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

ConditionWerner Syndrome
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorPRG Science & Technology Co., Ltd.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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 (T-score ≤ -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.

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • 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 groups

5 Participants
are divided into 1 treatment group
Group A: Treatment GroupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

The intensity of the event will be graded using CTCAE v6.0 criteria

Time frame
Baseline to Week 56 (including 4-week follow-up)
2

Incidence of Treatment-Related Adverse Events

Time frame
Baseline to Week 56 (including 4-week follow-up)
3

Incidence of Serious Adverse Events (SAEs)

Time frame
Baseline to Week 56 (including 4-week follow-up)
4

Incidence of TEAEs and SAEs leading to discontinuation of study medication

Time frame
Baseline to Week 56 (including 4-week follow-up)

Secondary outcomes

1

Change in Femur Bone Mineral Density (BMD) from baseline

Time frame
Baseline, Week 24, and Week 52
2

Change in Lumbar Spine BMD from baseline

Time frame
Baseline, Week 24, and Week 52
3

Change in Aortic Pulse Wave Velocity (m/s) from baseline

Time frame
Baseline, Week 24, and Week 52
4

Change in Insulin Resistance by Oral Glucose Tolerance Test (OGTT) from baseline

Time frame
Baseline, Week 24, and Week 52

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

PRG Science & Technology Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov
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