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A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Study details:
Eligible participants are:. Feeder participants: those who have completed a Phase 3 PTC Therapeutics (PTC) sponsored feeder study (including Study PTC923-MD-003-PKU). Non-feeder controlled participants: those who have not completed a feeder study and have blood Phe levels \<360 μmol/L at study entry.
Non-feeder uncontrolled participants: those who have not completed a feeder study and have blood Phe levels ≥360 μmol/L at study entry.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 0 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2022-02-14
Primary completion: 2026-04-30
Study completion finish: 2026-04-30
Study type
TREATMENT
Phase
PHASE3
Trial ID
NCT05166161
Intervention or treatment
DRUG: PTC923
Conditions
- • Phenylketonuria
Find a site
Closest Location:
Westmead Hospital
Research sites nearby
Select from list below to view details:
Westmead Hospital
Westmead, New South Wales, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
PARC Clinical Research
Adelaide, South Australia, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: PTC923
| DRUG: PTC923
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Number of Treatment-Emergent Adverse Events (TEAEs) | A TEAE is any unfavorable or unintended sign (including an abnormal laboratory finding), symptom or disease in a study participant who is administered study drug in this study | Baseline up to end of study (up to approximately 2.5 years) |
Change From Baseline in Dietary Phe/Protein Consumption at Week 26, Measured During Phe Tolerance Assessment Period | Phe tolerance is defined as the total amount of dietary Phe (milligrams \[mg\]/kilogram \[kg\] per day) ingested while maintaining blood Phe levels within the range of 40 to 360 micromoles (μmol)/liter (L) (defined as ≥40 to \<360 μmol/L). | Baseline, Week 26 |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
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 | QOL using PKU-QOL questionnaire will be assessed in the subset of participants who are able to complete the PKU-QOL (that is, participants whose primary language is English \[British or American\], Turkish, Dutch, German, Spanish, Italian, Portuguese, or French) (ages 6 to 8 years Parent PKU-QOL; ages 9 to 11 years Child PKU-QOL; ages 12 to 17 years Adolescent PKU-QOL; ages ≥18 years Adult PKU-QOL). | Baseline, Months 8, 14, 20, 26, 32, and 38 |
Change From Baseline in QOL Using the European Quality of Life - 5 Dimensions (EQ-5D) at Months 8, 14, 20, 26, 32, and 38 | QOL will be assessed using the EQ-5D (EQ-5D-Y Proxy Version 1 \[3 to 7 years\]; EQ-5D-Y \[8 to 15 years\]; EQ-5D-5L (\[≥16 years\]). | Baseline, Months 8, 14, 20, 26, 32, and 38 |
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