Urea Cycle DisorderPhase 2UNLOCKEDNCT03933410
UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder
A Phase 2 study testing KB195 in people with Urea Cycle Disorder, run by Kaleido Biosciences at 19 sites in 7 countries.
Trial summary
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Urea Cycle Disorder
- What is being tested?
- KB195
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 13 participants
- Where
- 19 sitesin 7 countries
- Study dates
- 17 Sep 2019to 2 Mar 2021
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Signed informed consent and willing to comply with protocol-specified procedures.
- Has any confirmed UCD other than N-acetyl glutamatesynthase (NAGS) deficiency.
- Is male or female, 12 to 70 years of age (inclusive)
- If ≥ 18 years old, has a BMI ≥20.0 and < 40.0 kg/m2. If < 18 years old, has a BMI between 5th percentile and 95th percentile and weight greater than 5th percentile according to age, sex and regionally appropriate growth chart
- Has evidence of poorly controlled disease on the current standard of care (SOC)
- If NBT is part of SOC, is on a stable dose and regimen for at least 4 weeks before Screening and the dose is expected to remain stable during the study
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- Is willing to maintain a stable diet throughout the course of study and is willing to continue usual exercise routine.
- If taking probiotics or prebiotics, is on a stable dose regimen for at least 4 weeks before Screening and the dose and regimen are expected to remain stable during the study
- Has a negative urine screen for drugs of abuse at Screening
- If male or female of child bearing potential, agree with use effective method of contraception for the duration of the study and 90 days after last dose of study product
Who cannot join
- Is at a high risk for metabolic decomposition.
- Has had a substantive change in diet or any other aspect of UCD management within 4 weeks before the Screening Visit
- Has used a systemic anti-infective within 4 weeks before the Screening Visit, or use is anticipated during the study
- Has been diagnosed with Citrullinemia Type II
- Is receiving any systemically administered immunosuppressant medication on a chronic basis
- Has changed the use of or dose of any drug or other compound to modulate GI motility within 4 weeks before the Screening Visit, or the use or dose is expected change during the course of the study
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- Has a history of or active GI or liver disease
- Has a prior solid organ transplantation including liver transplantation, or is anticipated to receive a liver transplant during study participation
- Has used an investigational drug, product, or device within 30 days before the Screening Visit
- Has a contraindication, sensitivity, or known allergy to the study drug
- Is considered, in the opinion of the PI, to likely be a poor attendee or unlikely for any reason to be able to comply with the study drug procedures
Trial locations and contacts
19 sites in 7 countries.
19 sites in 7 countries
United States6 sites
Johns Hopkins University School of Medicine
Baltimore, Maryland, 12105
Icahn School of Medicine at Mount Sinai-Clinical Research Unit
New York, New York, 10029
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Washington University School of Medicine
St Louis, Missouri, 63110
University of South Florida/ USF HEALTH
Tampa, Florida, 33606
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Spain4 sites
Hospital de Cruces
Barakaldo, 48903
Hosptial Universitari Vall d'Hebron
Barcelona, 08035
Hospital Universitario Reina Sofia
Córdoba, 14004
Hospital Universitario Virgen del Rocio
Seville, 41013
United Kingdom3 sites
National Hospital for Neurology and Neurosurgery
London, WC1N3BG
Salford Royal Hospital
Salford, Greater Manchester, M68HD
Salford Royal NHS Foundation Trust
Salford, M68HD
Belgium2 sites
Cliniques Universitaires Saint-Luc
Brussels, 1200
Universitair Ziekenhuis Gent
Ghent, 9000
Switzerland2 sites
Inselpital, Universitaetsklinik fur Kinderheikunde
Bern, 3010
Inselpital, Universitätsklinik für Kinderheilkunde
Bern, 3010
Germany1 site
Zentrum für Kinder- und Jugendmedizin Angelika-Lautenschläger-Klinik
Heidelberg, 69120
Turkey (Türkiye)1 site
Department of Child Health and Diseases, Department of Nutrition and Metabolism Istanbul University
Istanbul
About this study
Official title: A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A Urea Cycle Disorder With Inadequate Control on Standard of Care
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
Read the detailed description
We expect the trial to enroll approximately 24 Urea Cycle Disorder (UCD) patients on standard of care with elevated ammonia levels. The planned treatment duration is eight weeks, with a primary endpoint of proportion of subjects who achieve a ≥15% reduction from baseline in fasting ammonia at the end of treatment. Patients will also be followed for safety and tolerability. This clinical trial is intended to allow us to evaluate efficacy of KB195 in reducing ammonia in UCD patients.
- Conditions
- Urea Cycle Disorder
- Being tested
- KB195
- Keywords
- Microbiome, Elevated Ammonia, Disorder of the Urea Cycle Metabolism, Urea Cycle Disorder, KB195, Kaleido, UNLOCKED, Ornithine Transcarbamylase Deficiency (OTC), Carbamoyl Phosphatase Synthetase 1 (CPS1), Argininosuccinic Acid Synthetase (ASS1), Argininosuccinic Acid Lyase (ASL), Arginase (ARG 1), N-acetyl Glutamate Synthetase (NAGS), Ornithine Translocase (ORNT1), Microbiome Metabolic Therapy (MMT), Glycan, Nitrogen Binding Therapy (NBT), Quality of Life, Oligosaccharide, Pediatric
- Medical subject headings
- Urea Cycle Disorders, Inborn, Ornithine Carbamoyltransferase Deficiency Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KB195Drug
KB195 is a novel glycan
| Study group | Role | What they receive |
|---|---|---|
| KB195 | Experimental | KB195 is a novel glycan Receives: Drug: KB195 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects who achieve a ≥15% reduction from baseline in fasting plasma ammonia at the end of treatment.
