Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria (NCT02579044) | Clinical Trial Compass
By InvitationPhase 1/2
Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria
United States80 participantsStarted 2015-12
Plain-language summary
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.
Who can participate
Age range
18 Months – 25 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 normal (ULN) for age or Glomerular filtration rate (GFR) \>70 mL/min/1.73m2
* bilirubin ≤ 1.5x upper limit of normal for age
* SGPT (ALT) \< and SGOT (AST) ≤ 2.5x normal range for age
* serum albumin greater than or equal to 2 g/dL
* PT/PTT: INR \<1.3 (or \<3 on anticoagulants)
* Fasting LDL cholesterol within 1.5x ULN per institutional guidelines (ie, \<195 mg/dL for 2 - 18 years of age, \<240 mg/dL for subjects \>18 years old)\* and Fasting serum cholesterol \<300 mg/dL (\<7.75 mmol/L)\* and Fasting triglycerides \<2.5 ULN (\<325 mg/dL for ages 2 - 18, \<400 for ages \>18)\*
\*may be re-evaluated for eligibility after initiation of lipid-lowering therapy
* Signed informed consent according to institutional guidelines must be obtained and subject must begin therapy within twenty-eight (28) days.
Exclusion Criteria:
* Subjects must not be taking medications that significantly affect…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria
Timeframe: 12 Months
2
Number and type of dose-limiting toxicities when everolimus and lonafarnib are administered in combination to children with progeria