Werner Syndrome Clinical Trial
Official title:
A Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin for the Treatment of Bone Mineral Density (BMD) Loss in Subjects With Typical Werner Syndrome
This is a Phase 2, open-label study to A Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin for the Treatment of Bone Mineral Density (BMD) Loss in Subjects with Typical Werner Syndrome. There will be up to 5 subjects that will receive treatment with Progerinin twice daily for approximately 1 year.
Subjects who have successfully completed the Screening phase will enter the baseline and treatment phase of the study. Subjects will take the assigned treatment of Progerinin (2400 mg) split as 1200 mg BID in the morning and evening with food for 1-Year. To evaluate the safety and tolerability of Progerinin for the treatment of BMD loss in subjects with typical Werner syndrome. The secondary objectives of this study are: - To characterize the activity of Progerinin on bone metabolism in subjects with typical Werner syndrome. - To evaluate the pharmacokinetics (PK) of Progerinin sachet administered twice daily (BID) in subjects with typical Werner syndrome ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT04512963 -
Phase I Study of Progerinin in Healthy Volunteers
|
Phase 1 |