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Everolimus + Lonafarnib for Progeria
Study Summary
This trial is testing a combination of two drugs, everolimus and lonafarnib, to see if it is safe and effective in treating Hutchinson-Gilford Progeria Syndrome and other progeroid diseases.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowTimeline
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Trial Design
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Who is running the clinical trial?
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- You have a stomach or intestine problem that could affect how your body absorbs everolimus.You are allergic to any of the ingredients in the medicine.Your absolute poly count is higher than 1,000 per microliter.You are currently taking long-term medication that weakens your immune system.You have been diagnosed with progeria through genetic testing.You show signs of progeria according to the clinical trial team.You are currently taking lonafarnib as part of a specific medical study.You are taking medications that can greatly change how lonafarnib works in your body.You have a condition that makes you prone to bleeding, or you are taking a specific type of medication for blood thinning.You have HIV or a weakened immune system.Your platelet count is higher than 75,000 per microliter and you do not need blood transfusions to maintain this level.Your hemoglobin level is higher than 9 grams per deciliter.Your kidney function is within a certain range based on your age, or your glomerular filtration rate is above a specific level.Your bilirubin levels are within a certain range for your age.Your liver enzymes (SGPT and SGOT) are within the normal range for your age.Your blood albumin level is at least 2 g/dL.Your blood clotting tests show a normal result.Your LDL cholesterol level while fasting is higher than the normal range set by the hospital.Your cholesterol level when fasting is less than 300 mg/dL.Your fasting triglyceride levels are lower than 2.5 times the upper limit of normal.
- Group 1: Everolimus and Lonafarnib
- All Individual Drugs Already Approved - Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
- Approved for 10 Other Conditions - This treatment demonstrated efficacy for 10 other conditions.
- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
Is this research encompassing individuals over four decades old?
"This research is accepting volunteers that are between 18 months and 25 years of age."
Is this study currently seeking volunteers?
"This trial is not presently enrolling any further patients. It was first posted on December 1st 2015 and the latest update was September 1st 2022. If you are searching for other studies, right now there is one study seeking to recruit participants with progeria and 94 trials that are actively recruiting people undergoing this treatment."
What are the frequent applications of this therapeutic intervention?
"Rejection of a transplanted organ, particularly the liver, is commonly treated with this medication. Lenalidomide can also be utilized for kidney transplant rejection, waldenstrom macroglobulinemia and lung issues."
To what extent is enrollment being accepted for this clinical investigation?
"Sadly, this clinical trial is no longer open for recruitment. It was initially posted in December 2015 and its last update took place on September 1st 2022. Nevertheless, currently there are 95 trials related to progeria that are actively recruiting patients."
To whom is enrollment in this trial accessible?
"This clinical investigation is seeking 80 participants with progeria between 18 months and 25 years. Eligible candidates must have a verified genetic diagnosis, present the requisite symptoms for progeria, be taking lonafarnib under protocol 09-06-0298 without any Grade 3 or 4 toxicities in the past two months, not experienced major surgery within four weeks prior to enrollment, possess an Absolute poly count of more than 1,000/uL plus platelets greater than 75,000/uL (without transfusions), have hemoglobin levels over 9 g/dL as well as creatinine ≤ 1.5 times ULN or"
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