Boston Children's Hospital
This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth "progeria"). The study will be conducted at a single clinical site utilizing the Clinical and Translational Study Unit (CTSU) at Boston Children's Hospital. Lonafarnib will be administered at doses previously established in the pediatric population and in this population of progeria subjects. This study will first determine the dose-limiting toxicities (DLT) and the maximum tolerated dose (MTD) of everolimus when administered in combination with lonafarnib. It will then determine the efficacy of everolimus when administered at its MTD in combination with lonafarnib for disease in progeria.
Inclusion Criteria: * Genetically-confirmed progeria. * display clinical signs of progeria as per the clinical trial team. * currently receiving lonafarnib under protocol 09-06-0298 * have not experienced a grade 3 or 4 toxicity within two months preceding enrollment * willing and able to come to Boston for appropriate studies and examinations. * no recent fractures or major surgery (within four weeks) * Absolute poly count (Absolute neutrophil count + bands + monocytes) \>1,000/uL * platelets \>75,000/uL (transfusion independent) * hemoglobin \>9 g/dL * creatinine ≤ 1.5 times upper limit of…
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