NCT04512963

Phase I Study of Progerinin in Healthy Volunteers

Completed Phase 1 Last updated 22 September 2021

What this trial tests

Phase 1 trial testing Progerinin in Hutchinson-Gilford Progeria Syndrome in 64 participants. Completed in 27 July 2021.

Timeline

24 August 2020

Primary endpoint
27 July 2021

27 July 2021

Quick facts

Lead sponsor	PRG Science & Technology Co., Ltd.
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	quadruple
Primary purpose	treatment
Enrollment	64
Start date	24 August 2020
Primary completion	27 July 2021
Estimated completion	27 July 2021
Sites	1 location across United States

Drugs / interventions tested

• Progerinin — full drug profile →
• Placebo

Conditions studied

• Hutchinson-Gilford Progeria Syndrome — all drugs for Hutchinson-Gilford Progeria Syndrome →
• Werner Syndrome — all drugs for Werner Syndrome →

Sponsor

PRG Science & Technology Co., Ltd. — full company profile →

Who can join

Adults 18 to 45, any sex, with Hutchinson-Gilford Progeria Syndrome or Werner Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

PRG-PRO-001 is a Phase I, Randomized, Double-blind, Placebo-Controlled, Single Ascending Dose (SAD) Study including a food interaction study, followed by a Multiple Ascending Dose (MAD) Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers. This is a first-in-human study. The study aims to determine the safety and tolerability of Progerinin after single and multiple doses in healthy volunteers and to evaluate the pharmacokinetics (PK) of Progerinin after single and multiple dose administrations in healthy volunteers.

Publications & conference data

5 peer-reviewed publications reference this trial (live from Europe PMC):

1. Small-Molecule Therapeutic Perspectives for the Treatment of Progeria.
   Macicior J, Marcos-Ramiro B, Ortega-Gutiérrez S. · · 2021 · cited 15× · PMID 34281245 · DOI 10.3390/ijms22137190
2. Progerin, an Aberrant Spliced Form of Lamin A, Is a Potential Therapeutic Target for HGPS.
   Kim BH, Chung YH, Woo TG, Kang SM, et al · · 2023 · cited 7× · PMID 37759521 · DOI 10.3390/cells12182299
3. Splicing Variants, Protein-Protein Interactions, and Drug Targeting in Hutchinson-Gilford Progeria Syndrome and Small Cell Lung Cancer.
   Kim BH, Woo TG, Kang SM, Park S, et al · · 2022 · cited 5× · PMID 35205210 · DOI 10.3390/genes13020165
4. Lipodystrophic Laminopathies: From Dunnigan Disease to Progeroid Syndromes.
   Díaz-López EJ, Sánchez-Iglesias S, Castro AI, Cobelo-Gómez S, et al · · 2024 · cited 1× · PMID 39273270 · DOI 10.3390/ijms25179324
5. The pursuit of therapy for progeria.
   Saxena S, Shukla D. · · 2021 · cited 1× · PMID 34176790 · DOI 10.18632/aging.203254