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NCT02579044

Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria

Enrolling by Invitation
PHASE1Ages 18–25InterventionalTreatment
Boston Children's Hospital
~80 participants
Updated 2026-04-02 on ClinicalTrials.gov
What's tested:Everolimus and lonafarnib

At a glance

Recruiting sites
0 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria
Measured over 12 Months
+3 more outcomes measured

Conditions

Progeria

Explore related trials

ProgeriaLMNA Gene

Where it's being run

1 sites across 1 states
Massachusetts1

Study leadership

  • Monica Kleinman, M.D. · PRINCIPAL_INVESTIGATOR · Boston Children's Hospital

Who to contact

This trial hasn't published a contact. View it on ClinicalTrials.gov

Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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)\*
Signed informed consent according to institutional guidelines must be obtained and subject must begin therapy within twenty-eight (28) days.

Exclusion

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.

What this trial measures

  • Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria12 Months

    For Phase I portion of protocol

  • Number and type of dose-limiting toxicities when everolimus and lonafarnib are administered in combination to children with progeria12 Months

    For Phase I portion of protocol

  • Annual increase in weight gain24 Months

    For Phase II portion of protocol

  • Change in pulse wave velocity (PWV)24 months

    For Phase II portion of protocol