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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 baseline, weeks 1 and 2, weeks 3 and 4, and weeks 5 and 6 (average of each 2-week period)
Awards & highlights

Summary

This trial is testing a medication called PTC923 to see if it can lower blood phenylalanine levels in people with phenylketonuria (PKU). PKU patients have trouble breaking down phenylalanine, which can lead to health issues. The study will measure how well PTC923 works by comparing blood phenylalanine levels over time.

Eligible Conditions
  • Phenylketonuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, weeks 1 and 2, weeks 3 and 4, and weeks 5 and 6 (average of each 2-week period)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, weeks 1 and 2, weeks 3 and 4, and weeks 5 and 6 (average of each 2-week period) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
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 study objectives
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Part 2 Double-blind Phase: Mean Change From Baseline in Blood Phe Level at Each 2-Week Period (Averaged Over Each 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 at Each 2-Week Period (Averaged Over Each 2-Week Period) in Participants With Phe Reduction From Baseline ≥30% During Part 1
+2 more
Other study objectives
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
6,081 Total Patients Enrolled
~40 spots leftby Sep 2025
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