drugset / Trial / NCT05168774
FRDA Investigator Initiated Study (IIS) With Elamipretide
Phase 1/2
Completed
20 enrolled
Children's Hospital of Philadelphia
Friedreich's Ataxia Research Alliance · collabStealth BioTherapeutics Inc. · collab
RandomizedParallel-groupOpen-labelTreatment
Summary
To evaluate the safety, tolerability, and activity of Elamipretide in treating vision loss in Friedreich Ataxia (FRDA).
Timeline
- Start
- 2022-03-03
- Primary completion
- 2024-06-25
- Completion
- 2024-07-25
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Elamipretide | Peptide | 20 mg | Subcutaneous |
| Subject | Elamipretide | Peptide | 30 mg | Subcutaneous |
| Subject | Elamipretide | Peptide | 40 mg | Subcutaneous |
| Subject | Elamipretide | Peptide | 60 mg | Subcutaneous |