Active — Not RecruitingPhase 2

Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS)

United States10 participantsStarted 2025-01-13

Plain-language 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). 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.

Who can participate

Age range1 Year

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.
✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Change from baseline in plasma progerin concentration

Timeframe: Baseline and Month 4 of each cohort

Incidence of Dose Limiting Toxicities (DLTs)

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence of withdrawals due to Adverse Events (AEs)

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence of Treatment-Related Adverse Events

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence of Serious Adverse Events (SAEs)

Timeframe: From baseline to follow-up (approximately 13 months)

Change/shifts in laboratory values from baseline

Timeframe: From baseline to follow-up (approximately 13 months)

Change in blood pressure from baseline

Trial details

NCT IDNCT06775041

SponsorPRG Science & Technology Co., Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-03

View on ClinicalTrials.gov More Hutchinson-Gilford Progeria Syndrome trials

Plain-language summary

Who can participate

Age range1 Year

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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.
✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

Change from baseline in plasma progerin concentration

Timeframe: Baseline and Month 4 of each cohort

Incidence of Dose Limiting Toxicities (DLTs)

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence of withdrawals due to Adverse Events (AEs)

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence of Treatment-Related Adverse Events

Timeframe: From baseline to follow-up (approximately 13 months)

Incidence of Serious Adverse Events (SAEs)

Timeframe: From baseline to follow-up (approximately 13 months)

Change/shifts in laboratory values from baseline

Timeframe: From baseline to follow-up (approximately 13 months)

Change in blood pressure from baseline