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Clinical Trial 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.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT02579044
Study type Interventional
Source Boston Children's Hospital
Contact
Status Enrolling by invitation
Phase Phase 1/Phase 2
Start date December 2015
Completion date December 2023

See also
  Status Clinical Trial Phase
Completed NCT00094393 - Clinical Studies of Progeria
Completed NCT00425607 - Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria Phase 2
Completed NCT00879034 - A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria Phase 2
Active, not recruiting NCT00916747 - Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria Phase 2
Approved for marketing NCT03895528 - Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy