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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

Pivotal Trial

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, weeks 1 and 2, weeks 3 and 4, and weeks 5 and 6 (average of each 2-week period)

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

Diarrhoea

Upper respiratory tract infection

100%

80%

60%

40%

20%

Study treatment Arm

Part 2: Placebo

Part 1: Sepiapterin

Part 2: Sepiapterin

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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

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