Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria

PHASE2CompletedINTERVENTIONAL
Enrollment

29

Participants

Timeline

Start Date

May 31, 2007

Primary Completion Date

October 31, 2009

Study Completion Date

October 31, 2009

Conditions
ProgeriaHutchinson-Gilford Syndrome
Interventions
DRUG

Lonafarnib

Lonafarnib will be taken orally, twice per day, by all patients enrolled on this study. The drug is supplied to patients in capsule form, and for patients who are unable to swallow pills, the drug may be dissolved into solution. Every patient will start lonafarnib therapy at a dose of 115mg/kg. The study allows for patients to receive a dose escalation (up to 150mg/kg) if the drug is being well-tolerated. Every patient enrolled on this study will undergo two years of lonafarnib therapy.

Trial Locations (1)

02115

Children's Hospital Boston, Boston

Sponsors

Collaborators (1)

All Listed Sponsors
collaborator

Schering-Plough

INDUSTRY

lead

Monica E. Kleinman

OTHER