Ignite Creation Date:
2025-12-25 @ 12:26 AM
Ignite Modification Date:
2026-01-01 @ 6:52 AM
Study NCT ID:
NCT04505358
Status:
WITHDRAWN
Last Update Posted:
2022-11-17
First Post:
2020-07-23
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Evaluate PU-AD in Subjects With Amyotrophic Lateral Sclerosis
Sponsor:
Samus Therapeutics, Inc.
Organization:
Study Overview
Official Title:
A Randomized, Double-blind, Placebo-controlled Pilot Study to Evaluate the Biological Activity, Safety, and Pharmacokinetics of PU-AD in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Status:
WITHDRAWN
Status Verified Date:
2022-11
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Samus Therapeutics company closure
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ALS
Brief Summary:
This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment.
Detailed Description:
This is a multicenter, Phase 2a, randomized, double-blind, placebo-controlled pilot study to assess the biological activity, safety and pharmacokinetics of PU-AD compared to placebo in ALS. It will be conducted in approximately 20 sites in the US. Approximately 30 subjects will be enrolled in this study; subjects will be randomized 3:2 to receive either PU-AD 30 mg or matching placebo qd, added onto any current stable background treatment. Subjects who meet all inclusion criteria and none of the exclusion criteria will be eligible to participate in this study.
The study consists of a Screening Period (including background treatment stabilization, if required) (4 weeks), Treatment Period (24 weeks) and a safety Follow up Visit (4 weeks \[±1 week\] after the last dose of investigational medicinal product \[IMP\]). The expected study duration is up to 32 weeks.
The Screening visit will take place within 4 weeks prior to dosing, to assess eligibility of subjects. Eligible subjects will be randomized and then return to the site for baseline assessments at Week 1. During the 24 week Treatment Period, subjects will be administered 30 mg PU AD or matching placebo qd, orally, on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day.
The study consists of a Screening Period (including background treatment stabilization, if required) (4 weeks), Treatment Period (24 weeks) and a safety Follow up Visit (4 weeks \[±1 week\] after the last dose of investigational medicinal product \[IMP\]). The expected study duration is up to 32 weeks.
The Screening visit will take place within 4 weeks prior to dosing, to assess eligibility of subjects. Eligible subjects will be randomized and then return to the site for baseline assessments at Week 1. During the 24 week Treatment Period, subjects will be administered 30 mg PU AD or matching placebo qd, orally, on an empty stomach (1 hour prior to food or 2 hours after), at about the same time each day.
Study Oversight
Has Oversight DMC:
True
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?: