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SAR444836

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

Ensayo de Sanofi en Fenilcetonuria.

Más ensayos de: Fenilcetonuria.

Fase

Fase 1/2

Estado

En marcha, sin reclutar

Participantes

32

previstos

Centros

11

Fin del objetivo primario

abr 2030

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en ago 2023.

Qué mide

Incidence of treatment-emergent adverse events (TEAEs) (From Baseline to Week 260) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria (PKU) on a chronic, stable diet. The purpose of the study is to evaluate the safety and efficacy of SAR444836 in reducing phenylalanine (Phe) levels and in the elimination of a Phe restricted diet. Participants will receive a one-time intravenous (IV) administration of SAR444836. The study is constituted of 2 separate parts: a dose escalation part, and a dose expansion part where subsequent participants will be administered a safe and effective dose level identified during the dose escalation part. In both study parts, clinical and laboratory assessments will be collected to: a) assess the incidence of adverse events, and b) evaluate the effect of SAR444836 on reductions in blood Phe levels and maintenance of these Phe levels after elimination of a Phe restricted diet. The study duration will be for a minimum duration of 5 years following SAR444836 administration, for each participant and includes a 6-week screening phase, a 96-week treatment follow-up period after SAR444836 administration, followed by an additional 3 years of long-term safety (and efficacy) monitoring. There will be a total of 47 study visits. Many study visits may occur as remote visits and be performed by a qualified in-home service provider. Actual study duration for an individual participant may be longer than 5 years due to the administration of SAR444836 to participants in Stage 1A in a serial fashion, the duration of the screening period, and/or other factors such as delays in scheduling study visits.

Más ensayos de SNY

Todos los de SNY, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT05972629), actualizada en sep 2025.