Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients with Hutchinson-Gilford Progeria Syndrome (HGPS)

PHASE2RecruitingINTERVENTIONAL
Enrollment

16

Participants

Timeline

Start Date

January 31, 2025

Primary Completion Date

December 31, 2025

Study Completion Date

December 31, 2025

Conditions
Hutchinson-Gilford Progeria Syndrome
Interventions
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.

Trial Locations (1)

02115

RECRUITING

Boston Children's Hospital, Boston

All Listed Sponsors
lead

PRG Science & Technology Co., Ltd.

INDUSTRY