Urea Cycle Disorder Treatment with KB195: The UNLOCKED Study – Phase II

Study to Evaluate the Efficacy and Safety of KB195 in Subjects with a Urea Cycle Disorder with Inadequate Control on Standard of Care

Protocol Description

This international multi-center study evaluates the drug KB195 when used to treat urea cycle disorders (UCDs). Rare genetic conditions, UCDs cause accumulation of high levels of ammonia in the blood, which can lead to brain damage and other serious problems. Researchers will be monitoring to see if KB195 can reduce the amount of ammonia in blood of people with UCD.

Study Level: Phase II

Eligibility Criteria

Subject to certain exclusion criteria, candidates must have a confirmed UCD diagnosis for which the standard of care (dietary control) has not controlled the disease well.
Males and Females: Ages 12 to 70


All participants in the study will receive KB195, which will be provided as a powder to be mixed with water before being taken orally, twice a day. The dosage level will increase on days 5, 9 and 13. The final dose level will continue until the end of the dosing period (day 56). For screening, physical examinations, blood testing, and other evaluations, 9 study visits are scheduled. Additionally researchers will contact participants by telephone at specific intervals.
Visits: At least 10
Duration: About 4 months

Status: Open to Enrollment

Source(s) of Support

Kaleido Biosciences

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Mark Tumblin, Clinical Research Coordinator