A Study of PTC923 in Participants With Phenylketonuria

Status Completed

Clinical Trial Summary

The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (that is, the average of each respective treatment dose 2-week period of double-blind treatment).

Clinical Trial Description

The study includes 2 parts: Part 1 and 2. Part 1 of the study tests for responsiveness to PTC923, with 14 days of open-label treatment with PTC923. At the end of treatment in Part 1, the mean change in blood Phe levels over the 14-day treatment period for all participants will be assessed against their pretreatment (baseline) blood Phe level. Participants ≥2 years of age who experience a <15% reduction in blood Phe levels will be classified as non-responsive and participation in the study will be terminated. Participants (≥2 years of age) who experience a ≥15% reduction in blood Phe levels will continue into Part 2. Participants <2 years of age who experience ≥15% reduction in blood Phe levels will be offered the option to enroll directly into an open-label extension Study PTC923-MD-004-PKU. Participants <2 years of age who experience a <15% reduction in blood Phe levels will be classified as nonresponsive, and participation in the study will be terminated. Following the minimum 14-day PTC923 washout period, all eligible participants will be randomized in Part 2 to receive either PTC923 or placebo. After 6 weeks of treatment with either PTC923 or placebo, participants will be offered the option to enter an open-label extension Study PTC923-MD-004-PKU (NCT05166161). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT05099640
Study type	Interventional
Source	PTC Therapeutics
Contact
Status	Completed
Phase	Phase 3
Start date	September 30, 2021
Completion date	May 3, 2023

View Details

See also
Status	Clinical Trial	Phase
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	`NCT01965691` - Protein Requirements in Children With Phenylketonuria (PKU)	N/A
Completed	`NCT01965912` - Kuvan®'s Effect on the Cognition of Children With Phenylketonuria	Phase 4
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
Completed	`NCT01560286` - A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks	Phase 2