A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
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ClinicalTrials.gov Identifier: NCT05166161 |
Recruitment Status :
Recruiting
First Posted : December 21, 2021
Last Update Posted : April 9, 2024
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Tracking Information | |||||
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First Submitted Date ICMJE | December 8, 2021 | ||||
First Posted Date ICMJE | December 21, 2021 | ||||
Last Update Posted Date | April 9, 2024 | ||||
Actual Study Start Date ICMJE | February 14, 2022 | ||||
Estimated Primary Completion Date | February 7, 2025 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE |
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Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria | ||||
Official Title ICMJE | A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria | ||||
Brief Summary | 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. | ||||
Detailed Description | 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. |
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Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 3 | ||||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Phenylketonuria | ||||
Intervention ICMJE | Drug: PTC923
PTC923 powder for oral use will be suspended in water or apple juice prior to administration.
Other Name: Sepiapterin
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Study Arms ICMJE | Experimental: PTC923
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.
Intervention: Drug: PTC923
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Recruiting | ||||
Estimated Enrollment ICMJE |
200 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Estimated Study Completion Date ICMJE | February 7, 2025 | ||||
Estimated Primary Completion Date | February 7, 2025 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
Additional criteria for non-feeder participants who did not participate in a feeder study:
Confirmed diagnosis of a primary BH4 deficiency as evidenced by biallelic pathogenic mutations in 6-pyruvoyltetrahydropterin synthase, recessive GTP cyclohydrolase I, sepiapterin reductase, quinoid dihydropteridine reductase, or pterin-4-alphacarbinolamine dehydratase genes. |
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Sex/Gender ICMJE |
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Ages ICMJE | Child, Adult, Older Adult | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE |
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Listed Location Countries ICMJE | Australia, Brazil, Canada, Denmark, Georgia, Germany, Italy, Japan, Mexico, Netherlands, Portugal, Spain, Turkey, United Kingdom, United States | ||||
Removed Location Countries | France | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT05166161 | ||||
Other Study ID Numbers ICMJE | PTC923-MD-004-PKU 2021-000497-28 ( EudraCT Number ) |
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Has Data Monitoring Committee | Not Provided | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | PTC Therapeutics | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | PTC Therapeutics | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | PTC Therapeutics | ||||
Verification Date | April 2024 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |