To evaluate safety, tolerability and efficacy of SAR444836 in adult participants with PKU
Research type
Research Study
Full title
A Phase 1/Phase 2, open-label, dose-escalation, and dose expansion study to evaluate the safety, tolerability, and efficacy of SAR444836, an adeno-associated viral vector-mediated gene transfer of human phenylalanine hydroxylase, in adult participants with phenylketonuria.
IRAS ID
1009704
Contact name
Patrick Maury
Contact email
Sponsor organisation
Sanofi-Aventis Recherche & Développement
Clinicaltrials.gov Identifier
Research summary
The sponsor is testing a new medicine SAR444836 in patients with phenylketonuria (PKU). PKU is a genetic condition where the body can't break down an amino acid called phenylalanine properly. This happens due to changes in a gene responsible for making an enzyme called phenylalanine hydroxylase (PAH). This enzyme normally helps convert phenylalanine into another amino acid called tyrosine, which is important for many bodily functions. In PKU, high levels of phenylalanine and related compounds build up in the blood and brain, leading to brain damage, neurological issues, and learning problems.
SAR444836 uses a virus to deliver a working copy of the PAH gene into liver cells. The hope is that this gene will help produce enough of the missing enzyme to keep phenylalanine levels stable, similar to those in healthy individuals. The goal is to see if SAR444836 can safely reduce levels of phenylalanine (Phe) and allow for less strict dietary restrictions.
Participants will receive SAR444836 through a one-time intravenous injection after giving consent. The study has two parts: first part 1A, testing different doses of SAR444836 to find the safest and most effective one, and second part 1B, giving more participants the chosen dose to confirm its safety and effectiveness. Throughout the study, researchers will monitor participants for any side effects and measure how well SAR444836 lowers Phe levels both initially and after easing dietary restrictions. The trial is looking to recruit patients between 18-65 years of age excluding women of childbearing potential, across NHS hospital in the UK. The study will last approximately 102 weeks (about 2 years) for each participant whether in part 1A or 1B. This includes approximately 6 weeks for the screening period, 1 day for the treatment period and approximately 96 weeks for the follow-up period where various tests and questionnaires will be used to monitor patient safety and treatment efficiency.REC name
London - West London & GTAC Research Ethics Committee
REC reference
24/LO/0214
Date of REC Opinion
29 Jul 2024
REC opinion
Further Information Unfavourable Opinion