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

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

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 Overview

• Status: Completed
• Conditions: Urea Cycle Disorders
• Intervention / Treatment: Other: KB195 (a novel mixture of oligosaccharides)

• Study Type: Interventional
• Enrollment (Actual): 4
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Switzerland
  • Zürich, Switzerland, CH-8032
    • University Children's Hospital Zurich

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: BASIC_SCIENCE
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: KB195 Arm	Other: KB195 (a novel mixture of oligosaccharides); KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Time Frame
Incidence of adverse events	Baseline to Day 32

Collaborators and Investigators

• Sponsor: Kaleido Biosciences
• Investigators: Study Director: Mark Wingertzahn, Kaleido Biosciences

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 6, 2019
• Primary Completion (ACTUAL): September 1, 2019
• Study Completion (ACTUAL): September 1, 2019

Study Registration Dates

• First Submitted: December 7, 2018
• First Submitted That Met QC Criteria: January 4, 2019
• First Posted (ACTUAL): January 9, 2019

Study Record Updates

• Last Update Posted (ACTUAL): January 10, 2020
• Last Update Submitted That Met QC Criteria: January 8, 2020
• Last Verified: January 1, 2020

More Information

Terms related to this study

• Keywords: UCD; microbiome; Urea Cycle Disorders; ammonia; Kaleido; KB195
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Disease; Urea Cycle Disorders, Inborn
• Other Study ID Numbers: K013-118

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No