*A Phase 1, Safety, Tolerability, and PK study of PBI-4050 compared to Sodium Phenylbutyrate
Research type
Research Study
Full title
A Phase 1, Randomised, Open-Label, Crossover Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses of PBI-4050 compared to Sodium Phenylbutyrate in Healthy Subjects
IRAS ID
1005293
Contact name
Bruce Pritchard
Contact email
Sponsor organisation
Liminal BioSciences Ltd.
Eudract number
2022-000804-36
Clinicaltrials.gov Identifier
Research summary
This study is being conducted to find out if the drug Fezagepras (PBI-4050) is safe and can be used in the same way as the drug Ammonaps. PBI-4050 is being studied to treat hyperammonemia. Hyperammonemia is a condition where the body is unable to remove ammonia (which is a toxin) effectively because of diseases that either affect the kidney, liver or both. The way it removes the toxin is by a process called nitrogen scavenging. Ammonia plays a vital role in the nitrogen and amino acid metabolism. However, in high concentrations it is toxic and is implicated in several disease conditions one of which is hepatic encephalopathy.
Ammonaps is an approved drug that is available in pharmacies. Its active ingredient is Sodium Phenylbutyrate (PBA) which is used together with a proper diet to help treat urea cycle disorders (including a specific liver enzyme deficiency) that help remove ammonia (nitrogen) from the body. Ammonia is formed from the breakdown of protein in the body.
This study is a part of a process aimed at making a new drug product, Fezagepras (PBI-4050) available for the treatment of the condition mentioned above, which the drug product Ammonaps is already approved for. The study will also assess the scavenging capabilities of PBI-4050 in the selected higher doses. The scavenging capabilities of PBI-4050 will be compared to those of PBA, a well characterized, marketed, nitrogen scavenger in the estimated equivalent doses. This study design is crossover to eliminate bias. 16 healthy males and females of non-child bearing potential to be enrolled. Participants will receive either single oral dose of either 3.6 g (PBI-4050) in one period and 3 g Ammonaps (PBA) in another, or 7.2 g (PBI-4050) in one period, and 6 g Ammonaps (PBA) in their second period. The study duration is around 42 days at Celerion Belfast. Each volunteer to be on the trial for up to 16 days plus up to 28 screening days. The trial is funded by Liminal Biosciences as the sponsor.REC name
North East - York Research Ethics Committee
REC reference
22/NE/0147
Date of REC Opinion
28 Apr 2022
REC opinion
Further Information Favourable Opinion