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

Conditions

• Progeria

Eligibility Criteria

Sex

ALL

Age

18 Months - 25 Years

Healthy Volunteers

Not accepted

Interventions

• Everolimus and lonafarnib — DRUG

Study Details

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.

Key Dates

Start date

Dec 31, 2015

Status verified

Mar 2026

Primary completion

Mar 31, 2027

Completion

Mar 31, 2027

Study Design

Enrollment

80 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Other: Everolimus and Lonafarnib
  Single arm. Phase I: Lonafarnib with escalating doses of everolimus to determine MTD Phase II: Lonafarnib plus everolimus at MTD (efficacy assessment)

Primary Outcome Measure

Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria [ Time Frame: 12 Months ]

Locations (1)

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