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PTC923 for Phenylketonuria

Phase 3
Waitlist Available
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up predose, 0.5, 1, 2, 4, 6, 8, and 24 hours postdose at day 1; 2 and 6 hours postdose at day 14
Awards & highlights

Study Summary

This trial is testing a new drug to see if it can reduce blood Phe levels in people with phenylketonuria.

Eligible Conditions
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~predose, 0.5, 1, 2, 4, 6, 8, and 24 hours postdose at day 1; 2 and 6 hours postdose at day 14
This trial's timeline: 3 weeks for screening, Varies for treatment, and predose, 0.5, 1, 2, 4, 6, 8, and 24 hours postdose at day 1; 2 and 6 hours postdose at day 14 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phenylketonuria (Phe) Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1
Part 2 Double-blind Phase: Percent Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1
Secondary outcome measures
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Part 1 Open-label Run-in Phase: Area Under the Concentration-time Curve From Time 0 to 24 Hours Postdose (AUC0-24h) of Sepiapterin and BH4 Following the First Dose of Sepiapterin at 60 mg/kg
Part 1 Open-label Run-in Phase: Plasma Concentration of Tetrahydrobiopterin (BH4) and Sepiapterin
+5 more
Other outcome measures
Part 1 Open-label Run-in Phase: Mean Change From Baseline (Part 1) in Blood Phe Level to Weeks 1 and 2 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1
Part 1 Open-label Run-in Phase: Percent Change From Baseline (Part 1) in Blood Phe Level to Weeks 1 and 2 (Averaged Over a 2-week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1
Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level to Weeks 5 and 6 (Averaged Over a 2-week Period) in Classical PKU Participants With Phe Reduction From Baseline ≥30% During Part 1
+1 more

Side effects data

From 2023 Phase 3 trial • 157 Patients • NCT05099640
5%
Diarrhoea
4%
Upper respiratory tract infection
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 2: Placebo
Part 1: Sepiapterin
Part 2: Sepiapterin

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Part 2: PTC923Experimental Treatment1 Intervention
Participants will receive PTC923 20 mg/kg daily for Weeks 1 and 2, then PTC923 40 mg/kg daily for Weeks 3 and 4, then PTC923 60 mg/kg daily for Weeks 5 and 6.
Group II: Part 1: PTC923Experimental Treatment1 Intervention
Participants will receive PTC923 7.5 milligrams (mg)/kilogram (kg) (participants 0 to <6 months of age), 15 mg/kg (participants 6 to <12 months of age), 30 mg/kg (participants 12 months to <2 years of age), or 60 mg/kg (participants ≥2 years of age) orally once daily for 14 days.
Group III: Part 2: PlaceboPlacebo Group1 Intervention
Participants will receive equivalent quantities of placebo to match the 20 to 40 to 60 mg/kg dose escalation of the PTC923 treatment arm.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
PTC923
2019
Completed Phase 3
~170

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,073 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many people are eligible to partake in this research?

"A total of 178 patients that meet the study's inclusion criteria are required in order to carry out the clinical trial. The sponsor, PTC Therapeutics, will be administering the trial at various sites including The Children's Hospital of Philadelphia in Philadelphia, Pennsylvania and Medical College of Wisconsin in Milwaukee, Wisconsin."

Answered by AI

Are there short or long-term risks associated with PTC923?

"PTC923 is in Phase 3 of clinical trials, which puts its safety at a 3."

Answered by AI

How many different sites are responsible for conducting this trial?

"Currently, there are 14 different hospitals participating in this study. 3 of these locations are The Children's Hospital of Philadelphia in Philadelphia, Medical College of Wisconsin in Milwaukee, and UF College of Medicine, Department of Pediatrics Division of Genetics and Metabolism in Gainesville. The other 11 locations are _____, _____, _____, _____, _____, _____, _____, _____, _____, _____, _____."

Answered by AI

Who else is applying?

What state do they live in?
Michigan
How old are they?
< 18
What site did they apply to?
Indiana University School of Medicine
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
Recent research and studies
clinicaltrials.govStudy
A Study of PTC923 in Participants With Phenylketonuria
2021. "A Study of PTC923 in Participants With Phenylketonuria". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05099640.
~45 spots leftby Apr 2025
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