Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Status:
Active, not recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
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.
Phase:
Phase 2
Details
Lead Sponsor:
Boston Children's Hospital Boston Children’s Hospital