Ignite Creation Date:
2024-05-06 @ 3:03 PM
Last Modification Date:
2024-10-26 @ 1:42 PM
Study NCT ID:
NCT04512963
Status:
COMPLETED
Last Update Posted:
2021-09-22
First Post:
2020-08-06
Brief Title:
Phase I Study of Progerinin in Healthy Volunteers
Sponsor:
PRG Science Technology Co Ltd
Organization:
PRG Science Technology Co Ltd
Study Overview
Official Title:
A Phase I Randomized Double-Blind Placebo-Controlled Single Ascending Dose SAD Study Including a Food Interaction Study Followed by a Multiple Ascending Dose MAD Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers
Status:
COMPLETED
Status Verified Date:
2021-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
PRG-PRO-001 is a Phase I Randomized Double-blind Placebo-Controlled Single Ascending Dose SAD Study including a food interaction study followed by a Multiple Ascending Dose MAD Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers This is a first-in-human study The study aims to determine the safety and tolerability of Progerinin after single and multiple doses in healthy volunteers and to evaluate the pharmacokinetics PK of Progerinin after single and multiple dose administrations in healthy volunteers
Detailed Description:
PRG Science Technology Co Ltd PRG ST is developing Progerinin SLC-D011 for the treatment of the rare aging diseases Hutchinson-Gilford Progeria Syndrome HGPS and Werner Syndrome WS
Progerin an abnormal splice variant of the inner nuclear membrane protein lamin A is implicated in the pathology of HGPS and WS It is believed that the extremely strong binding between lamin A and progerin is responsible for the nuclear abnormality phenotype observed in HGPS WS is caused by functional defect of WRN RecQ4L DNA helicase and rapid accumulation of progerin in WRN deficient condition is thought to be the cause of premature aging in WS PRG ST has shown that Progerinin binds specifically to progerin reduces its expression in both HGPS and WS cells Progerinin further prevents progerin-lamin A in HGPS cells In a progeria mouse model LmnaG609GG609G treatment with Progerinin via intraperitoneal ip injection 20 mgkg twice per week could increase the body weight and extend the life span of LmnaG609GG609G mice up to 21 weeks In the LmnaG609G mouse model improved morphology such as status of coat hair and body size increased body weight and extended life span for about 16 weeks was noted following Progerinin treatment In addition Progerinin can suppress muscle weakness including the heart muscle Safety pharmacology studies did not indicate any Progerinin-related effects on vital organs and systems including respiratory cardiovascular and central nervous system
Prior to studies in the disease states this study PRG-PRO-001 an initial first-in-human study will be conducted in healthy volunteers to assess the safety tolerability and pharmacokinetics of Progerinin PRG-PRO-001 is a Phase I Randomized Double-blind Placebo-Controlled Single Ascending Dose SAD Study including a food interaction study followed by a Multiple Ascending Dose MAD Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers This is a first-in-human study The study aims to determine the safety and tolerability of Progerinin after single and multiple doses in healthy volunteers and to evaluate the pharmacokinetics PK of Progerinin after single and multiple dose administrations in healthy volunteers The estimated enrollment is around 56 healthy volunteers with 40 subjects estimated for the SAD phase and 16 subjects for the MAD Phase at one site in the USA
Expanded Access Program is available For more details please use the link in the References Section below to access our companys main webpage on Expanded Access Program
Progerin an abnormal splice variant of the inner nuclear membrane protein lamin A is implicated in the pathology of HGPS and WS It is believed that the extremely strong binding between lamin A and progerin is responsible for the nuclear abnormality phenotype observed in HGPS WS is caused by functional defect of WRN RecQ4L DNA helicase and rapid accumulation of progerin in WRN deficient condition is thought to be the cause of premature aging in WS PRG ST has shown that Progerinin binds specifically to progerin reduces its expression in both HGPS and WS cells Progerinin further prevents progerin-lamin A in HGPS cells In a progeria mouse model LmnaG609GG609G treatment with Progerinin via intraperitoneal ip injection 20 mgkg twice per week could increase the body weight and extend the life span of LmnaG609GG609G mice up to 21 weeks In the LmnaG609G mouse model improved morphology such as status of coat hair and body size increased body weight and extended life span for about 16 weeks was noted following Progerinin treatment In addition Progerinin can suppress muscle weakness including the heart muscle Safety pharmacology studies did not indicate any Progerinin-related effects on vital organs and systems including respiratory cardiovascular and central nervous system
Prior to studies in the disease states this study PRG-PRO-001 an initial first-in-human study will be conducted in healthy volunteers to assess the safety tolerability and pharmacokinetics of Progerinin PRG-PRO-001 is a Phase I Randomized Double-blind Placebo-Controlled Single Ascending Dose SAD Study including a food interaction study followed by a Multiple Ascending Dose MAD Study to Evaluate the Safety Tolerability Pharmacokinetics and Pharmacodynamic Profile of Progerinin in Healthy Volunteers This is a first-in-human study The study aims to determine the safety and tolerability of Progerinin after single and multiple doses in healthy volunteers and to evaluate the pharmacokinetics PK of Progerinin after single and multiple dose administrations in healthy volunteers The estimated enrollment is around 56 healthy volunteers with 40 subjects estimated for the SAD phase and 16 subjects for the MAD Phase at one site in the USA
Expanded Access Program is available For more details please use the link in the References Section below to access our companys main webpage on Expanded Access Program
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None