PTC923

DRUG6 trials

Sponsors

PTC Therapeutics Inc., PTC Therapeutics

Conditions

BH4 DeficiencyHyperphenylalaninemiaMetabolic Disorders - PhenylketonuriaPhenylketonuria

Phase 1

A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

CompletedNCT03519711

PTC TherapeuticsBH4 Deficiency, Hyperphenylalaninemia

Start: 2019-01-03End: 2020-10-02Updated: 2023-11-14

Phase 3

A Study of PTC923 in Participants With Phenylketonuria

CompletedNCT05099640

PTC TherapeuticsPhenylketonuria

Start: 2021-09-30End: 2023-05-03Updated: 2024-01-10

A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

CompletedNCT05166161

PTC TherapeuticsPhenylketonuria

Start: 2022-02-14End: 2026-07-21Updated: 2026-09-09

A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria

Active, not recruitingCTIS2023-509229-31-00

PTC Therapeutics Inc.Metabolic Disorders - Phenylketonuria

Start: 2022-09-28Target: 80Updated: 2025-10-30

A Phase 3, Randomized, Crossover, Open-Label, Active-Controlled Study of Sepiapterin versus Sapropterin in Participants With Phenylketonuria ≥2 years of Age

CompletedCTIS2023-506238-61-00

PTC Therapeutics Inc.Phenylketonuria

Start: 2024-05-10End: 2025-01-27Target: 119Updated: 2025-12-22

A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin

RecruitingCTIS2024-514435-20-00

PTC Therapeutics Inc.Phenylketonuria

Start: 2025-12-23Target: 60Updated: 2025-08-15

Related Papers

Effect of long-term sepiapterin treatment on dietary phenylalanine tolerance in patients with phenylketonuria: interim results from the Phase 3 APHENITY Extension Study.

2026-01-12

Sepiapterin as a treatment for people living with phenylketonuria: a plain language summary of the APHENITY trial.

2025-12-09

Effects of oral sepiapterin on blood Phe concentration in a broad range of patients with phenylketonuria (APHENITY): results of an international, phase 3, randomised, double-blind, placebo-controlled trial.

2024-10-0121 citations