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

NCT06775041 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2026-02-09

No results posted yet for this study

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.

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.

Sponsors & Collaborators

  • PRG Science & Technology Co., Ltd.

    lead INDUSTRY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-01-13
Primary Completion
2026-03-31
Completion
2026-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06775041 on ClinicalTrials.gov