TrialPhase 2Completed
Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
- Sponsor
- PRG Science & Technology Co., Ltd. (Industry)
- Enrolment
- 10 enrolled
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Interventions
- Progerinin; Lonafarnib
- Ages
- children, adults, older adults
- Registry
- NCT06775041
From the registry
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.
Primary outcomes
- Change from baseline in plasma progerin concentration
- Incidence of Dose Limiting Toxicities (DLTs)
- Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
- Incidence of withdrawals due to Adverse Events (AEs)