Hutchinson-Gilford Progeria SyndromePhase 2NCT06775041
Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
A Phase 2 study testing Progerinin and Lonafarnib in people with Hutchinson-Gilford Progeria Syndrome, run by PRG Science & Technology Co., Ltd. at 1 site in United States.
Trial summary
Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Hutchinson-Gilford Progeria Syndrome
- What is being tested?
- Progerinin and Lonafarnib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Jan 2025to 30 Apr 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subjects will be eligible for enrollment in the study only if they meet ALL the following criteria at time of Screening:
- Subjects ≥ 1 year of age and weight ≥ 17.6 lb (8 kg).
- Subject must have confirmatory mutational analysis showing the classic HGPS mutation (c. 1824 C>T), nonclassic HGPS, Zmpset24 gene mutation, or other LMNA mutation (all subjects will havedocumentation of genetic testing prior to enrollment). *Only subjects with progerin producing mutations will be eligible for randomization. Other PL are eligible to enroll as naïve subjects (see note for criterion #5).
- Subject must display clinical signs of Progeria as per the clinical trial team.
- Subject must be willing and able to come to Boston for appropriate assessments and examinations.
- Subject must be taking lonafarnib for at least 4 months prior to enrollment and have no history of grade 3 or 4 side effects that can be probably or possibly attributed to lonafarnib for at least 2 months prior to enrollment.
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- Note: Subjects with no prior treatment (i.e., no commercial or managed access program source) with lonafarnib may be enrolled to initiate treatment with lonafarnib monotherapy. These subjects will initiate lonafarnib therapy in preparation for an upcoming phase of the study following the completion of phase 2a. These subjects will be excluded from the analysis of this phase 2a study. These subjects must be at least 12 months of age per lonafarnib package insert.
- Subjects must have had no recent fractures or major surgery (within four weeks).
- Subject must have adequate organ and marrow function as defined by the following parameters:
- Blood: APC (ANC + bands + monocytes) >1,000/µL, platelets >75,000/µL (transfusion independent); hemoglobin >9 g/dL.
- Renal: creatinine ≤ 1.5 times normal for age or Glomerular Filtration Rate (GFR) >70 mL/min/1.73m2.
- Hepatic: bilirubin ≤1.5x upper limit of normal for age; Serum Glutamic Pyruvic Transaminase (SGPT) (Alanine Transaminase, [ALT]) < and Serum Glutamic-Oxaloacetic Transaminase (SGOT) (Aspartate Aminotransferase, [AST]) ≤ 2.5x normal range for age.
- The subject is willing to provide written informed assent form, when possible, to participate in the study after reading the informed assent form and the information provided and has had the opportunity to discuss the study with the investigator or designee. Additionally, the subject's legally authorized guardian (LAR) is willing to provide written informed consent.
- The subject is able to communicate satisfactorily with the investigator and to participate in, and comply with, the requirements of the study.
- The subject (or LAR) is able to understand the nature of the study and any potential hazards associated with participating in it.
- Negative pregnancy test for female subjects of childbearing potential and those who have not been surgically sterilized or who do not have verbal or laboratory confirmation of two years postmenopausal status. Women of childbearing potential (WOCBP) and Women of non-childbearing potential are eligible to participate. Women of childbearing potential should use an acceptable method of birth control and agree to continue to use this method for the duration of the study and for 90 days after taking the last dose of Progerinin.
- Acceptable methods of contraception include abstinence, female subject/partner's use of hormonal contraceptive (oral, implanted, or injected) in conjunction with a barrier method (WOCBP only) (e.g., diaphragm, cervical cap, male condom, and female condom and spermicidal foam, sponges, and film), female subject/partner's use of an intrauterine device (IUD), or if the female subject/partner is surgically sterile (e.g., bilateral tubal ligation, hysterectomy) or two years postmenopausal at time of screening. All male subjects/partners (excluding men who have been sterilized) must agree to consistently and correctly use a condom for the duration of the study and for 90 days after taking the study drug. In addition, subjects may not donate sperm for the duration of the study and for 90 days after taking study drug.
Who cannot join
- Subjects meeting ANY of the following criteria at time of Screening will be excluded from enrollment:
- Other than the drugs used in this protocol, other drugs targeted to treat Progeria are excluded. Drugs to treat symptoms of Progeria are permitted.
