Status:
COMPLETED
A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Lead Sponsor:
Boston Children's Hospital
Collaborating Sponsors:
Dana-Farber Cancer Institute
Brigham and Women's Hospital
Conditions:
Progeria
Hutchinson-Gilford Syndrome
Eligibility:
All Genders
Phase:
PHASE2
Brief Summary
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 sch...
Detailed Description
Progerias are rare "premature aging" diseases in which children die of severe atherosclerosis leading to strokes and heart attacks. It is a multisystem disease with objective clinical markers for dise...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
March 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 1 2009
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT00879034
Start Date
March 1 2009
End Date
April 1 2009
Last Update
June 13 2019
Active Locations (1)
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1
Children's Hospital Boston
Boston, Massachusetts, United States, 02115