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

NCT02579044 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 80

Last updated 2026-04-02

No results posted yet for this study

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.

Conditions

  • Progeria

Interventions

DRUG

Everolimus and lonafarnib

Sponsors & Collaborators

Principal Investigators

  • Monica Kleinman, M.D. · Boston Children's Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Months
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-12-31
Primary Completion
2027-03-31
Completion
2027-03-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02579044 on ClinicalTrials.gov