Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria
Status:
Enrolling by invitation
Trial end date:
2023-12-01
Target enrollment:
Participant gender:
Summary
This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in
Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth
"progeria"). The study will be conducted at a single clinical site utilizing the Clinical and
Translational Study Unit (CTSU) at Boston Children's Hospital. Lonafarnib will be
administered at doses previously established in the pediatric population and in this
population of progeria subjects. This study will first determine the dose-limiting toxicities
(DLT) and the maximum tolerated dose (MTD) of everolimus when administered in combination
with lonafarnib. It will then determine the efficacy of everolimus when administered at its
MTD in combination with lonafarnib for disease in progeria.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital