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

Not Applicable

Completed

• Conditions: Urea Cycle Disorders
• Interventions: Other: KB195 (a novel mixture of oligosaccharides)

• Registration Number: NCT03797131
• Lead Sponsor: Kaleido Biosciences

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-12-07
• Study First Submitted QC: 2019-01-04
• Study Start: 2019-01-06
• Study First Posted: 2019-01-09
• Primary Completion: 2019-09-01
• Study Completion: 2019-09-01
• Status Verified: 2020-01
• Last Update Submitted: 2020-01-08
• Last Update Posted: 2020-01-10

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 4

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
KB195 Arm	KB195 (a novel mixture of oligosaccharides)	-

Primary Outcome Measures

Name	Time	Method
Change in labelled (15N-urea) and total urea excretion in urine	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	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	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 Outcome Measures

Name	Time	Method
Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires	Baseline to Day 32	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
Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires	Baseline to Day 32	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).

Trial Locations

Locations (1)

University Children's Hospital Zurich; 🇨🇭 Zürich, Switzerland