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

Urea Cycle Disorders Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03797131
• Other study ID #: K013-118
• Status: Completed
• Phase: N/A
• Start date: January 6, 2019
• Est. completion date: September 1, 2019

Study information

• Verified date: January 2020
• Source: Kaleido Biosciences
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: September 1, 2019
• Est. primary completion date: September 1, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Disease
• Urea Cycle Disorders
• Urea Cycle Disorders, Inborn

Intervention

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

Locations

Country	Name	City	State
Switzerland	University Children's Hospital Zurich	Zürich

Sponsors (1)

Lead Sponsor	Collaborator
Kaleido Biosciences

Country where clinical trial is conducted

Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Incidence of adverse events		Baseline to Day 32
Primary	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
Primary	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
Primary	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	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
Secondary	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