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NCT03519711 · ClinicalTrials.gov registry record · Phase 1
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
A Phase 1 study of BH4 Deficiency and Hyperphenylalaninemia, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 8
- Enrollment target
- 4
- Study locations
NCT03519711: Completed Phase 1 study of BH4 Deficiency and Hyperphenylalaninemia, sponsored by PTC Therapeutics.
NCT03519711 is a Phase 1 study of BH4 Deficiency and Hyperphenylalaninemia that has completed, run by PTC Therapeutics. The registered enrollment target is 8 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (87% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03519711, a Phase 1 study of BH4 Deficiency and Hyperphenylalaninemia, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 8 participants
- Enrollment target
- 4
- Study locations
Study 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).
Primary Outcome
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs.
Conditions Studied
Interventions
- DRUG PTC923
Study Locations (4)
Minnesota
- University of Minnesota - Minneapolis
Texas
- UT Southwestern - Dallas
Utah
- University of Utah Hospital - Salt Lake City
Wisconsin
- Marshfield Clinic - Marshfield
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2019-01-03 |
| Est. Completion | 2020-10-02 |
| Phase | Phase 1 |
What the finished NCT03519711 record still lists
NCT03519711 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 8 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (87% lower).
The record links to 2 conditions, with BH4 Deficiency appearing as the primary indexed condition, and to 1 intervention - of which PTC923 is the first listed.
NCT03519711 reports a single indexed study location in Minnesota, Texas, Utah.
Frequently Asked Questions
What is clinical trial NCT03519711 about?
NCT03519711 is a clinical study titled "A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia". 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).
What is the current status of trial NCT03519711?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2019-01-03. Estimated completion is 2020-10-02.
What conditions does trial NCT03519711 study?
This clinical trial studies the following conditions: BH4 Deficiency, Hyperphenylalaninemia.
What interventions are being tested in trial NCT03519711?
The interventions under investigation include: PTC923 (DRUG).
Who is sponsoring clinical trial NCT03519711?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03519711 being conducted?
This trial has 4 study locations across Minnesota, Texas, Utah, Wisconsin. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03519711's enrollment target sits among peer trials
8 1959th of 2000 higher than 29 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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