UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder (UNLOCKED)
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ClinicalTrials.gov Identifier: NCT03933410 |
Recruitment Status :
Terminated
(Kaleido Biosciences, Inc. closed the K020-218 trial due to business reasons, and not due to any safety concerns.)
First Posted : May 1, 2019
Last Update Posted : January 25, 2022
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Urea Cycle Disorder | Drug: KB195 | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 13 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | K020-218 is a single arm, open-label study |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 |
Actual Study Start Date : | September 17, 2019 |
Actual Primary Completion Date : | February 2, 2021 |
Actual Study Completion Date : | March 2, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: KB195
KB195 is a novel glycan
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Drug: KB195
KB195 is a novel glycan |
- 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 ]
- 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 ]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 from baseline to end of treatment in Bristol Stool Scale (BSS) scoring. [ Time Frame: Day -28 to Day 84 ]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)

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Ages Eligible for Study: | 18 Years to 65 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- 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
- 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
Key Exclusion Criteria:
- 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
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03933410

Study Director: | Mark Wingertzahn, PhD | Kaleido Biosciences |
Responsible Party: | Kaleido Biosciences |
ClinicalTrials.gov Identifier: | NCT03933410 |
Other Study ID Numbers: |
K020-218 |
First Posted: | May 1, 2019 Key Record Dates |
Last Update Posted: | January 25, 2022 |
Last Verified: | January 2022 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Kaleido Microbiome Elevated Ammonia Disorder of the Urea Cycle Metabolism Urea Cycle Disorder KB195 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 Glutamine |
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 |