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

June 25, 2026 updated by: Arrowhead Pharmaceuticals

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
39

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2170
        • Research Site 8
    • Victoria
      • Melbourne, Victoria, Australia, 3004
        • Research Site 7
  • Canada
      • Edmonton, Canada, 8440
        • Research Site 9
    • Quebec
      • Montreal, Quebec, Canada, H2X 0C1
        • Research Site 2
      • Montreal, Quebec, Canada, H3A 2B4
        • Research Site 1
  • France
      • Paris, France, 75651
        • Research Site 15
  • Germany
    • Baden-Wurttemberg
      • Tübingen, Baden-Wurttemberg, Germany, 72026
        • Research Site 13
      • Ulm, Baden-Wurttemberg, Germany, 89081
        • Research Site 14
  • Italy
      • Milan, Italy, 20133
        • Research Site 16
  • New Zealand
      • Auckland, New Zealand, 0622
        • Research Site 4
      • Christchurch, New Zealand, 8011
        • Research Site 3
  • Spain
      • Barcelona, Spain, 08035
        • Research Site 10
      • Barcelona, Spain, 08036
        • Research Site 11
      • Seville, Spain, 41013
        • Research Site 12
  • Taiwan
      • Kaohsiung City, Taiwan, 833401
        • Research Site 5
      • Taipei, Taiwan, 112201
        • Research Site 6

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

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

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Placebo Comparator: Placebo
(0.9% NaCl)
Drug: Placebo
calculated volume to match active treatment by IT administration
Experimental: ARO-ATXN2
ARO-ATXN2 Injection
Drug: ARO-ATXN2 Injection
single dose of ARO-ATXN2 by intrathecal (IT) administration

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time
Time Frame: Through End of Study (EOS), Day 253
Through End of Study (EOS), Day 253

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Pharmacokinetics (PK) of ARO-ATXN2: Maximum Observed Plasma Concentration (Cmax)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
PK of ARO-ATXN2: Time to Maximum Observed Plasma Concentration (Tmax)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
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
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)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
PK of ARO-ATXN2: Elimination Half-life (t1/2)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
PK of ARO-ATXN2: Apparent Systemic Clearance (CL/F)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
PK of ARO-ATXN2: Recovery of Unchanged Drug Excreted in Urine (Ae)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
PK of ARO-ATXN2: Renal Clearance (CLr)
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
Percentage of Administered Drug Recovered in Urine
Time Frame: Through 24 hours post-dose
Through 24 hours post-dose
Change from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over Time
Time Frame: Baseline through End of Study (EOS), Day 253
Baseline through End of Study (EOS), Day 253
Change from Baseline in Glucose in CSF Over Time
Time Frame: Baseline through End of Study (EOS), Day 253
Baseline through End of Study (EOS), Day 253
Change from Baseline in Cell Count in CSF Over Time
Time Frame: Baseline through End of Study (EOS), Day 253
Baseline through End of Study (EOS), Day 253

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Arrowhead Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 17, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 1, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 1, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 4, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 29, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 25, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AROATXN2-1001
  • UTN U111-1308 5875 (Other Identifier: WHO)
  • 2024-514763-25 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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