Status:
UNKNOWN
Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria
Lead Sponsor:
Boston Children's Hospital
Collaborating Sponsors:
Schering-Plough
Merck Sharp & Dohme LLC
Conditions:
Progeria
Eligibility:
All Genders
Phase:
PHASE2
Brief 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 mutat...
Eligibility Criteria
Inclusion
- Genetic Diagnosis: All patients must have confirmatory mutational analysis showing mutation in the lamin A gene.
- Clinical Diagnosis: Patients must display clinical signs of progeria as per the clinical trial team.
- Travel: Patients must be willing and able to come to Boston for appropriate studies and examinations at initiation of study and at months 6, 12, 18 and 24 on study.
- Patient must have adequate organ and marrow function as defined by the following parameters:
- Blood: APC (ANC + bands + monocytes = APC) \> 1,000/microliters, Platelets \> 75,000/microliters (transfusion independent); Hemoglobin \>9g/dl.
- Renal: creatinine Less than or equal 1.5 times normal for age or GFR \> 70 ml/min/1.73m2.
- Hepatic: bilirubin Less than or equal to 1.5 x upper limit of normal for age; SGPT (ALT) \< and SGOT (AST) \< 5 x normal range for age.
- PT/PTT: PT/PTT \< 120% upper limit of normal OR PI approval
- No overt renal, hepatic, pulmonary disease or immune dysfunction.
- 25-hydroxyvitamin D ≥ 20 ng/ml within 4 weeks of bisphosphonate infusion.
- Signed informed consent according to institutional guidelines must be obtained and patient must begin therapy within twenty eight (28) days.
Exclusion
- Other than the drugs used in this protocol, 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 pregnant or 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 :
August 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2023
Estimated Enrollment :
85 Patients enrolled
Trial Details
Trial ID
NCT00916747
Start Date
August 1 2009
End Date
December 1 2023
Last Update
February 27 2023
Active Locations (1)
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1
Children's Hospital Boston
Boston, Massachusetts, United States, 02115