drugset / Trial / NCT06775041
Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
RandomizedSequentialOpen-labelTreatment
Summary
Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). Subjects in the randomized study arms will continue to take the standard of care (SOC), lonafarnib, and will be randomized to either take SOC alone or in combination with progerinin.
Timeline
- Start
- 2025-01-13
- Primary completion
- 2026-03
- Completion
- 2026-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Progerinin | Small molecule | 500 mg | Oral |
| Subject | Progerinin | Small molecule | 1500 mg | Oral |
| Background | Lonafarnib | Small molecule | — | — |