NCT03533257

Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

Phase II Study to Assess the Safety, Tolerability, and Target Engagement of AMX0035, a Fixed Combination of Sodium Phenylbutyrate and Tauroursodeoxycholic Acid for the Treatment of Alzheimer's Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 95 (actual)
• Sponsor: Amylyx Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 55 Years – 89 Years
• Healthy volunteers: Not accepted

Summary

The study is a 24-week, randomized, double-blind, multi-site, placebo-controlled study in participants with mild cognitive impairment (MCI) or early dementia due to Alzheimer's disease (AD).

Detailed description

The study is a 24-week, randomized, double-blind, multi-site, placebo-controlled study in participants with mild cognitive impairment (MCI) or early dementia due to Alzheimer's disease (AD). The study is designed to evaluate the safety, tolerability, drug target engagement and neurobiological effects of treatment with AMX0035 over 24 weeks. The study is designed to yield deep phenotyping insight for the purposes of demonstrating the effects of AMX0035 on mechanistic targets of engagement and disease biology. The study will evaluate diverse disease-relevant markers and produce an informative dataset that will allow for evaluation and correlation of imaging-based markers, neurobiological changes, functional measures, and cognitive outcomes. Participants receive orally administered study drug twice daily for a treatment duration of approximately 24 weeks and attend clinic visits at Screening, Baseline, Week 6, Week 12, Week 18, and Week 24.

Conditions

• Alzheimer Disease

Interventions

Type	Name	Description
DRUG	AMX0035	Combination Therapy of PB and TURSO
DRUG	Placebo	Placebo

Timeline

• Start date: 2018-09-14
• Primary completion: 2020-10-22
• Completion: 2020-11-06
• First posted: 2018-05-23
• Last updated: 2025-03-07
• Results posted: 2025-03-07

Locations

11 sites across 1 country: United States

Regulatory

• FDA-regulated drug study