Spinocerebellar Ataxia Type 2Phase 1NCT06672445
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
A Phase 1 study testing ARO-ATXN2 Injection in people with Spinocerebellar Ataxia Type 2, run by Arrowhead Pharmaceuticals at 16 sites in 8 countries.
Trial 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)… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Spinocerebellar Ataxia Type 2
- What is being tested?
- ARO-ATXN2 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 39 participants
- Where
- 16 sitesin 8 countries
- Study dates
- 17 Dec 2024to Dec 2026
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
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- 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.
Trial locations and contacts
16 sites in 8 countries.
16 sites in 8 countries
Canada3 sites
Research Site 9
Edmonton, 8440
Research Site 2
Montreal, Quebec, H2X 0C1
Research Site 1
Montreal, Quebec, H3A 2B4
Spain3 sites
Research Site 10
Barcelona, 08035
Research Site 11
Barcelona, 08036
Research Site 12
Seville, 41013
Australia2 sites
Research Site 7
Melbourne, Victoria, 3004
Research Site 8
Sydney, New South Wales, 2170
Germany2 sites
Research Site 13
Tübingen, Baden-Wurttemberg, 72026
Research Site 14
Ulm, Baden-Wurttemberg, 89081
New Zealand2 sites
Research Site 4
Auckland, 0622
Research Site 3
Christchurch, 8011
Taiwan2 sites
Research Site 5
Kaohsiung City, 833401
Research Site 6
Taipei, 112201
About this study
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
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
- Being tested
- ARO-ATXN2 Injection, Placebo
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ARO-ATXN2 InjectionDrug
single dose of ARO-ATXN2 by intrathecal (IT) administration
PlaceboDrug
calculated volume to match active treatment by IT administration
| Study group | Role | What they receive |
|---|---|---|
| ARO-ATXN2 | Experimental | ARO-ATXN2 Injection Receives: Drug: ARO-ATXN2 Injection |
| Placebo | Placebo comparator | (0.9% NaCl) Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time
Measured: Through End of Study (EOS), Day 253
Secondary outcomes (13)
Pharmacokinetics (PK) of ARO-ATXN2: Maximum Observed Plasma Concentration (Cmax)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Time to Maximum Observed Plasma Concentration (Tmax)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Measured: 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)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Elimination Half-life (t1/2)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Apparent Systemic Clearance (CL/F)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Recovery of Unchanged Drug Excreted in Urine (Ae)
Measured: Through 24 hours post-dose
PK of ARO-ATXN2: Renal Clearance (CLr)
Measured: Through 24 hours post-dose
Percentage of Administered Drug Recovered in Urine
Measured: Through 24 hours post-dose
Change from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over Time
Measured: Baseline through End of Study (EOS), Day 253
Change from Baseline in Glucose in CSF Over Time
Measured: Baseline through End of Study (EOS), Day 253
Change from Baseline in Cell Count in CSF Over Time
Measured: Baseline through End of Study (EOS), Day 253
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Nov 2024
- Study start17 Dec 2024
- Primary completionDec 2026 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Type 2Studies of the same condition, open and under way.
- More trials run by Arrowhead PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arrowhead Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06672445
- Sponsor's protocol ID
- AROATXN2-1001
- Other IDs
- UTN U111-1308 5875, 2024-514763-25
- Status last verified
- Jun 2026
- First submitted
- 1 Nov 2024
- Last updated
- 29 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No