Treatment for Progeria

Phase-Based Progress Estimates
< 65
All Sexes
What conditions do you have?

Study Summary

This trial is testing a combination of two drugs, everolimus and lonafarnib, to see if it is safe and effective in treating Hutchinson-Gilford Progeria Syndrome and other progeroid diseases.

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

4 Primary · 2 Secondary · Reporting Duration: 24 Months

12 Months
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria
Number and type of dose-limiting toxicities when everolimus and lonafarnib are administered in combination to children with progeria
12 months
Trough levels of everolimus in combination with lonafarnib in progeria
24 Months
Annual increase in weight gain
Markers of progeria-specific activity
24 months
Change in pulse wave velocity (PWV)

Trial Safety

Safety Progress

1 of 3

Trial Design

0 Treatment Group

80 Total Participants · 0 Treatment Group

Primary Treatment: Treatment · No Placebo Group · Phase 1 & 2

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 24 months

Who is running the clinical trial?

Boston Children's HospitalLead Sponsor
710 Previous Clinical Trials
5,324,093 Total Patients Enrolled
2 Trials studying Progeria
90 Patients Enrolled for Progeria
Monica Kleinman, M.D.Principal InvestigatorBoston Children's Hospital

Eligibility Criteria

Age < 65 · All Participants · 10 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have not experienced a grade 3 or 4 toxicity within two months preceding enrollment.
The absolute neutrophil count (ANC) is greater than 1,000/uL.
You have platelets of greater than 75,000/uL.
You have a creatinine level below the upper limit of normal.
You are willing and able to come to Boston for appropriate studies and examinations.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 29th, 2021

Last Reviewed: November 22nd, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.