drugset / Trial / NCT03570931
A Study to Assess Efficacy and Safety of RT001 in Subjects With Infantile Neuroaxonal Dystrophy
NaSingle-groupOpen-labelTreatment
Summary
The purpose of this study is to evaluate the efficacy and safety of RT001 in patients with Infantile Neuroaxonal Dystrophy (INAD).
Timeline
- Start
- 2018-11-05
- Primary completion
- 2020-08-09
- Completion
- 2022-06-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | RT001 | Protein / enzyme biologic | 3.84 g | Oral |