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

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.

Timeline

Start

2019-08-12

Primary completion

2024-08-12

Completion

2024-08-12

Publications

• Mabrouk OS, Tichler B, Arnold HM, Thoma EC, Alexanian SM, Chen J, Liu TY, Hirschhorn B, Llorens Arenas R, Annand JW, Kapadnis U, Shomo AA, Glajch KE, Ferber K, Moroishi Y, Czerkowicz J, Inra J, Postuma RB, Gurevich T, Mir P, Morris HR, Aldred J, Brodsky MA, Ellenbogen A, Larson D, Tolleson CM, Siderowf A, Tzoulis C, Balaguer E, Marti MJ, Zhao HT, Kordasiewicz HB, Lane R, Hirst WD, Fradette S, Graham DL. LRRK2-targeting antisense oligonucleotide in Parkinson's disease: a phase 1 randomized controlled trial. Nat Med. 2026 Apr;32(4):1421-1431. doi: 10.1038/s41591-026-04262-4. Epub 2026 Mar 24.

Drugs

Indications