NCT04987671

Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS

A Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS

• Status: Unknown
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 14 (estimated)
• Sponsor: Amylyx Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the pharmacokinetic and pharmacodynamic effect after a single dose or at steady state after multiple doses of AMX0035 in adults with sporadic ALS.

Detailed description

AMX0035 is a proprietary combination of two small molecules, phenylbutyrate (PB) and taurursodiol. The combination of PB and taurursodiol in AMX0035 is intended to block neuronal death and neurotoxic inflammation through simultaneous reduction of endoplasmic reticulum (ER) stress and mitochondrial stress. The pharmacokinetic of AMX0035 has been evaluated in healthy volunteers. This Phase IIa trial is intended to evaluate pharmacokinetic of PB and taurursodiol upon single dose multiple dose administration of AMX0035 in patients with ALS.

Conditions

• ALS

Interventions

Type	Name	Description
DRUG	AMX0035	Fixed dose combination of Sodium Phenylbutyrate and taurursodiol.

Timeline

• Start date: 2021-08-05
• Primary completion: 2022-05-11
• Completion: 2023-09-01
• First posted: 2021-08-03
• Last updated: 2023-07-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study