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A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia

NCT03519711 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 8

Last updated 2023-11-14

Study results available
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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

  • BH4 Deficiency
  • Hyperphenylalaninemia

Interventions

DRUG

PTC923

PTC923 will be administered per dose and schedule specified in arm description.

Sponsors & Collaborators

Principal Investigators

  • Neil Smith, PharmD · Censa Pharmaceuticals

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-01-03
Primary Completion
2020-10-02
Completion
2020-10-02
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03519711 on ClinicalTrials.gov