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

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

EU CTIS

Registry ID

CTIS2023-506238-61-00

Acronym

PTC923-PKU-301

Enrollment

119

Registered

2024-04-15

Start date

2024-05-10

Completion date

2025-01-27

Last updated

2025-12-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Phenylketonuria

Brief summary

Mean change in blood Phe levels from baseline to Weeks 3 and 4 of each treatment period (the average of the last 2 weeks of each treatment period) in Part 2

Detailed description

Proportion of participants with baseline blood Phe levels ≥600 µmol/L who achieve Phe levels <600 µmol/L after each treatment period in Part 2, Proportion of participants reaching blood Phe <360 µmol/L after each treatment period in Part 2, AEs, physical examinations, vital sign assessments, 12-lead ECGs, and routine clinical laboratory assessments

Interventions

DRUGPTC923

DRUGKuvan 100 mg soluble tablets

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Mean change in blood Phe levels from baseline to Weeks 3 and 4 of each treatment period (the average of the last 2 weeks of each treatment period) in Part 2	—

Secondary

Measure	Time frame
Proportion of participants with baseline blood Phe levels ≥600 µmol/L who achieve Phe levels <600 µmol/L after each treatment period in Part 2, Proportion of participants reaching blood Phe <360 µmol/L after each treatment period in Part 2, AEs, physical examinations, vital sign assessments, 12-lead ECGs, and routine clinical laboratory assessments	—

Countries

Czechia, Denmark, France, Germany, Italy, Netherlands, Poland, Slovenia, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026