Clinical Trial: Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria

Brief 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.

Detailed Summary:
Sponsor: Boston Children’s Hospital

Current Primary Outcome:

  • Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria [ Time Frame: 12 Months ]
    For Phase I portion of protocol
  • Number and type of dose-limiting toxicities when everolimus and lonafarnib are administered in combination to children with progeria [ Time Frame: 12 Months ]
    For Phase I portion of protocol
  • Annual increase in weight gain [ Time Frame: 24 Months ]
    For Phase II portion of protocol
  • Change in pulse wave velocity (PWV) [ Time Frame: 24 months ]
    For Phase II portion of protocol


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Markers of progeria-specific activity [ Time Frame: 24 Months ]
    For Phase II portion of protocol
  • Trough levels of everolimus in combination with lonafarnib in progeria [ Time Frame: 12 months ]
    For Phase I portion of protocol


Original Secondary Outcome: Same as current

Information By: Boston Children’s Hospital

Dates:
Date Received: October 13, 2015
Date Started: December 2015
Date Completion:
Last Updated: December 23, 2015
Last Verified: December 2015