- Subjects are taking medications that significantly affect the metabolism of Progerinin.
- 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 documented by history and confirmed by serology if positive for history.
- 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 Progerinin (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 who have used marijuana or other (Tetrahydrocannabinol) THC containing products either recreationally or for medical purposes within three months prior to entering the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital
Boston, Massachusetts, 02115
About this study
Official title: A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). Subjects in the randomized study arms will continue to take the standard of care (SOC), lonafarnib, and will be randomized to either take SOC alone or in combination with progerinin.
Read the detailed description
During this trial, a total of 10 subjects will be randomized in a 4:1 ratio to receive treatment with progerinin plus lonafarnib vs lonafarnib alone. Dose escalation will occur through intra-subject dose titration. Two ascending doses of progerinin will be assessed. The dose levels proposed in this study were determined using body weight based on adult doses assessed in previously completed studies, and on a simulated population pharmacokinetic (PK) model. If systemic exposures of Progerinin in pediatric patients are lower than those in adults, a dose escalation may be added at the end of the study.
Progerinin will be administered, with food. The study treatment is available in sachets of 250 mg or 350 mg and the doses in the study range from 500 to 1500 mg daily, dosed BID (twice a day).
Lonafarnib is considered as the standard of care (SOC) and all subjects in the randomized study arms will continue taking lonafarnib for the duration of the study. Subjects who are not already taking lonafarnib will also have the opportunity to initiate lonafarnib on this study as part of the lonafarnib naïve arm (separate from the randomized study arms).
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Being tested
- Progerinin, Lonafarnib
- Medical subject headings
- Progeria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ProgerininDrug
Dosing will be determined based on body weight. Dosing ranges from 500mg to 1500mg daily. Progerinin is available in sachets of 250 mg or 350 mg for oral use.
LonafarnibDrug
Lonafarnib is considered as the standard of care (SOC). Subjects in the randomized arms will continue taking lonafarnib per the package insert. Subjects in the lonafarnib naïve arm will initiate lonafarnib as per FDA-approved study 07-01-7505.
| Study group | Role | What they receive |
|---|---|---|
| Progerinin + Lonafarnib | Experimental | Subjects who have successfully completed the Screening phase will enter the treatment phase of the study and be randomized to a treatment arm. Subjects randomized to the progerinin + lonafarnib arm will take progerinin at a dose dependent on their body weight and will continue taking lonafarnib per the package insert.
For each dose cohort, subjects will complete a 28-day treatment period of toxicity assessment, and a minimum four-month treatment period for plasma progerin evaluation. After the last subject completes the toxicity assessment period, the independent Data Monitoring Committee (iDMC) will meet to confirm that subjects can be escalated to the next dose. All subjects will remain on the current dose until the iDMC decision is confirmed. Each subject will only be escalated to the next dose after their 4-month (±2 weeks) plasma progerin sample is collected. Receives: Drug: Progerinin; Drug: Lonafarnib |
| Lonafarnib Naïve | Other | Subjects with no prior treatment with lonafarnib (i.e., no commercial or managed access program source) may be enrolled to initiate treatment with lonafarnib monotherapy. These subjects will initiate lonafarnib therapy in preparation for an upcoming phase of the study following the completion of phase 2a. These subjects will be excluded from the analysis of this phase 2a study. They will come to the study site for a baseline visit and at one year for assessment as described later in this protocol. Receives: Drug: Lonafarnib |
| Lonafarnib | Active comparator | Subjects in the main study arms (Progerinin + Lonafarnib and non-naïve Lonafarnib) will continue taking lonafarnib per the package insert. Subjects randomized to the lonafarnib alone arm will not take progerinin during this study. Receives: Drug: Lonafarnib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in plasma progerin concentration
Measured: Baseline and Month 4 of each cohort
Incidence of Dose Limiting Toxicities (DLTs)
Measured: From baseline to follow-up (approximately 13 months)
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Measured: From baseline to follow-up (approximately 13 months)
Incidence of withdrawals due to Adverse Events (AEs)
Measured: From baseline to follow-up (approximately 13 months)
Incidence of Treatment-Related Adverse Events
Measured: From baseline to follow-up (approximately 13 months)
Incidence of Serious Adverse Events (SAEs)
Measured: From baseline to follow-up (approximately 13 months)
Change/shifts in laboratory values from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in blood pressure from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in heart rate from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in respiratory rate from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in temperature from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, ventricular rate (beats per minute), from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, PR interval (msec), from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, QRS interval (msec), from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, QT interval (msec), from baseline
Measured: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, QTc interval (msec), from baseline
Measured: From baseline to follow-up (approximately 13 months)
Maximum observed plasma drug concentration (Cmax)
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of each cohort
Apparent terminal elimination half-life (t1/2)
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Time to maximum observed plasma drug concentration (Tmax)
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Area under the plasma drug concentration-time curve (AUC) from time 0 to the end of the dosing period (AUC0-τ)
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Clearance (CL)
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Covariates: Age
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of each cohort
Covariates: Body weight
Measured: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of each cohort
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2025
- Study start13 Jan 2025
- Primary completion30 Mar 2026
- Study completion30 Apr 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hutchinson-Gilford Progeria SyndromeStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- PRG Science & Technology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06775041
- Sponsor's protocol ID
- PRG-PRO-005
- Status last verified
- Jan 2026
- First submitted
- 11 Dec 2024
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a, Randomized, Open-Label Study to Determine the Optimal Dose and Evaluate the Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)
- ClinicalTrials.gov ID
- NCT06775041
- Acronym / sponsor ID
- PRG-PRO-005
- Conditions
- Hutchinson-Gilford Progeria Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- PRG Science & Technology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 13 Jan 2025 (actual)
- Primary completion
- 30 Mar 2026 (actual)
- Study completion
- 30 Apr 2026 (actual)
- First posted
- 14 Jan 2025 (actual)
- 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.
Researchers will compare treatment with progerinin plus lonafarnib vs lonafarnib alone to assess optimal dosing, safety, tolerability, and pharmacokinetics in patients with Hutchinson-Gilford Progeria Syndrome (HGPS). Subjects in the randomized study arms will continue to take the standard of care (SOC), lonafarnib, and will be randomized to either take SOC alone or in combination with progerinin.
Detailed description
During this trial, a total of 10 subjects will be randomized in a 4:1 ratio to receive treatment with progerinin plus lonafarnib vs lonafarnib alone. Dose escalation will occur through intra-subject dose titration. Two ascending doses of progerinin will be assessed. The dose levels proposed in this study were determined using body weight based on adult doses assessed in previously completed studies, and on a simulated population pharmacokinetic (PK) model. If systemic exposures of Progerinin in pediatric patients are lower than those in adults, a dose escalation may be added at the end of the study.
Progerinin will be administered, with food. The study treatment is available in sachets of 250 mg or 350 mg and the doses in the study range from 500 to 1500 mg daily, dosed BID (twice a day).
Lonafarnib is considered as the standard of care (SOC) and all subjects in the randomized study arms will continue taking lonafarnib for the duration of the study. Subjects who are not already taking lonafarnib will also have the opportunity to initiate lonafarnib on this study as part of the lonafarnib naïve arm (separate from the randomized study arms).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Progerinin + Lonafarnib | Experimental | Drug: Progerinin, Drug: LonafarnibSubjects who have successfully completed the Screening phase will enter the treatment phase of the study and be randomized to a treatment arm. Subjects randomized to the progerinin + lonafarnib arm will take progerinin at a dose dependent on their body weight and will continue taking lonafarnib per the package insert. For each dose cohort, subjects will complete a 28-day treatment period of toxicity assessment, and a minimum four-month treatment period for plasma progerin evaluation. After the last subject completes the toxicity assessment period, the independent Data Monitoring Committee (iDMC) will meet to confirm that subjects can be escalated to the next dose. All subjects will remain on the current dose until the iDMC decision is confirmed. Each subject will only be escalated to the next dose after their 4-month (±2 weeks) plasma progerin sample is collected. |
| Lonafarnib Naïve | Other | Drug: LonafarnibSubjects with no prior treatment with lonafarnib (i.e., no commercial or managed access program source) may be enrolled to initiate treatment with lonafarnib monotherapy. These subjects will initiate lonafarnib therapy in preparation for an upcoming phase of the study following the completion of phase 2a. These subjects will be excluded from the analysis of this phase 2a study. They will come to the study site for a baseline visit and at one year for assessment as described later in this protocol. |
| Lonafarnib | Active comparator | Drug: LonafarnibSubjects in the main study arms (Progerinin + Lonafarnib and non-naïve Lonafarnib) will continue taking lonafarnib per the package insert. Subjects randomized to the lonafarnib alone arm will not take progerinin during this study. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (17 listed)
- Subjects will be eligible for enrollment in the study only if they meet ALL the following criteria at time of Screening:
- Subjects ≥ 1 year of age and weight ≥ 17.6 lb (8 kg).
