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

NCT06672445 | Recruiting Phase 1 DMC FDA Drug

• 3 other identifiers: AROATXN2-1001UTN U111-1308 58752024-514763-25
• Study Type: interventional
• Target: 36
• Locations: 8 countries
• Sites: 16

Brief 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.

Conditions

Spinocerebellar Ataxia Type 2

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time

  Through End of Study (EOS), Day 253

Secondary Outcomes (13)

• Pharmacokinetics (PK) of ARO-ATXN2: Maximum Observed Plasma Concentration (Cmax)

  Through 24 hours post-dose

• PK of ARO-ATXN2: Time to Maximum Observed Plasma Concentration (Tmax)

  Through 24 hours post-dose

• PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)

  Through 24 hours post-dose

• PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quatifiable Plasma Concentration (AUClast)

  Through 24 hours post-dose

• PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)

  Through 24 hours post-dose

Study Arms (2)

ARO-ATXN2

EXPERIMENTAL

ARO-ATXN2 Injection

Drug: ARO-ATXN2 Injection

Placebo

PLACEBO COMPARATOR

(0.9% NaCl)

Drug: Placebo

Interventions

Placebo DRUG

calculated volume to match active treatment by IT administration

Placebo

ARO-ATXN2 Injection DRUG

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

ARO-ATXN2

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Non-pregnant, non-lactating
• 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
• 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

You may not qualify if:

• Uncontrolled hypertension (blood pressure \>160/100 mmHg)
• History of having received stem cell therapy
• Clinically significant cardiac, liver, or renal disease
• Human immunodeficiency virus (HIV) infection (seropositive at Screening)
• Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening
• Intellectual disability or significant behavioral neuropsychiatric manifestation
• 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
• Presence of an implanted shunt for drainage of CSF or an implanted central nervous system (CNS) catheter

Sponsors & Collaborators

• Arrowhead Pharmaceuticals: lead

Study Sites (16)

Research Site 8

Sydney, New South Wales, 2170, Australia

RECRUITING

Research Site 7

Melbourne, Victoria, 3004, Australia

RECRUITING

Research Site 2

Montreal, Quebec, H2X 0C1, Canada

RECRUITING

Research Site 1

Montreal, Quebec, H3A 2B4, Canada

RECRUITING

Research Site 9

Edmonton, 8440, Canada

RECRUITING

Research Site 15

Paris, Paris, 75651, France

RECRUITING

Research Site 13

Tübingen, Baden-Wurttemberg, 72026, Germany

RECRUITING

Research Site 14

Ulm, Baden-Wurttemberg, 89081, Germany

RECRUITING

Research Site 16

Milan, 20133, Italy

RECRUITING

Research Site 4

Auckland, 0622, New Zealand

RECRUITING

Research Site 3

Christchurch, 8011, New Zealand

RECRUITING

Research Site 10

Barcelona, 08035, Spain

RECRUITING

Research Site 11

Barcelona, 08036, Spain

RECRUITING

Research Site 12

Seville, 41013, Spain

RECRUITING

Research Site 5

Kaohsiung City, 833401, Taiwan

RECRUITING

Research Site 6

Taipei, 112201, Taiwan

RECRUITING

MeSH Terms

Conditions

Spinocerebellar Ataxias

Condition Hierarchy (Ancestors)

Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Medical Monitor

CONTACT

626-304-3400; AROATXN2@arrowheadpharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 1, 2024
• First Posted: November 4, 2024
• Study Start: December 17, 2024
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: February 6, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

CA (3); IT (1); AU (2); DE (2); FR (1); TW (2); NZ (2); ES (3)