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

Status Recruiting

Clinical Trial Summary

The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.

Clinical Trial Description

Eligible participants are: Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study (including Study PTC923-MD-003-PKU). Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels <360 μmol/L at study entry. Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT05166161
Study type	Interventional
Source	PTC Therapeutics
Contact	Patient Advocacy
Phone	1-866-562-4620
Email	medinfo@ptcbio.com
Status	Recruiting
Phase	Phase 3
Start date	February 14, 2022
Completion date	February 7, 2025

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT05099640` - A Study of PTC923 in Participants With Phenylketonuria	Phase 3
Completed	`NCT01924026` - Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study	N/A
Completed	`NCT01428258` - Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria	N/A
Completed	`NCT00925054` - Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU	Phase 2
Completed	`NCT00778206` - PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry
Recruiting	`NCT05948020` - Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria	N/A
Recruiting	`NCT05781399` - First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria	Phase 1
Completed	`NCT02555579` - Simplified Diet Approach in Phenylketonuria	N/A
Completed	`NCT03097250` - MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria
Completed	`NCT01965912` - Kuvan®'s Effect on the Cognition of Children With Phenylketonuria	Phase 4
Completed	`NCT01965691` - Protein Requirements in Children With Phenylketonuria (PKU)	N/A
Completed	`NCT00688844` - Nutritional and Neurotransmitter Changes in PKU Subjects on BH4	N/A
Completed	`NCT00789568` - A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult Subjects	Phase 1
Terminated	`NCT01465100` - Liver Cell Transplant for Phenylketonuria	Phase 1/Phase 2
Completed	`NCT01732471` - Phase 3 Open-label Study to Evaluate the Response and Safety of Kuvan® in Subjects With Phenylketonuria	Phase 3
Completed	`NCT04879277` - Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria	N/A
Completed	`NCT02176603` - Observational Study of Endothelial Dysfunction in Phenylketonuria	N/A
Completed	`NCT01819727` - An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165	Phase 3
Terminated	`NCT01904708` - Moderate Intensity Exercise and Phenylketonuria	N/A
Completed	`NCT01869972` - Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia	N/A