drugset / Trial / NCT03519711
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With Hyperphenylalaninemia
RandomizedParallel-groupOpen-labelTreatment
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).
Timeline
- Start
- 2019-01-03
- Primary completion
- 2020-10-02
- Completion
- 2020-10-02
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Sepiapterin | Small molecule | 2.5 mg/kg | Oral |
| Subject | Sepiapterin | Small molecule | 5 mg/kg | Oral |
| Subject | Sepiapterin | Small molecule | 10 mg/kg | Oral |
| Subject | Sepiapterin | Small molecule | 20 mg/kg | Oral |