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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
~43 spots leftby Jun 2025
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