A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Status:
Completed
Trial end date:
2009-04-01
Target enrollment:
Participant gender:
Summary
This is an open label single arm feasibility trial. A combination of two oral agents
(pravastatin and lonafarnib) and one intravenous (IV) agent (zoledronic acid) will be
administered at doses and schedule currently applied in pediatrics. These agents all target
farnesylation pathways at different points. Our goal is to inhibit farnesylation of abnormal
lamin, the disease-causing protein in Hutchinson-Gilford Progeria Syndrome and progeroid
laminopathies (henceforth "progeria"). The drugs will include the intravenous bisphosphonate
zoledronic acid, oral HMG co-reductase inhibitor pravastatin and the oral farnesyltransferase
inhibitor (FTI) lonafarnib (SCH 66336). Patients with genetically confirmed progeria will be
eligible for this protocol. Treatment will be initiated for 4 weeks duration and may be
extended depending on tolerability. This study will assess the feasibility of this treatment
regimen in the first 4 weeks. If tolerated for 4 weeks, patients can be treated with this
regimen for up to 6 months.
Phase:
Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital
Collaborators:
Brigham and Women's Hospital Dana-Farber Cancer Institute Schering-Plough