NCT06775041

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)

• Status: Active Not Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: PRG Science & Technology Co., Ltd. · Industry
• Sex: All
• Age: 1 Year
• Healthy volunteers: Not accepted

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.

Detailed description

During this trial, a total of 10 subjects will be randomized in a 4:1 ratio to receive treatment with progerinin plus lonafarnib vs lonafarnib alone. Dose escalation will occur through intra-subject dose titration. Two ascending doses of progerinin will be assessed. The dose levels proposed in this study were determined using body weight based on adult doses assessed in previously completed studies, and on a simulated population pharmacokinetic (PK) model. If systemic exposures of Progerinin in pediatric patients are lower than those in adults, a dose escalation may be added at the end of the study. Progerinin will be administered, with food. The study treatment is available in sachets of 250 mg or 350 mg and the doses in the study range from 500 to 1500 mg daily, dosed BID (twice a day). Lonafarnib is considered as the standard of care (SOC) and all subjects in the randomized study arms will continue taking lonafarnib for the duration of the study. Subjects who are not already taking lonafarnib will also have the opportunity to initiate lonafarnib on this study as part of the lonafarnib naïve arm (separate from the randomized study arms).

Conditions

• Hutchinson-Gilford Progeria Syndrome

Interventions

Type	Name	Description
DRUG	Progerinin	Dosing will be determined based on body weight. Dosing ranges from 500mg to 1500mg daily. Progerinin is available in sachets of 250 mg or 350 mg for oral use.
DRUG	Lonafarnib	Lonafarnib is considered as the standard of care (SOC). Subjects in the randomized arms will continue taking lonafarnib per the package insert. Subjects in the lonafarnib naïve arm will initiate lonafarnib as per FDA-approved study 07-01-7505.

Timeline

• Start date: 2025-01-13
• Primary completion: 2026-03-01
• Completion: 2026-03-01
• First posted: 2025-01-14
• Last updated: 2026-02-09

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study