Everolimus + Lonafarnib for Progeria
Palo Alto (17 mi)Overseen byMonica Kleinman, M.D.
Age: < 65
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 1 & 2
Waitlist Available
Sponsor: Boston Children's Hospital
No Placebo Group
Breakthrough Therapy
Trial Summary
What is the purpose of this trial?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.
Eligibility Criteria
Treatment Details
1Treatment groups
Experimental Treatment
Group I: Everolimus and LonafarnibExperimental Treatment1 Intervention
Single arm. Phase I: Lonafarnib with escalating doses of everolimus to determine MTD Phase II: Lonafarnib plus everolimus at MTD (efficacy assessment)
Everolimus is already approved in United States, European Union, United States for the following indications:
🇺🇸 Approved in United States as Afinitor for:
- Advanced renal cell carcinoma
- Subependymal giant cell astrocytoma
- Progressive neuroendocrine tumors of pancreatic origin
- Advanced hormone receptor-positive, HER2-negative breast cancer
- Tuberous sclerosis complex-associated partial-onset seizures
🇪🇺 Approved in European Union as Votubia for:
- Subependymal giant cell astrocytoma
- Renal angiomyolipoma
- Tuberous sclerosis complex-associated partial-onset seizures
🇺🇸 Approved in United States as Zortress for:
- Prevention of organ rejection in kidney transplant patients
Find a clinic near you
Research locations nearbySelect from list below to view details:
Boston Children's HospitalBoston, MA
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Who is running the clinical trial?
Boston Children's HospitalLead Sponsor