Clinical Trials Logo
  1. Home
  2. Spinocerebellar Ataxia Type 3
  3. NCT05160558

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

Spinocerebellar Ataxia Type 3 Clinical Trial

Official title:
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

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05160558
Study type Interventional
Source Biogen
Contact
Status Terminated
Phase Phase 1
Start date February 2, 2022
Completion date July 25, 2023
View Details
See also
  Status Clinical Trial Phase
Completed NCT00992771 - Study to Determine the Safety and Tolerability of Varenicline (Chantix®) in Treating Spinocerebellar Ataxia Type 3 Phase 2
Completed NCT01096082 - Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3 Phase 2/Phase 3
Active, not recruiting NCT03701399 - Troriluzole in Adult Subjects With Spinocerebellar Ataxia Phase 3
Active, not recruiting NCT04229823 - Natural History of Oculomotor Neurophysiology in Ataxic and Pre-ataxic Carriers of SCA3/MJD
Active, not recruiting NCT04419974 - Astrocytic Markers and the Pre-ataxic Period of SCA3/MJD - BIGPRO Study Astrocytes
Terminated NCT05490563 - STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia Phase 2/Phase 3
Withdrawn NCT04301284 - Study of CAD-1883 for Spinocerebellar Ataxia Phase 2
Withdrawn NCT01096095 - Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3 Phase 2
Active, not recruiting NCT04268147 - Instrumented Data Exchange for Ataxia Study
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Active, not recruiting NCT05826171 - Priming Motor Learning Through Exercise in People With Spinocerebellar Ataxia N/A
Recruiting NCT04399265 - Efficacy Of Oral Trehalose In Spinocerebellar Ataxia 3 N/A
Completed NCT03885167 - Identification of Biomarkers in Spinocerebellar Ataxia 3
Recruiting NCT05822908 - A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD Phase 1/Phase 2
Not yet recruiting NCT03378414 - Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia Phase 2
Completed NCT05502432 - Repetitive Transcranial Magnetic Stimulation in SCA3 Patients N/A
Recruiting NCT04714307 - Neuropsychiatry and Cognition in SCA3/MJD
Recruiting NCT05557786 - Treatment of Transcranial Alternating Current Stimulation（tACS）on Cerebellar Ataxia
Recruiting NCT01060371 - Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias