Purpose

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

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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, 18 to 65 years of age (inclusive)
  • Has a BMI ≥20.0 and < 40.0 kg/m2
  • 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

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 received a vaccination within 2 weeks before the Screening Visit
  • 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

Study Design

Phase
Phase 2
Study Type
Interventional
Intervention Model
Single Group Assignment
Intervention Model Description
K020-218 is a single arm, open-label study
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
KB195
KB195 is a novel glycan
  • Drug: KB195
    KB195 is a novel glycan

Recruiting Locations

Children's National and nearby locations

Children's National Medical Center
Washington, District of Columbia 20010

More Details

NCT ID
NCT03933410
Status
Recruiting
Sponsor
Kaleido Biosciences

Study Contact

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

Detailed Description

We expect the trial to enroll approximately 24 adult 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 plasma ammonia levels, as measured by plasma ammonia 24-hour area under the curve. 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 and support the inclusion of pediatric patients as soon as possible.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.