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

NCT ID: NCT03933410

Last Updated: 2022-01-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 13 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-09-17
• Study Completion Date: 2021-03-02

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

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

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

KB195

KB195 is a novel glycan

Group Type: EXPERIMENTAL

KB195

Intervention Type: DRUG

KB195 is a novel glycan

Interventions

KB195

KB195 is a novel glycan

Intervention Type: DRUG

Eligibility Criteria

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

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Kaleido Biosciences

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mark Wingertzahn, PhD

Role: STUDY_DIRECTOR

Kaleido Biosciences

Locations

Children's National Medical Center

Washington D.C., District of Columbia, United States

University of South Florida/ USF HEALTH

Tampa, Florida, United States

Johns Hopkins University School of Medicine

Baltimore, Maryland, United States

Washington University School of Medicine

St Louis, Missouri, United States

Icahn School of Medicine at Mount Sinai-Clinical Research Unit

New York, New York, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Universitair Ziekenhuis Gent

Ghent, , Belgium

Zentrum für Kinder- und Jugendmedizin Angelika-Lautenschläger-Klinik

Heidelberg, , Germany

Hospital de Cruces

Barakaldo, , Spain

Hosptial Universitari Vall d'Hebron

Barcelona, , Spain

Hospital Universitario Reina Sofia

Córdoba, , Spain

Hospital Universitario Virgen del Rocio

Seville, , Spain

Inselpital, Universitaetsklinik fur Kinderheikunde

Bern, , Switzerland

Inselpital, Universitätsklinik für Kinderheilkunde

Bern, , Switzerland

Department of Child Health and Diseases, Department of Nutrition and Metabolism Istanbul University

Istanbul, , Turkey (Türkiye)

Salford Royal Hospital

Salford, Greater Manchester, United Kingdom

National Hospital for Neurology and Neurosurgery

London, , United Kingdom

Salford Royal NHS Foundation Trust

Salford, , United Kingdom

Countries

United States; Belgium; Germany; Spain; Switzerland; Turkey (Türkiye); United Kingdom

Other Identifiers

K020-218

Identifier Type: -

Identifier Source: org_study_id