Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
Primary Purpose
Urea Cycle Disorders
Status
Completed
Phase
Not Applicable
Locations
Switzerland
Study Type
Interventional
Intervention
KB195 (a novel mixture of oligosaccharides)
Sponsored by
About this trial
This is an interventional basic science trial for Urea Cycle Disorders focused on measuring KB195, Urea Cycle Disorders, UCD, microbiome, Kaleido, ammonia
Eligibility Criteria
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
Sites / Locations
- University Children's Hospital Zurich
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
KB195 Arm
Arm Description
Outcomes
Primary Outcome Measures
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
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
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
Secondary Outcome Measures
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
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).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03797131
Brief Title
Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
January 2020
Overall Recruitment Status
Completed
Study Start Date
January 6, 2019 (Actual)
Primary Completion Date
September 1, 2019 (Actual)
Study Completion Date
September 1, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Kaleido Biosciences
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Urea Cycle Disorders
Keywords
KB195, Urea Cycle Disorders, UCD, microbiome, Kaleido, ammonia
7. Study Design
Primary Purpose
Basic Science
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
KB195 Arm
Arm Type
Experimental
Intervention Type
Other
Intervention Name(s)
KB195 (a novel mixture of oligosaccharides)
Intervention Description
KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.
Primary Outcome Measure Information:
Title
Change in labelled (15N-nitrogen) and total nitrogen excretion in urine
Description
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
Time Frame
Baseline to Day 25
Title
Change in labelled (15N-urea) and total urea excretion in urine
Description
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
Time Frame
Baseline to Day 25
Title
Change in labelled (15N-ammonia) and total ammonia excretion in urine
Description
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
Time Frame
Baseline to Day 25
Secondary Outcome Measure Information:
Title
Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires
Description
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
Time Frame
Baseline to Day 32
Title
Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires
Description
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).
Time Frame
Baseline to Day 32
Other Pre-specified Outcome Measures:
Title
Incidence of adverse events
Time Frame
Baseline to Day 32
10. Eligibility
Sex
All
Minimum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mark Wingertzahn
Organizational Affiliation
Kaleido Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
University Children's Hospital Zurich
City
Zürich
ZIP/Postal Code
CH-8032
Country
Switzerland
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
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