ProgeriaPhase 1/2NCT02579044
Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria
A Phase 1/2 study testing Everolimus and lonafarnib in people with Progeria, run by Boston Children's Hospital at 1 site in United States.
Trial summary
This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth "progeria"). Read more about this study
- Who is it for?
- Ages 18 months to 25 years
- Conditions the study is for
- Progeria
- What is being tested?
- Everolimus and lonafarnib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2015to Mar 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
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- 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.
Who cannot join
- 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
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth "progeria"). The study will be conducted at a single clinical site utilizing the Clinical and Translational Study Unit (CTSU) at Boston Children's Hospital. Lonafarnib will be administered at doses previously established in the pediatric population and in this population of progeria subjects. This study will first determine the dose-limiting toxicities (DLT) and the maximum tolerated dose (MTD) of everolimus when administered in combination with lonafarnib. It will then determine the efficacy of everolimus when administered at its MTD in combination with lonafarnib for disease in progeria.
- Conditions
- Progeria
- Being tested
- Everolimus and lonafarnib
- Medical subject headings
- Progeria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Everolimus and lonafarnibDrug
| Study group | Role | What they receive |
|---|---|---|
| Everolimus and Lonafarnib | Other | Single arm. Phase I: Lonafarnib with escalating doses of everolimus to determine MTD Phase II: Lonafarnib plus everolimus at MTD (efficacy assessment) Receives: Drug: Everolimus and lonafarnib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria
For Phase I portion of protocol
Measured: 12 Months
Number and type of dose-limiting toxicities when everolimus and lonafarnib are administered in combination to children with progeria
For Phase I portion of protocol
Measured: 12 Months
Annual increase in weight gain
For Phase II portion of protocol
Measured: 24 Months
Change in pulse wave velocity (PWV)
For Phase II portion of protocol
Measured: 24 months
Secondary outcomes (2)
Markers of progeria-specific activity
For Phase II portion of protocol
Measured: 24 Months
Trough levels of everolimus in combination with lonafarnib in progeria
For Phase I portion of protocol
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 2015 (estimated)
- Study startDec 2015
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ProgeriaStudies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Monica E. Kleinman, Boston Children's Hospital
- Investigators
- Monica Kleinman, M.D., Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02579044
- Sponsor's protocol ID
- P00017505
- Status last verified
- Mar 2026
- First submitted
- 13 Oct 2015
- Last updated
- 2 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase I/II Trial of Everolimus in Combination With Lonafarnib in Progeria
- ClinicalTrials.gov ID
- NCT02579044
- Acronym / sponsor ID
- P00017505
- Conditions
- Progeria
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Study start
- Dec 2015 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 19 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase I/II dose-escalation trial of everolimus in combination with lonafarnib in Hutchinson-Gilford Progeria Syndrome (HGPS) and progeroid laminopathies (henceforth "progeria"). The study will be conducted at a single clinical site utilizing the Clinical and Translational Study Unit (CTSU) at Boston Children's Hospital. Lonafarnib will be administered at doses previously established in the pediatric population and in this population of progeria subjects. This study will first determine the dose-limiting toxicities (DLT) and the maximum tolerated dose (MTD) of everolimus when administered in combination with lonafarnib. It will then determine the efficacy of everolimus when administered at its MTD in combination with lonafarnib for disease in progeria.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Everolimus and Lonafarnib | Other | Drug: Everolimus and lonafarnibSingle arm. Phase I: Lonafarnib with escalating doses of everolimus to determine MTD Phase II: Lonafarnib plus everolimus at MTD (efficacy assessment) |
Who may qualify
- Ages
- 18 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (17 listed)
- 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.
Key exclusion criteria (14 listed)
- 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:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Everolimus and lonafarnib | Drug | Everolimus and Lonafarnib |
Primary outcome
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria
Time frame: 12 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 progeria
Time frame: 12 Months
For Phase I portion of protocol
Annual increase in weight gain
Time frame: 24 Months
For Phase II portion of protocol
Change in pulse wave velocity (PWV)
Time frame: 24 months
For Phase II portion of protocol
2 secondary outcomes
Markers of progeria-specific activity
Time frame: 24 Months
Trough levels of everolimus in combination with lonafarnib in progeria
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2026.
Related trials for Progeria
Not yet recruitingObservational study
A Study Observing Disease Progression in a Patient With Hutchinson-Gilford Progeria Syndrome (HGPS)
Hutchinson-Gilford Progeria Syndrome
Not yet recruitingObservational study
Creating Stem Cells From a Progeria Patient's Blood to Test Candidate Treatments in the Lab
Hutchinson-Gilford Progeria Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Monica Kleinman, M.D. principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Everolimus and lonafarnib in people with Progeria, run by Boston Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 2 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum-tolerated dose (MTD) of everolimus when administered orally in combination with lonafarnib in subjects with progeria (time frame: 12 Months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Apr 2026.