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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 is a Phase 1 study of BH4 Deficiency and Hyperphenylalaninemia that has completed, run by PTC Therapeutics. The registered enrollment target is 8 participants. The trial reports 4 study locations across 4 states.
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).
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03519711
The ClinicalTrials.gov registry entry for NCT03519711 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 8 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
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 4 study locations spanning 4 distinct geographic areas - top geographies include 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.
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