Urea Cycle DisordersNCT03797131
Clinical Food Study to Evaluate the Effect of KB195 on Gut Nitrogen Metabolism in Patients With Urea Cycle Disorders
A study testing KB195 (a novel mixture of oligosaccharides) in people with Urea Cycle Disorders, run by Kaleido Biosciences at 1 site in Switzerland.
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. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Urea Cycle Disorders
- What is being tested?
- KB195 (a novel mixture of oligosaccharides)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteSwitzerland
- Study dates
- 6 Jan 2019to 1 Sep 2019
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
- Conditions
- Urea Cycle Disorders
- Being tested
- KB195 (a novel mixture of oligosaccharides)
- Keywords
- KB195, Urea Cycle Disorders, UCD, microbiome, Kaleido, ammonia
- Medical subject headings
- Urea Cycle Disorders, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB195 (a novel mixture of oligosaccharides)Other
KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days.
| Study group | Role | What they receive |
|---|---|---|
| KB195 Arm | Experimental | Not described Receives: Other: KB195 (a novel mixture of oligosaccharides) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: Baseline to Day 25
Secondary outcomes (2)
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
Measured: Baseline to Day 32
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).
Measured: Baseline to Day 32
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jan 2019
- Study start6 Jan 2019
- Primary completion1 Sep 2019
- Study completion1 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle DisordersStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kaleido Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Wingertzahn, Study director, Kaleido Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03797131
- Sponsor's protocol ID
- K013-118
- Status last verified
- Jan 2020
- First submitted
- 7 Dec 2018
- Last updated
- 10 Jan 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jan 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03797131
- Acronym / sponsor ID
- K013-118
- Conditions
- Urea Cycle Disorders
- Keywords
- KB195, Urea Cycle Disorders, UCD, microbiome, Kaleido, ammonia
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Kaleido BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 6 Jan 2019 (actual)
- Primary completion
- 1 Sep 2019 (actual)
- Study completion
- 1 Sep 2019 (actual)
- First posted
- 9 Jan 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| KB195 Arm | Experimental | Other: KB195 (a novel mixture of oligosaccharides) |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KB195 (a novel mixture of oligosaccharides)KB195 (a novel mixture of oligosaccharides) for oral intake for 21 days. | Other | KB195 Arm |
Primary outcome
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
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
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
2 secondary outcomes
Change in Gastrointestinal Tolerability Questionnaire (GITQ) through the collection of daily patient questionnaires
Time frame: Baseline to Day 32
Change in Bristol Stool Scale (BSS) through the collection of daily patient questionnaires
Time frame: Baseline to Day 32
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jan 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark Wingertzahn study director
Kaleido Biosciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing KB195 (a novel mixture of oligosaccharides) in people with Urea Cycle Disorders, run by Kaleido Biosciences at 1 site in Switzerland.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Jan 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Switzerland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in labelled (15N-nitrogen) and total nitrogen excretion in urine (time frame: Baseline to Day 25). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Jan 2020.