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Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Sponsor: Arrowhead Pharmaceuticals
Summary
Adult participants with spinocerebellar ataxia type 2 (SCA2) who carry ≥33 cytosine, adenine, guanine (CAG) repeats in the ATXN2 gene, and who have met all protocol eligibility criteria will be randomized to receive a single dose of ARO-ATXN2 or placebo and be evaluated for safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) parameters.
Official title: A Phase 1 Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-ATXN2 in Adult Subjects With Spinocerebellar Ataxia Type 2
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
36
Start Date
2024-12-17
Completion Date
2026-12
Last Updated
2026-02-06
Healthy Volunteers
No
Conditions
Interventions
ARO-ATXN2 Injection
single dose of ARO-ATXN2 by intrathecal (IT) administration
Placebo
calculated volume to match active treatment by IT administration
Locations (16)
Research Site 8
Sydney, New South Wales, Australia
Research Site 7
Melbourne, Victoria, Australia
Research Site 2
Montreal, Quebec, Canada
Research Site 1
Montreal, Quebec, Canada
Research Site 9
Edmonton, Canada
Research Site 15
Paris, Paris, France
Research Site 13
Tübingen, Baden-Wurttemberg, Germany
Research Site 14
Ulm, Baden-Wurttemberg, Germany
Research Site 16
Milan, Italy
Research Site 4
Auckland, New Zealand
Research Site 3
Christchurch, New Zealand
Research Site 10
Barcelona, Spain
Research Site 11
Barcelona, Spain
Research Site 12
Seville, Spain
Research Site 5
Kaohsiung City, Taiwan
Research Site 6
Taipei, Taiwan