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NCT Number: NCT06672445

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Spinocerebellar Ataxia Type 2 Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Ataxias Spinocerebellar Degenerations

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Research Site 8, Sydney, New South Wales, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

  • 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

Note: Additional inclusion/exclusion criteria may apply per protocol.

Treatment and study plan

ARO-ATXN2 Injection

Drug

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

Placebo

Drug

calculated volume to match active treatment by IT administration

Primary outcomes

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

    Time frame: Through End of Study (EOS), Day 253

Secondary outcomes

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

    Time frame: Through 24 hours post-dose

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

    Time frame: Through 24 hours post-dose

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

    Time frame: Through 24 hours post-dose

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

    Time frame: Through 24 hours post-dose

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

    Time frame: Through 24 hours post-dose

  6. PK of ARO-ATXN2: Elimination Half-life (t1/2)

    Time frame: Through 24 hours post-dose

  7. PK of ARO-ATXN2: Apparent Systemic Clearance (CL/F)

    Time frame: Through 24 hours post-dose

  8. PK of ARO-ATXN2: Recovery of Unchanged Drug Excreted in Urine (Ae)

    Time frame: Through 24 hours post-dose

  9. PK of ARO-ATXN2: Renal Clearance (CLr)

    Time frame: Through 24 hours post-dose

  10. Percentage of Administered Drug Recovered in Urine

    Time frame: Through 24 hours post-dose

  11. Change from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over Time

    Time frame: Baseline through End of Study (EOS), Day 253

  12. Change from Baseline in Glucose in CSF Over Time

    Time frame: Baseline through End of Study (EOS), Day 253

  13. Change from Baseline in Cell Count in CSF Over Time

    Time frame: Baseline through End of Study (EOS), Day 253

Sponsors and collaborators

Lead sponsor

Arrowhead Pharmaceuticals

Industry

Registry information

Official study 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

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Nov 4, 2024
Registry last updated
Jun 29, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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