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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
Genetically-confirmed progeria
Display clinical signs of progeria as per the clinical trial team
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 months
Awards & highlights

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 below
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You have been diagnosed with progeria through genetic testing.
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You show signs of progeria according to the clinical trial team.
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You are currently taking lonafarnib as part of a specific medical study.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 more
Secondary outcome measures
Markers of progeria-specific activity
Trough levels of everolimus in combination with lonafarnib in progeria

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
756 Previous Clinical Trials
5,579,139 Total Patients Enrolled
2 Trials studying Progeria
90 Patients Enrolled for Progeria
Monica Kleinman, M.D.Principal InvestigatorBoston Children's Hospital

Media Library

Everolimus (mTOR inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT02579044 — Phase 1 & 2
Progeria Research Study Groups: Everolimus and Lonafarnib
Progeria Clinical Trial 2023: Everolimus Highlights & Side Effects. Trial Name: NCT02579044 — Phase 1 & 2
Everolimus (mTOR inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT02579044 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this research encompassing individuals over four decades old?

"This research is accepting volunteers that are between 18 months and 25 years of age."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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"

Answered by AI
~9 spots leftby Mar 2025