New drug combo aims to slow Progeria's effects
NCT ID NCT02579044
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests whether adding everolimus to the existing drug lonafarnib can better control progeria, a rare disease that causes rapid aging in children. About 80 children with confirmed progeria will take both pills by mouth. The first part finds the safest dose of everolimus, and the second part checks if the combination improves weight gain and blood vessel health.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Dec 2015
- Expected to finish
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Mar 2027
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 months to 25 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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 the metabolism of lonafarnib * Subjects receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Subjects with endocrine deficiencies are allowed to receive physiologic or stress doses of steroids if necessary. Topical or inhaled steroids are allowed. * Subjects who have had a major surgery or significant traumatic injury within 4 weeks of start of study drug; have not recovered from the side effects of any major surgery (defined as requiring general anesthesia but excluding a procedure for insertion of central venous access); or who may require major surgery during the course of the study. * 13.2.5 Subjects with an active bleeding diathesis or on oral anti-vitamin K medication (except low dose coumadin). * Subjects who have any severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study such as: * known severely impaired lung function * active (acute or chronic) or uncontrolled severe infections. * liver disease such as cirrhosis, chronic active hepatitis or chronic persistent hepatitis. * History of hepatitis B or hepatitis C * Other concurrent severe and/or uncontrolled medical disease that could compromise participation in the study (i.e., uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration). * A known history of Human Immunodeficiency Virus (HIV) seropositivity or known immunodeficiency. * Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of everolimus (e.g., ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection). A nasogastric tube (NG tube) or gastric tube (G tube) is allowed. * Subjects who have known or suspected hypersensitivity to any of the excipients included in the formulation should not be treated. * Subjects must not be pregnant or breast-feeding. Female subjects of childbearing potential must have negative serum or urine pregnancy test. Sexually active male and female subjects of reproductive potential must agree to use a medically accepted form of birth control while on study and up to 10 weeks after treatment. It is permissible for female subjects to take oral contraceptives or other hormonal methods while receiving treatment with everolimus.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
The full official record for this study. This one lists no contact details, but it is the first place any would appear.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States