Measured: Day -1 to Day 55
Secondary outcomes (5)
Proportion of subjects normalizing their fasting plasma ammonia concentrations from above the upper limit of normal at baseline to below the upper limit of normal at the end of treatment.
Measured: Day -1 to Day 55
Number of subjects experiencing adverse events (AEs)
Measured: Day -28 to Day 84
Number of subjects experiencing severe adverse events (SAEs)
Measured: Day -28 to Day 84
Change from baseline to end of treatment in Gastrointestinal Tolerability Questionnaire (GITQ) scores
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: Day -28 to Day 84
Change from baseline to end of treatment in Bristol Stool Scale (BSS) scoring.
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: Day -28 to Day 84
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 May 2019
- Study start17 Sep 2019
- Primary completion2 Feb 2021
- Study completion2 Mar 2021
Why it stopped: Kaleido Biosciences, Inc. closed the K020-218 trial due to business reasons, and not due to any safety concerns.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Urea Cycle DisorderStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- K020-218 is a single arm, open-label study
- Masking
- None (open label)
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kaleido Biosciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Mark Wingertzahn, PhD, Study director, Kaleido Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03933410
- Sponsor's protocol ID
- K020-218
- Acronym
- UNLOCKED
- Status last verified
- Jan 2022
- First submitted
- 29 Apr 2019
- Last updated
- 25 Jan 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-label Study to Evaluate the Efficacy and Safety of KB195 in Subjects With A Urea Cycle Disorder With Inadequate Control on Standard of Care
- ClinicalTrials.gov ID
- NCT03933410
- Acronym / sponsor ID
- UNLOCKED / K020-218
- Conditions
- Urea Cycle Disorder
- Keywords
- Microbiome, Elevated Ammonia, Disorder of the Urea Cycle Metabolism, Urea Cycle Disorder, KB195, Kaleido, UNLOCKED, Ornithine Transcarbamylase Deficiency (OTC)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Kaleido Biosciences, Inc. closed the K020-218 trial due to business reasons, and not due to any safety concerns.
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Kaleido BiosciencesIndustry (pharma, biotech, devices)
- Study start
- 17 Sep 2019 (actual)
- Primary completion
- 2 Feb 2021 (actual)
- Study completion
- 2 Mar 2021 (actual)
- First posted
- 1 May 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.
UNLOCKED: A Phase 2 Trial to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care
Detailed description
We expect the trial to enroll approximately 24 Urea Cycle Disorder (UCD) patients on standard of care with elevated ammonia levels. The planned treatment duration is eight weeks, with a primary endpoint of proportion of subjects who achieve a ≥15% reduction from baseline in fasting ammonia at the end of treatment. Patients will also be followed for safety and tolerability. This clinical trial is intended to allow us to evaluate efficacy of KB195 in reducing ammonia in UCD patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- K020-218 is a single arm, open-label study
| Arm | Type | Receives |
|---|---|---|
| KB195 | Experimental | Drug: KB195KB195 is a novel glycan |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Signed informed consent and willing to comply with protocol-specified procedures.
- Has any confirmed UCD other than N-acetyl glutamatesynthase (NAGS) deficiency.
- Is male or female, 12 to 70 years of age (inclusive)
- If ≥ 18 years old, has a BMI ≥20.0 and < 40.0 kg/m2. If < 18 years old, has a BMI between 5th percentile and 95th percentile and weight greater than 5th percentile according to age, sex and regionally appropriate growth chart
- Has evidence of poorly controlled disease on the current standard of care (SOC)
Key exclusion criteria (11 listed)
- Is at a high risk for metabolic decomposition.
- Has had a substantive change in diet or any other aspect of UCD management within 4 weeks before the Screening Visit
- Has used a systemic anti-infective within 4 weeks before the Screening Visit, or use is anticipated during the study
- Has been diagnosed with Citrullinemia Type II
- Is receiving any systemically administered immunosuppressant medication on a chronic basis
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 |
|---|---|---|
| KB195KB195 is a novel glycan | Drug | KB195 |
Primary outcome
Proportion of subjects who achieve a ≥15% reduction from baseline in fasting plasma ammonia at the end of treatment.
Time frame: Day -1 to Day 55
5 secondary outcomes
Proportion of subjects normalizing their fasting plasma ammonia concentrations from above the upper limit of normal at baseline to below the upper limit of normal at the end of treatment.
Time frame: Day -1 to Day 55
Number of subjects experiencing adverse events (AEs)
Time frame: Day -28 to Day 84
Number of subjects experiencing severe adverse events (SAEs)
Time frame: Day -28 to Day 84
Change from baseline to end of treatment in Gastrointestinal Tolerability Questionnaire (GITQ) scores
Time frame: Day -28 to Day 84
Change from baseline to end of treatment in Bristol Stool Scale (BSS) scoring.
Time frame: Day -28 to Day 84
Results and safety
The study was stopped early (reason given: Kaleido Biosciences, Inc. closed the K020-218 trial due to business reasons, and not due to any safety concerns.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jan 2022.
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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, PhD study director
Kaleido Biosciences
19 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing KB195 in people with Urea Cycle Disorder, run by Kaleido Biosciences at 19 sites in 7 countries.
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 10 inclusion and 11 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: stopped early, as of the sponsor's last update on 25 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States, Spain, United Kingdom, Belgium, Switzerland and 2 more countries. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Kaleido Biosciences, Inc. closed the K020-218 trial due to business reasons, and not due to any safety concerns.). Results are not available in the source record.
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?
Proportion of subjects who achieve a ≥15% reduction from baseline in fasting plasma ammonia at the end of treatment. (time frame: Day -1 to Day 55). 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 25 Jan 2022.