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A Phase II Pilot Study of Zoledronic Acid, Pravastatin, and Lonafarnib (SCH66336) for Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) and Progeroid Laminopathies

Phase 2
Not Enrolling

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Trial Information

A Phase II Pilot Study of Zoledronic Acid, Pravastatin, and Lonafarnib (SCH66336) for Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) and Progeroid Laminopathies

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.

Inclusion Criteria:

- Genetic Diagnosis: All patients must have confirmatory mutational analysis showing
mutation in the lamin A gene.

- Patients must display clinical signs of progeria as per the clinical trial team.

- Patients must be willing and able to come to Boston for appropriate studies and
examinations at initiation of study and at week 4 of study.

- Patient must have adequate organ and marrow function as defined by study parameters

Exclusion Criteria:

- Other than the drugs used in this protocol, other drugs targeted to treat Progeria
are excluded. Drugs to treat symptoms of Progeria are permitted.

- Patients must not be taking medications that significantly affect the metabolism of
lonafarnib at the time they start lonafarnib.

- Patient must have no uncontrolled infection.

- Subjects who have known or suspected hypersensitivity to any of the excipients
included in the formulation should not be treated.

- Patients must not be pregnancy of breast-feeding. Female patients of childbearing
potential must have negative serum or urine pregnancy test. Male and female patients
of reproductive potential must agree to use a medically accepted form of birth
control while on study and up to 10 weeks after treatment. It is permissible for
female patients to take oral contraceptives or other hormonal methods while receiving
treatment with lonafarnib.

Type of Study:


Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

The primary objective of this study is to evaluate the feasibility of administering intravenous zoledronic acid, oral pravastatin and oral lonafarnib, to patients with Progeria for a minimum of 4 weeks

Outcome Time Frame:

4 weeks

Safety Issue:



United States: Food and Drug Administration

Study ID:




Start Date:

March 2009

Completion Date:

April 2009

Related Keywords:

  • Progeria
  • Progeria



Children's Hospital BostonBoston, Massachusetts  02115