At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 8 enrolled
Drug / intervention
PTC923drug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 1/2, Open-Label, Randomized Parallel Arm, Intra-patient Dose Escalation Study to Evaluate the Safety, Pharmacokinetics and Preliminary Efficacy of CNSA-001(Sepiapterin) in Primary Tetrahydrobiopterin Deficient Patients With Hyperphenylalaninemia
In Brief
A Phase 2 clinical trial evaluating PTC923 for BH4 Deficiency and Hyperphenylalaninemia. Completed, enrolled 8 participants across 4 sites.
Detailed Summary
This study has been designed to demonstrate the safety, pharmacokinetics (PK) and preliminary efficacy of PTC923 (CNSA-001) in reducing blood phenylalanine concentrations in participants with hyperphenylalaninemia due to primary BH4 deficiency (PBD).
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsBH4 Deficiency, Hyperphenylalaninemia
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
20192020202120222023202420252026
First PostedMay 2018
Enrollment StartJan 2019
Primary CompletionOct 2020
TodayJul 2026
First PostedMay 9, 2018
Enrollment StartJan 3, 2019
Primary CompletionOct 2, 2020
TodayJul 2, 2026
Enrollment to primary: 1.7 yearsPosted 8.1 years ago
Interventions
PTC923drug
PTC923 will be administered per dose and schedule specified in arm description.