NCT05160558

A Pharmacokinetics and Safety Study of BIIB132 in Adults With Spinocerebellar Ataxia 3

A Phase 1, Blinded, Randomized, Placebo-controlled Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Ascending Doses of BIIB132 Administered Intrathecally to Adults With Spinocerebellar Ataxia 3

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Biogen · Industry
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to evaluate the safety and tolerability of multiple ascending doses of BIIB132 administered via intrathecal (IT) injection to participants with spinocerebellar ataxia type 3 (SCA3). The secondary objective of this study is to characterize the multiple-dose pharmacokinetics (PK) of BIIB132 administered via IT injection to participants with SCA3.

Detailed description

BIIB132 is an investigational anti-sense oligonucleotide developed to target ataxin-3 (ATXN3) pre-messenger ribonucleic acid (pre-mRNA). Preclinical studies have shown that lowering of ATXN3 protein is associated with decreased progression of SCA3-like disease. This trial consists of a blinded 12 week study period with a 26 week follow up period to evaluate the safety and tolerability of intrathecal BIIB132 and to assess the effect on treatment response biomarkers in symptomatic SCA3 participants.

Conditions

• Spinocerebellar Ataxia Type 3

Interventions

Type	Name	Description
DRUG	BIIB132	Administered as specified in the treatment arm
DRUG	BIIB132-Matching Placebo	Administered as specified in the treatment arm

Timeline

• Start date: 2022-02-02
• Primary completion: 2023-07-25
• Completion: 2023-07-25
• First posted: 2021-12-16
• Last updated: 2023-08-04

Locations

20 sites across 6 countries: United States, Germany, Israel, Netherlands, Portugal, United Kingdom

Regulatory

• FDA-regulated drug study