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

• ClinicalTrials.gov Identifier: NCT03797131
• Recruitment Status: Completed
• First Posted: January 9, 2019
• Last Update Posted: January 10, 2020
• Sponsor: Kaleido Biosciences
• Information provided by (Responsible Party): Kaleido Biosciences

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.

Condition or disease	Intervention/treatment	Phase
Urea Cycle Disorders	Other: KB195 (a novel mixture of oligosaccharides)	Not Applicable

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Basic Science
• 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
• Actual Study Start Date: January 6, 2019
• Actual Primary Completion Date: September 1, 2019
• Actual Study Completion Date: September 1, 2019

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures :

1. Change in labelled (15N-nitrogen) and total nitrogen excretion in urine [ Time Frame: 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
2. Change in labelled (15N-urea) and total urea excretion in urine [ Time Frame: 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
3. Change in labelled (15N-ammonia) and total ammonia excretion in urine [ Time Frame: 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

Secondary Outcome Measures :

1. Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires [ Time Frame: 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
2. Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires [ Time Frame: 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).

Other Outcome Measures:

1. Incidence of adverse events [ Time Frame: Baseline to Day 32 ]

Eligibility Criteria

• Ages Eligible for Study: 14 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

Switzerland

University Children's Hospital Zurich

Zürich, Switzerland, CH-8032

Sponsors and Collaborators

Kaleido Biosciences

Investigators

• Study Director: Mark Wingertzahn; Kaleido Biosciences

More Information

• Responsible Party: Kaleido Biosciences
• ClinicalTrials.gov Identifier: NCT03797131
• Other Study ID Numbers: K013-118
• First Posted: January 9, 2019
• Last Update Posted: January 10, 2020
• Last Verified: January 2020

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Kaleido Biosciences:

KB195; Urea Cycle Disorders; UCD; microbiome; Kaleido; ammonia

Additional relevant MeSH terms:

Urea Cycle Disorders, Inborn; Disease; Pathologic Processes; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases