Trials Sponsors Conditions Drugs Pricing

Conditions

Hutchinson-Gilford Progeria Syndrome Werner Syndrome

Keywords

HGPSWSProgeria

Outcome Measures

Primary Outcomes (14)

Incidence of Dose Limiting Toxicities (DLTs)
Any clinically significant adverse event (AE)/serious adverse event (SAE) or clinically significant laboratory abnormality which is classified as \> Grade 2 (according to the National Cancer Institute Common Terminology Criteria for Adverse Events \[CTCAE\] version 5.0), where applicable, deemed by the investigator as at least "possibly, probably or definitely related" to Progerinin but unrelated to concurrent illness, or concomitant medications.
Day 1 through 7 days after the last study drug administration
Incidence of Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) is defined as any unfavorable or unintended sign, symptom, or disease that occurs or is reported by the patient to have occurred, or a worsening of a pre-existing condition. An adverse event may or may not be related to the study treatment.
Day 1 through 7 days after the last study drug administration
Incidence of withdrawals due to Adverse Events (AEs)
Incidence of withdrawals due to Adverse Events (AEs) defined above
Day 1 through 7 days after the last study drug administration

Study Arms (2)

SAD Phase including Food Interaction

EXPERIMENTAL

Single Ascending Dose (SAD) study with up to 6 cohorts + Food interaction phase on two cohorts

Drug: ProgerininDrug: Placebo

MAD Phase

EXPERIMENTAL

Multiple Ascending Dose (MAD) study with up to 2 cohorts

Drug: ProgerininDrug: Placebo

Interventions

ProgerininDrug

100 mg/g nano-suspension for oral use

Also known as: SLC-D011

MAD PhaseSAD Phase including Food Interaction

PlaceboDrug

A substance that has no therapeutic effect, used as a control in testing Progerinin

Eligibility Criteria

Age18 Years - 45 Years

Sexall

Healthy VolunteersYes

Age GroupsAdult (18-64)

You may qualify if:

Subjects will be eligible for enrollment in the study only if they meet ALL of the following criteria:
Healthy male and female subjects, 18 to 45 years of age, inclusive.

Sponsors & Collaborators

PRG Science & Technology Co., Ltd.
Industrylead
Amarex Clinical Research
Othercollaborator

Study Sites (1)

PRG S&T Investigational Site

Glendale, California, 91206, United States

MeSH Terms

Conditions

ProgeriaWerner Syndrome

Condition Hierarchy (Ancestors)

LaminopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesDNA Repair-Deficiency Disorders

Enrollment

64 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 1
Allocation: RANDOMIZED
Masking: QUADRUPLE
Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose: TREATMENT
Intervention Model: SEQUENTIAL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

You may not qualify if:

Subjects will be eligible for enrollment in the study only if they meet NONE of the following criteria:
The subject has a history of severe allergic or anaphylactic reactions.
The subject has a known allergy or hypersensitivity to any component of the formulation.
The subject has a medical history or current evidence of any clinically significant (as determined by the investigator) cardiac, endocrine (including diabetes), hematologic, hepatobiliary (abnormal 12lanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], gamma-glutamyl transpeptidase \[GGT\], or total bilirubin), immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, or renal condition, or other major disease.
The subject has a history of any malignant disease.
The subject has a history of more than one herpes zoster episode or multimetameric herpes zoster.
The subject has a history of an opportunistic infection (e.g. cytomegalovirus, pneumocystis carinii, aspergillosis, clostridium difficile).
The subject has a history of or ongoing chronic or recurrent infectious disease (e.g. infected indwelling prosthesis, osteomyelitis, chronic sinusitis).
The subject has had major trauma or surgery in the 2 months before screening or at any time between screening and check-in.
The subject has had an acute infection within 2 weeks before screening or at any time between screening and check-in including, but not limited to, history, signs, or symptoms of a common cold (eg, mild rhinorrhea), untreated oral/dental abnormalities (e.g. untreated dental caries as determined by examination of the mouth), or untreated disruption of the skin.
The subject has clinically significant abnormal ECG findings at screening, check-in visits, or predose, as determined by the Investigator.
The subject has a supine blood pressure measurement outside the ranges of 90 to 140 mm Hg systolic or 45 to 90 mm Hg diastolic (measured after a rest of at least 5 minutes) at screening, check-in, or predose. Note: If either value is out of the range, blood pressure measurements may be repeated in the supine position at intervals of 5 to 10 minutes up to 3 times. If the mean systolic or diastolic measurement continues to exceed the stated limits, the subject will be excluded.
The subject has a pulse of fewer than 45 beats per minute (bpm) or greater than 100 bpm (measured after a rest of at least 5 minutes) at screening, check-in, or predose.
The subject tests positive for tuberculosis (TB) at screening by the QuantiFERON-TB Gold Test, or has a history of latent, inadequately treated, or active TB.
The subject has a known history of, or a positive test result for, hepatitis B surface antigen (HbsAg), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) types 1 or 2 at screening.
+11 more criteria

Phase I Study of Progerinin in Healthy Volunteers

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (14)

Study Arms (2)

SAD Phase including Food Interaction

MAD Phase

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Links

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

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

Keywords

Outcome Measures

Primary Outcomes (14)

Study Arms (2)

SAD Phase including Food Interaction

MAD Phase

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

Related Links

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations