K020-218 UNLOCKED: Phase 2, Open Label, KB195 in Urea Cycle Disorders
Research type
Research Study
Full 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
IRAS ID
262079
Contact name
Robin Lachmann
Contact email
Sponsor organisation
Kaleido Biosciences, Inc.
Eudract number
2018-004842-40
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
138734, IND
Duration of Study in the UK
1 years, 1 months, 30 days
Research summary
The study is being done to find out how safe the study drug, KB195, is and how effective it is in patients with a urea cycle disorder (UCD). UCDs are a rare condition caused by a mutation that makes it hard for the body to remove a substance called ammonia. This mutation leads to high levels of ammonia in the blood and this can cause problems, including the possibility of causing swelling and damage in the brain. The goal of current treatment for UCD is to lower the amount of ammonia to a safe level in the blood. This may be done through dietary changes (such as eating a smaller amount of protein) or taking dietary supplements or medication. While standard-of-care treatment has been shown to help manage the symptoms of UCD for some patients, for others, they do not. That is why there is a need to find new treatment options for UCD.
The study drug, KB195, is a powder provided in a packet; each packet contains 9 grams of study drug powder. The powder will need to be mixed with about half a glass of water before taking it. Participants will be asked to take the study drug twice per day (once in the morning and once in the evening) for 8 weeks during the treatment period. The participants will receive 4 different dose levels of the study drug, starting with the lowest, Dose Level 1, to the highest, Dose Level 4.
The study includes a screening period (4 weeks); a Baseline visit after successful screening (1 day); the treatment period (8 weeks); the follow-up period (4 weeks); and an end-of-study visit (1 day).
Approximately 29 patients with UCDs are planned to participate in this study.
REC name
London - West London & GTAC Research Ethics Committee
REC reference
19/LO/0959
Date of REC Opinion
30 Aug 2019
REC opinion
Further Information Favourable Opinion