- Subject must have confirmatory mutational analysis showing the classic HGPS mutation (c. 1824 C>T), nonclassic HGPS, Zmpset24 gene mutation, or other LMNA mutation (all subjects will havedocumentation of genetic testing prior to enrollment). *Only subjects with progerin producing mutations will be eligible for randomization. Other PL are eligible to enroll as naïve subjects (see note for criterion #5).
- Subject must display clinical signs of Progeria as per the clinical trial team.
- Subject must be willing and able to come to Boston for appropriate assessments and examinations.
Key exclusion criteria (14 listed)
- Subjects meeting ANY of the following criteria at time of Screening will be excluded from enrollment:
- Other than the drugs used in this protocol, other drugs targeted to treat Progeria are excluded. Drugs to treat symptoms of Progeria are permitted.
- Subjects are taking medications that significantly affect the metabolism of Progerinin.
- 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 |
|---|---|---|
| ProgerininDosing will be determined based on body weight. Dosing ranges from 500mg to 1500mg daily. Progerinin is available in sachets of 250 mg or 350 mg for oral use. | Drug | Progerinin + Lonafarnib |
| LonafarnibLonafarnib is considered as the standard of care (SOC). Subjects in the randomized arms will continue taking lonafarnib per the package insert. Subjects in the lonafarnib naïve arm will initiate lonafarnib as per FDA-approved study 07-01-7505. | Drug | Lonafarnib, Lonafarnib Naïve, Progerinin + Lonafarnib |
Primary outcome
Change from baseline in plasma progerin concentration
Time frame: Baseline and Month 4 of each cohort
Incidence of Dose Limiting Toxicities (DLTs)
Time frame: From baseline to follow-up (approximately 13 months)
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Time frame: From baseline to follow-up (approximately 13 months)
Incidence of withdrawals due to Adverse Events (AEs)
Time frame: From baseline to follow-up (approximately 13 months)
Incidence of Treatment-Related Adverse Events
Time frame: From baseline to follow-up (approximately 13 months)
Incidence of Serious Adverse Events (SAEs)
Time frame: From baseline to follow-up (approximately 13 months)
Change/shifts in laboratory values from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in blood pressure from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in heart rate from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in respiratory rate from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in temperature from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, ventricular rate (beats per minute), from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, PR interval (msec), from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, QRS interval (msec), from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, QT interval (msec), from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Change in Electrocardiogram (ECG) parameter, QTc interval (msec), from baseline
Time frame: From baseline to follow-up (approximately 13 months)
Maximum observed plasma drug concentration (Cmax)
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of each cohort
Apparent terminal elimination half-life (t1/2)
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Time to maximum observed plasma drug concentration (Tmax)
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Area under the plasma drug concentration-time curve (AUC) from time 0 to the end of the dosing period (AUC0-τ)
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Clearance (CL)
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of cohort1 & cohort 2
Covariates: Age
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of each cohort
Covariates: Body weight
Time frame: Baseline, and pre-dose (0 hr) and 2-5 hr post-dose at Month 4 of each cohort
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Progerinin and Lonafarnib in people with Hutchinson-Gilford Progeria Syndrome, run by PRG Science & Technology Co., Ltd. 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: finished, as of the sponsor's last update on 2 Oct 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 phase mean?
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change from baseline in plasma progerin concentration (time frame: Baseline and Month 4 of each cohort). The study lists 23 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 Oct 2026.