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.
Subjects who have successfully completed the Screening phase will enter the baseline and treatment phase of the study. Subjects will take the assigned treatment of Progerinin (2400 mg) split as 1200 mg BID in the morning and evening with food for 1-Year. To evaluate the safety and tolerability of Progerinin for the treatment of BMD loss in subjects with typical Werner syndrome. The secondary objectives of this study are: * To characterize the activity of Progerinin on bone metabolism in subjects with typical Werner syndrome. * To evaluate the pharmacokinetics (PK) of Progerinin sachet administered twice daily (BID) in subjects with typical Werner syndrome
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
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.
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
The intensity of the event will be graded using CTCAE v5.0 criteria
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Incidence of Treatment-Related Adverse Events
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Incidence of Serious Adverse Events (SAEs)
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Incidence of TEAEs and SAEs leading to discontinuation of study medication
Time frame: week 54
Changes in Blood Hemoglobin (g/dl) from baseline
Time frame: week 54
Changes in Blood White Blood Cell (cell per microliter) from baseline
Time frame: week 54
Changes in Blood Platelets (cell per microliter) from baseline
Time frame: week 54
Changes in Serum Creatinine (µmol/L) from baseline
Time frame: week 54
Changes in Serum Alanine Aminotransferase (IU/l) from baseline
Time frame: week 54
Changes in Serum Aspartate Aminotransferase (IU/l) from baseline
Time frame: week 54
Changes in Serum Alkaline Phosphatase (microkatal per liter) from baseline
Time frame: week 54
Changes in weight (kg) from baseline
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Time frame: week 54
Changes in pulse rate (beats per minute) from baseline
Time frame: week 54
Changes in respiratory rate (breaths per minute) from baseline
Time frame: week 54
Changes in temperature (°F) from baseline
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Changes in systolic Blood pressure (mm Hg) from baseline
Time frame: week 54
Changes in Diastolic Blood pressure (mm Hg) from baseline
Time frame: week 54
Change in ECG ventricular rate from baseline (beats per minute)
Time frame: week 54
Change in ECG PR interval (msec)
Time frame: week 54
Change in ECG QRS interval (msec)
Time frame: week 54
Change in ECG QT interval (msec)
Time frame: week 54
Change in ECG QTc interval (msec)
Time frame: week 54