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

Arrowhead Pharmaceuticals

Status and phase: Enrolling; Phase 1

Conditions: Spinocerebellar Ataxia Type 2

Treatments: Drug: Placebo; Drug: ARO-ATXN2 Injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT06672445

2024-514763-25 (EudraCT Number)

UTN U111-1308 5875 (Other Identifier)

AROATXN2-1001

Details and patient eligibility

About

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.

Enrollment: 36 estimated patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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
• Coagulation parameters within normal ranges at Screening
• 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
• 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 design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

36 participants in 2 patient groups, including a placebo group

ARO-ATXN2

Experimental group

Description:

ARO-ATXN2 Injection

Treatment:

Drug: ARO-ATXN2 Injection

Placebo

Placebo Comparator group

Description:

(0.9% NaCl)

Treatment:

Drug: Placebo