Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Boston Children's Hospital
With
Schering-Plough
Merck Sharp & Dohme LLC
Eiger BioPharmaceuticals
Age
Any age
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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:
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.
You may not be if
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.
