Trials Sponsors Conditions Drugs Pricing

Conditions

Werner Syndrome

Outcome Measures

Primary Outcomes (20)

Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
The intensity of the event will be graded using CTCAE v6.0 criteria
Baseline to Week 56 (including 4-week follow-up)
Incidence of Treatment-Related Adverse Events
Baseline to Week 56 (including 4-week follow-up)
Incidence of Serious Adverse Events (SAEs)
Baseline to Week 56 (including 4-week follow-up)
Incidence of TEAEs and SAEs leading to discontinuation of study medication
Baseline to Week 56 (including 4-week follow-up)
Changes in Blood Chemistry from Baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Hematology from Baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Urinalysis Results from Baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Height (cm) from Baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Weight (kg) from baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Temperature (°C) from baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Pulse Rate (beats per minute) from baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Respiratory Rate (breaths per minute) from baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Systolic Blood pressure (mmHg) from baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Diastolic Blood pressure (mmHg) from baseline
Baseline to Week 56 (including 4-week follow-up)
Changes in Physical Examination findings from baseline
Baseline to Week 56 (including 4-week follow-up)
Change in ECG Ventricular Rate (beats per minute) from baseline
Baseline to Week 56 (including 4-week follow-up)
Change in ECG PR interval (msec) from baseline
Baseline to Week 56 (including 4-week follow-up)
Change in ECG QRS interval (msec) from baseline
Baseline to Week 56 (including 4-week follow-up)
Change in ECG QT interval (msec) from baseline
Baseline to Week 56 (including 4-week follow-up)
Change in ECG QTc interval (msec) from baseline
Baseline to Week 56 (including 4-week follow-up)

Secondary Outcomes (14)

Change in Femur Bone Mineral Density (BMD) from baseline
Baseline, Week 24, and Week 52
Change in Lumbar Spine BMD from baseline
Baseline, Week 24, and Week 52
Change in Aortic Pulse Wave Velocity (m/s) from baseline
Baseline, Week 24, and Week 52
Change in Insulin Resistance by Oral Glucose Tolerance Test (OGTT) from baseline
Baseline, Week 24, and Week 52
Change in Lipid Profile from baseline
Baseline, Week 24, and Week 52
+9 more secondary outcomes

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

ProgerininDrug

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

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age18 Years+

Sexall

Healthy VolunteersNo

Age GroupsAdult (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.

Sponsors & Collaborators

PRG Science & Technology Co., Ltd.
Industrylead

Study Sites (1)

University of Michigan

Ann Arbor, Michigan, 48109, United States

RECRUITING

MeSH Terms

Conditions

Werner Syndrome

Condition Hierarchy (Ancestors)

Genetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesDNA Repair-Deficiency DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Central Study Contacts

Christine Lee

CONTACT

301-540-2600 christinelee@kcrnresearch.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

October 2026

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

Primary Completion

December 2027

Estimated

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (20)

Secondary Outcomes (14)

Study Arms (1)

Treatment Group

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (20)

Secondary Outcomes (14)

Study Arms (1)

Treatment Group

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Central Study Contacts

Study Design

Study Record Dates

You may not qualify if:

Source record

Locations