Urea Cycle Disorders
Conditions
Keywords
KB195, Urea Cycle Disorders, UCD, microbiome, Kaleido, ammonia
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.
Interventions
OTHERKB195 (a novel mixture of oligosaccharides)
KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.
Sponsors
Kaleido Biosciences
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
14 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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). |
Other
| Measure | Time frame |
|---|---|
| Incidence of adverse events | Baseline to Day 32 |
Countries
Switzerland
Outcome results
None listed