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New drug combo aims to slow Progeria's effects

NCT ID NCT02579044

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 80 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2015

Expected to finish

Mar 2027

An estimate. End dates often move.

Lead sponsor

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

18 months to 25 years

Sex

Anyone

Healthy volunteers

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

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.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. 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.

    Open the record ↗

  3. 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

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States