Recruiting

ARO-ATXN2 for Spinocerebellar Ataxia Type 2 with CAG Repeats

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What is being tested

ARO-ATXN2 Injection

+ Placebo

Drug
Who is being recruted

Ataxia+12

+ Brain Diseases

+ Central Nervous System Diseases

From 18 to 65 Years
+12 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Placebo-ControlledPhase 1
Interventional
Study Start: December 2024
See protocol details

Summary

Principal SponsorArrowhead Pharmaceuticals
Study ContactMedical Monitor
Last updated: February 7, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: December 17, 2024

Actual date on which the first participant was enrolled.

This study aims to evaluate a new treatment called ARO-ATXN2 for adults with spinocerebellar ataxia type 2 (SCA2), a genetic condition that affects coordination and balance. Participants in this study must have a specific genetic marker, which is at least 33 repeats of the CAG sequence in the ATXN2 gene. The study is important as it seeks to understand the safety and effects of ARO-ATXN2, which could lead to new treatment options for those affected by this challenging condition. Participants will be randomly assigned to receive either a single dose of ARO-ATXN2 or a placebo, which is an inactive substance. The study will monitor how the body processes ARO-ATXN2 and how it affects the body, along with checking for any side effects. This will help researchers determine whether the treatment is safe and well-tolerated, laying the groundwork for future research and potential therapies.

Official TitleA 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
NCT06672445
Principal SponsorArrowhead Pharmaceuticals
Study ContactMedical Monitor
Last updated: February 7, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

36 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 65 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

AtaxiaBrain DiseasesCentral Nervous System DiseasesCerebellar AtaxiaCerebellar DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsSpinal Cord DiseasesSpinocerebellar DegenerationsNeurodegenerative DiseasesHeredodegenerative Disorders, Nervous SystemSpinocerebellar AtaxiasDyskinesiasGenetic Diseases, Inborn

Criteria

4 inclusion criteria required to participate
Diagnosis of symptomatic SCA2 and ≥33 CAG repeats in the ATXN2 gene based on source verifiable medical records or genetic testing at Screening
Scale of Assessment and Rating of Ataxia (SARA) score ≤14
Non-pregnant, non-lactating
Subjects of childbearing potential must agree to use highly effective contraception in addition to a condom during the study and for at least 90 days following the end of the study or last dose of study drug, whichever is later. Subjects must not donate sperm or eggs during the study and for at least 90 days following the end of the study or last dose of study drug whichever is later
8 exclusion criteria prevent from participating
Uncontrolled hypertension (blood pressure >160/100 mmHg)
Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening
Human immunodeficiency virus (HIV) infection (seropositive at Screening)
Any contraindications to lumbar puncture, including INR >1.4, platelet count <100,000, and use of anticoagulant or antiplatelet medications that cannot be safely interrupted
Show More Criteria

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

2 intervention groups are designated in this study

50% chance of being blinded to the placebo group

Treatment Groups

Group I

Experimental
ARO-ATXN2 Injection

Group II

Placebo
(0.9% NaCl)

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 16 locations

Recruiting

Research Site 8

Sydney, AustraliaOpen Research Site 8 in Google Maps
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Research Site 7

Melbourne, Australia
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Research Site 2

Montreal, Canada
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Research Site 1

Montreal, Canada
Recruiting
16 Study Centers