Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria

Progeria Clinical Trial

Official title: An Open Label Phase II Trial of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome(HGPS) and Progeroid Laminopathies

Status Active, not recruiting

Clinical Trial Summary

Hutchinson-Gilford Progeria Syndrome (Progeria) is a rare autosomal disease that results in premature death at a median age of 13 years due to cardiovascular and cerebralvascular compromise. The mutation for this disease has been identified and results in a mutant form of lamin A that cannot be de-farnesylated. This study evaluates the combination of pravastain (a statin), lonafarnib (a farnesyltransferase inhibitor) and zoledronic acid (a bisphosphonate) in an open label phase II efficacy trial in children with Progeria. These agents all target farnesylation pathways at different points. Patients with genetically confirmed progeria will be eligible for this protocol. Treatment will be initiated for 24 months duration. Clinical and biologic parameters will be examined to assess response.

Clinical Trial Description

n/a

Related Conditions & MeSH terms

• Progeria

• NCT number: NCT00916747
• Study type: Interventional
• Source: Boston Children's Hospital
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: August 2009
• Completion date: December 2023