Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Condition(s) studied
Investigational drug(s) / intervention(s)
ARO-ATXN2 Injection: single dose of ARO-ATXN2 by intrathecal (IT) administration
Placebo: calculated volume to match active treatment by IT administration
Study 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.
Eligibility
Primary outcome measure(s)
- Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time — Through End of Study (EOS), Day 253
Trial sites (16)
| Facility | City | Region | Status |
|---|---|---|---|
| Research Site 8 | Sydney | New South Wales | |
| Research Site 7 | Melbourne | Victoria | |
| Research Site 2 | Montreal | Quebec | |
| Research Site 1 | Montreal | Quebec | |
| Research Site 9 | Edmonton | Canada | |
| Research Site 15 | Paris | France | |
| Research Site 13 | Tübingen | Baden-Wurttemberg | |
| Research Site 14 | Ulm | Baden-Wurttemberg | |
| 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 |
More Arrowhead Pharmaceuticals trials in Italy
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06672445 on ClinicalTrials.gov ↗ ← All trials in Italy