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

NCT00425607 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 29

Last updated 2019-06-25

Study results available
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Summary

This is an open label dose adjusted phase II trial of the oral farnesyltransferase inhibitor (FTI) lonafarnib (SCH66336) for patients with HGPS and progeroid laminopathies.

Conditions

  • Progeria
  • Hutchinson-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.

Sponsors & Collaborators

  • Schering-Plough

    collaborator INDUSTRY

  • Monica E. Kleinman

    lead OTHER

Principal Investigators

  • Mark W Kieran, MD, PhD · Dana-Farber Cancer Institute, Children's Hospital Boston

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-05-31
Primary Completion
2009-10-31
Completion
2009-10-31

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00425607 on ClinicalTrials.gov