Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin in Werner Syndrome

NCT05847179 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: PRG-PRO-004
• Study Type: interventional
• Target: 5
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a Phase 2, open-label study to A Phase 2, Open-Label Study to Evaluate the Safety and Tolerability of Progerinin for the Treatment of Bone Mineral Density (BMD) Loss in Subjects with Typical Werner Syndrome. There will be up to 5 subjects that will receive treatment with Progerinin twice daily for approximately 1 year.

Conditions

Werner Syndrome

Outcome Measures

Primary Outcomes (22)

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

  The intensity of the event will be graded using CTCAE v5.0 criteria

  week 54

• Incidence of Treatment-Related Adverse Events

  week 54

• Incidence of Serious Adverse Events (SAEs)

  week 54

• Incidence of TEAEs and SAEs leading to discontinuation of study medication

  week 54

• Changes in Blood Hemoglobin (g/dl) from baseline

  week 54

• Changes in Blood White Blood Cell (cell per microliter) from baseline

  week 54

• Changes in Blood Platelets (cell per microliter) from baseline

  week 54

• Changes in Serum Creatinine (µmol/L) from baseline

  week 54

• Changes in Serum Alanine Aminotransferase (IU/l) from baseline

  week 54

• Changes in Serum Aspartate Aminotransferase (IU/l) from baseline

  week 54

• Changes in Serum Alkaline Phosphatase (microkatal per liter) from baseline

  week 54

• Changes in weight (kg) from baseline

  week 54

• Changes in pulse rate (beats per minute) from baseline

  week 54

• Changes in respiratory rate (breaths per minute) from baseline

  week 54

• Changes in temperature (°F) from baseline

  week 54

• Changes in systolic Blood pressure (mm Hg) from baseline

  week 54

• Changes in Diastolic Blood pressure (mm Hg) from baseline

  week 54

• Change in ECG ventricular rate from baseline (beats per minute)

  week 54

• Change in ECG PR interval (msec)

  week 54

• Change in ECG QRS interval (msec)

  week 54

• Change in ECG QT interval (msec)

  week 54

• Change in ECG QTc interval (msec)

  week 54

Study Arms (1)

Treatment Group

EXPERIMENTAL

all subjects will receive Progerinin 2400mg (1200mg twice daily) after morning and evening meals for a year

Drug: Progerinin

Interventions

Progerinin DRUG

This is a Phase 2, open-label study to evaluate the safety and Tolerability of Progerinin for the treatment of BMD Bone Mineral Density (BMD) Loss in Subjects with Typical Werner Syndrome. . There will be up to 5 subjects that will receive treatment with Progerinin twice daily for approximately 1 year. This study will have three phases: Screening Phase, Treatment Phase, and Follow-Up Phase.

Treatment Group

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects must be at least ≥ 18 years of age.
• Subjects should have a confirmed diagnosis of typical Werner syndrome by genetic analysis.
• Subjects should be osteopenic (T-score between -1.0 and -2.5) or have confirmed osteoporosis (Tscore ≤ -2.5) at screening/baseline. Subjects may continue to receive bone supplements (e.g., bisphosphonates, calcium supplements, Vitamin D supplements, etc.) as appropriate per standard of care throughout the study.
• Male or non-pregnant, non-lactating female.
• Subjects screening blood chemistry inclusive of metabolic panel, hematology, and urine analysis results should be acceptable to the investigator.
• Negative pregnancy test for female subjects as described below. Women of child bearing potential (WOCBP) and Women not of child-bearing potential are eligible to participate. Both women of childbearing potential and women of non-child-bearing potential should use an approved method of birth control and agrees to continue to use this method for the duration of the study and for 90 days after last treatment.
• 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), female subject/partner's use of an intrauterine device (IUD), or if the female subject/partner is surgically sterile or two years post-menopausal. All male subjects/partners must agree to use a In addition, subjects may not donate sperm for the duration of the study and for 90 days after last treatment.
• Females who are less than two (2) years post-menopausal, those with tubal ligations and those using contraception must have a negative serum pregnancy test at baseline within the one (1) week prior to the first study medication administration. Every six weeks, and at study termination a pregnancy test should be performed, either serum or urine stick test. However, if the urine result is positive, a serum pregnancy test will be performed.
• Provide signed written informed consent and willingness, ability to comply with study requirements.
• Subject must have a projected life expectancy of ≥ 12 months in the opinion of the Investigator.

You may not qualify if:

• Subject with clinical signs seen at screening are at the final stage of Werner syndrome progression, and completion of the study is difficult to be assessed, including:
• Subjects who received continuous or intermittent home oxygen therapy for 6 months before obtaining consent
• Subjects who received at least 2 hospitalizations for pneumonia during the 12 months prior to obtaining consent
• Subjects who have at least 10% net weight loss and have not recovered. This includes significant net weight loss over the last six months.
• Subjects with significant dehydration as judged by the principal investigator.
• Subjects with pericardial fluid, ascites and pleural effusion.
• Therapy with investigational drugs within 30 days of beginning study medication.
• History of prior malignancy, except for adequately treated in situ cancer, basal cell, squamous cell skin cancer, or other cancers (e.g., breast, prostate) for which the subject has been disease-free for at least 3 years.
• Any serious medical condition, laboratory abnormality, psychiatric illness, or comorbidity that, in the judgment of the Investigator, would make the subject inappropriate for the study.
• Serious systemic fungal, bacterial, viral, or other infection that is not controlled or requires intravenous (IV) treatment for infection(s).
• Subjects with known history of having Acquired Immunodeficiency Syndrome (AIDS) or with a history known to be infected with Human Immunodeficiency Virus (HIV).
• Subjects with a history of serious drug hypersensitivity or allergic reaction such as anaphylaxis or any component of the formulation.
• Clinically significant bleeding within 2 weeks prior to baseline (e.g., gastrointestinal \[GI\] bleeding, intracranial hemorrhage).
• Medical or recreational use of marijuana or THC-containing compounds within 3 months of screening visit and for the duration of the trial.
• Pregnant or lactating women.

Sponsors & Collaborators

• PRG Science & Technology Co., Ltd.: lead

MeSH Terms

Conditions

Werner Syndrome

Condition Hierarchy (Ancestors)

Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; DNA Repair-Deficiency Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Anand Balasubramanian

CONTACT

301-956-2531; anandb@amarexcro.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2023
• First Posted: May 6, 2023
• Study Start (Estimated): July 1, 2026
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: January 23, 2026

Record last verified: 2026-01