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PTC923 for Phenylketonuria

Phase 3
Recruiting
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of PKU with hyperphenylalaninemia (HPA) documented by past medical history of at least 2 blood Phe measurements ≥600 μmol/L
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to end of study (up to approximately 2.5 years)
Awards & highlights

Study Summary

This trial will study the long-term effects of PTC923 in people with phenylketonuria. It will also look at how PTC923 affects dietary intake of phenylalanine and protein.

Who is the study for?
This trial is for individuals with Phenylketonuria (PKU) who have had high blood phenylalanine levels. They must stick to their current diet unless told otherwise, not be on certain other PKU treatments, and use effective contraception if applicable. People with significant lab abnormalities, renal disease, gastrointestinal issues that affect drug absorption, or serious uncontrolled neuropsychiatric illness cannot join.Check my eligibility
What is being tested?
The study tests the long-term safety of a drug called PTC923 in people with PKU. It also looks at how the treatment might allow participants to change their dietary intake of phenylalanine/protein from what they usually consume.See study design
What are the potential side effects?
While specific side effects are not listed here, this trial will monitor any adverse reactions to PTC923 over an extended period as it relates to its safety profile in treating individuals with PKU.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have PKU with two blood tests showing Phe levels over 600 μmol/L.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to end of study (up to approximately 2.5 years)
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to end of study (up to approximately 2.5 years) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From Baseline in Dietary Phe/Protein Consumption at Week 26, Measured During Phe Tolerance Assessment Period
Number of Treatment-Emergent Adverse Events (TEAEs)
Secondary outcome measures
Change From Baseline in QOL Using the European Quality of Life - 5 Dimensions (EQ-5D) at Months 8, 14, 20, 26, 32, and 38
Change From Baseline in Quality of Life (QOL) Using Phenylketonuria-Quality of Life (PKU-QOL) Questionnaire at Months 8, 14, 20, 26, 32, and 38

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

1Treatment groups
Experimental Treatment
Group I: PTC923Experimental Treatment1 Intervention
Participants will receive PTC923 7.5 mg/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 a minimum of 12 months or until participant experiences lack of efficacy, adverse events (AEs) that lead to discontinuation, withdraws from treatment, or PTC923 is authorized and commercially available in the specific country.
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
6,030 Total Patients Enrolled

Media Library

PTC923 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05166161 — Phase 3
Phenylketonuria Research Study Groups: PTC923
Phenylketonuria Clinical Trial 2023: PTC923 Highlights & Side Effects. Trial Name: NCT05166161 — Phase 3
PTC923 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05166161 — Phase 3

Frequently Asked Questions

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

Is PTC923 a danger to human health?

"PTC923 has been studied in Phase 3 clinical trials, meaning that there is some evidence of efficacy as well as multiple rounds of data supporting safety. Therefore, our team at Power has given it a safety rating of 3."

Answered by AI

How many research sites are currently conducting this experiment?

"The primary locations for this trial are University of Texas Health Science Center of Texas in Houston, Texas, Children's Medical Center Dallas in Dallas, Utah, and University of Utah, Division of Medical Genetics (pediatric and adult clinic) in Salt Lake City, Colorado. There are also 14 other potential sites for this trial."

Answered by AI

How many people will be given lenalidomide in this experiment?

"As this study requires 200 patients that meet very specific conditions, PTC Therapeutics will be conducting the trial from different locations including University of Texas Health Science Center of Texas in Houston, Texas and Children's Medical Center Dallas in Dallas, Utah."

Answered by AI

Who else is applying?

What site did they apply to?
University of Utah, Division of Medical Genetics (pediatric and adult clinic)
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
2022. "A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05166161.
All > Phenylketonuria
~51 spots leftby Feb 2025
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