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mTOR inhibitor
Everolimus + Lonafarnib for Progeria
Phase 1 & 2
Waitlist Available
Led By Monica Kleinman, M.D.
Research Sponsored by Boston Children's Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights
All Individual Drugs Already Approved
Approved for 10 Other Conditions
No Placebo-Only Group
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.
Eligible Conditions
- Progeria
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 months
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Annual increase in weight gain
Change in pulse wave velocity (PWV)
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria
+1 moreSecondary study objectives
Markers of progeria-specific activity
Trough levels of everolimus in combination with lonafarnib in progeria
Awards & Highlights
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.
Trial Design
1Treatment groups
Experimental Treatment
Group I: Everolimus and LonafarnibExperimental Treatment1 Intervention
Single arm. Phase I: Lonafarnib with escalating doses of everolimus to determine MTD Phase II: Lonafarnib plus everolimus at MTD (efficacy assessment)
Find a Location
Who is running the clinical trial?
Boston Children's HospitalLead Sponsor
783 Previous Clinical Trials
5,581,201 Total Patients Enrolled
2 Trials studying Progeria
90 Patients Enrolled for Progeria
Monica Kleinman, M.D.Principal InvestigatorBoston Children's Hospital
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- 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.
Research Study Groups:
This trial has the following groups:- Group 1: Everolimus and Lonafarnib
Awards:
This trial has 3 awards, including:- 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.
Timeline:
This trial has the following timeline:- 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.
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