g., gas, abdominal pain, calculated on a scale from 0 (None/Not applicable) to a maximum score of 60 (Severe/Much more than usual) for all questions | Baseline to Day 32 | 
| Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires | Evaluate the effect of KB195 on self-report questionnaires including the Bristol Stool Scale, an assessment of stool consistency on a scale from 1 (separate hard lumps, like nuts, hard to pass) through 7 (watery, no solid pieces, entirely liquid). | Baseline to Day 32 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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KB195, Urea Cycle Disorders, UCD, microbiome, Kaleido, ammonia