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RECRUITING
NCT06672445
PHASE1

Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2

Sponsor: Arrowhead Pharmaceuticals

View on ClinicalTrials.gov

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

Interventions

DRUG

ARO-ATXN2 Injection

single dose of ARO-ATXN2 by intrathecal (IT) administration

DRUG

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