NCT03976349

A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease

A Phase 1 Single- and Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB094 Administered Intrathecally to Adults With Parkinson's Disease

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 62 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 35 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) injection to participants with Parkinson's Disease (PD). The secondary objective of this study is to evaluate the pharmacokinetic (PK) profile of BIIB094.The study is open for PD patients with verified presence or absence of variations in the leucine-rich repeated kinase 2 (LRRK2) gene, but also for patients without any verified PD-related genetic variant.

Conditions

• Parkinson's Disease

Interventions

Type	Name	Description
DRUG	BIIB094	Administered as specified in the treatment arm.
DRUG	Placebo	Administered as specified in the treatment arm.

Timeline

• Start date: 2019-08-12
• Primary completion: 2024-08-12
• Completion: 2024-08-12
• First posted: 2019-06-06
• Last updated: 2025-09-23

Locations

18 sites across 6 countries: United States, Canada, Israel, Norway, Spain, United Kingdom

Regulatory

• FDA-regulated drug study