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Clinical trial information and results are updated daily from ClinicalTrials.gov. The latest data update was conducted on 01/20/2021.

UNLOCKED: A Phase 2, Open-label Trial With KB195 in Subjects With a Urea Cycle Disorder

Clinicaltrials.gov identifier NCT03933410

Recruitment Status Recruiting

First Posted May 1, 2019

Last update posted October 7, 2020

Study Description

Brief 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

  • Condition or Disease:Urea Cycle Disorder
  • Intervention/Treatment: Drug: KB195
  • Phase: Phase 2
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
  • Study Type: Interventional
  • Estimated Enrollment: 24 participants
  • 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 2019
  • Estimated Primary Completion Date: October 2021
  • Estimated Study Completion Date: November 2021
Arms and interventions
Arm Intervention/treatment
Experimental: KB195
KB195 is a novel glycan
Drug: KB195
KB195 is a novel glycan
Outcome Measures
  • Primary Outcome Measures: 1. 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 ]
  • Secondary Outcome Measures: 1. Number of subjects experiencing adverse events (AEs) [ Time Frame: Day -28 to Day 84 ]
  • 2. Number of subjects experiencing severe adverse events (SAEs) [ Time Frame: Day -28 to Day 84 ]
  • 3. 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 ]
  • 4. 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
  • 5. 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)
Eligibility Criteria
  • Ages Eligible for Study: 12 to 70 Years (Child, Adult, Older Adult)
  • Sexes Eligible for Study: All
  • Accepts Healthy Volunteers: No
Criteria

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

Contacts and Locations
Contacts

Contact: Mark Wingertzahn, PhD (617) 674-9000 clinicalstudies@kaleido.com

Locations
Show 19 Study Locations
Sponsors and Collaborators

Kaleido Biosciences

Investigators

Study Director: Mark Wingertzahn, PhD Kaleido Biosciences

More Information
  • Responsible Party: Kaleido Biosciences
  • ClinicalTrials.gov Identifier: NCT03933410 History of Changes
  • Other Study ID Numbers: K020-218
  • First Posted: May 1, 2019 Key Record Dates
  • Last Update Posted: October 7, 2020
  • Last Verified: October 2020
  • Studies a U.S. FDA-regulated Drug Product: Yes
  • Studies a U.S. FDA-regulated Device Product: No
  • Keywords provided by Kaleido Biosciences: Microbiome
    Elevated Ammonia
    Disorder of the Urea Cycle Metabolism
    Urea Cycle Disorder
    KB195
    Kaleido
    UNLOCKED
    Ornithine Transcarbamylase Deficiency (OTC)
    Glycan
    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)
    Nitrogen Binding Therapy (NBT)
    Quality of Life
    Oligosaccharide
    Pediatric
    Glutamine
  • Additional relevant MeSH terms: Urea Cycle Disorders, Inborn Disease