NCT04505358

Evaluate PU-AD in Subjects With Amyotrophic Lateral Sclerosis

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• Status: Withdrawn
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Samus Therapeutics, Inc. · Industry
• Sex: All
• Age: 20 Years – 80 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Amyotrophic Lateral Sclerosis (ALS)

Interventions

Type	Name	Description
DRUG	PU-AD	active vs placebo
DRUG	Placebo	active vs placebo

Timeline

• Start date: 2023-01-01
• Primary completion: 2024-12-01
• Completion: 2025-01-01
• First posted: 2020-08-10
• Last updated: 2022-11-17

Regulatory

• FDA-regulated drug study