NCT03519711View on ClinicalTrials.gov

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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

8

Participants

Timeline

Start Date

January 3, 2019

Primary Completion Date

October 2, 2020

Study Completion Date

October 2, 2020

Conditions
BH4 DeficiencyHyperphenylalaninemia
Interventions
DRUG

PTC923

PTC923 will be administered per dose and schedule specified in arm description.

Trial Locations (4)

54449

Marshfield Clinic, Marshfield

55454

University of Minnesota, Minneapolis

75390

UT Southwestern, Dallas

84132

University of Utah Hospital, Salt Lake City

Sponsors

Lead Sponsor

PTC Therapeutics
All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY