NCT03797131

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jan 2019

Shorter than P25 for not_applicable

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

December 7, 2018

Completed
1 month until next milestone

Study Start

First participant enrolled

January 6, 2019

Completed
3 days until next milestone

First Posted

Study publicly available on registry

January 9, 2019

Completed
8 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2019

Completed
Last Updated

January 10, 2020

Status Verified

January 1, 2020

Enrollment Period

8 months

First QC Date

December 7, 2018

Last Update Submit

January 8, 2020

Conditions

Urea Cycle Disorders

Keywords

KB195Urea Cycle DisordersUCDmicrobiomeKaleidoammonia

Outcome Measures

Primary Outcomes (3)

  • 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 Outcomes (2)

  • 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

Other Outcomes (1)

  • Incidence of adverse events

    Baseline to Day 32

Study Arms (1)

KB195 Arm

EXPERIMENTAL
Other: KB195 (a novel mixture of oligosaccharides)

Interventions

KB195 (a novel mixture of oligosaccharides)OTHER

KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.

KB195 Arm

Eligibility Criteria

Age14 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Children's Hospital Zurich

Zurich, CH-8032, Switzerland

Location

MeSH Terms

Conditions

Urea Cycle Disorders, Inborn

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesAmino Acid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Mark Wingertzahn

    Kaleido Biosciences

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NA
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 7, 2018

First Posted

January 9, 2019

Study Start

January 6, 2019

Primary Completion

September 1, 2019

Study Completion

September 1, 2019

Last Updated

January 10, 2020

Record last verified: 2020-01

Data Sharing

IPD Sharing
Will not share

Locations

CH(1)