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 Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
36
single dose of ARO-ATXN2 by intrathecal (IT) administration
calculated volume to match active treatment by IT administration
Research Site 8
Sydney, New South Wales, Australia
RECRUITINGResearch Site 7
Melbourne, Victoria, Australia
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time
Time frame: Through End of Study (EOS), Day 253
Pharmacokinetics (PK) of ARO-ATXN2: Maximum Observed Plasma Concentration (Cmax)
Time frame: Through 24 hours post-dose
PK of ARO-ATXN2: Time to Maximum Observed Plasma Concentration (Tmax)
Time frame: 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
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
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Time frame: Through 24 hours post-dose
PK of ARO-ATXN2: Elimination Half-life (t1/2)
Time frame: Through 24 hours post-dose
PK of ARO-ATXN2: Apparent Systemic Clearance (CL/F)
Time frame: Through 24 hours post-dose
PK of ARO-ATXN2: Recovery of Unchanged Drug Excreted in Urine (Ae)
Time frame: Through 24 hours post-dose
PK of ARO-ATXN2: Renal Clearance (CLr)
Time frame: Through 24 hours post-dose
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Research Site 2
Montreal, Quebec, Canada
RECRUITINGResearch Site 1
Montreal, Quebec, Canada
RECRUITINGResearch Site 9
Edmonton, Canada
RECRUITINGResearch Site 15
Paris, Paris, France
RECRUITINGResearch Site 13
Tübingen, Baden-Wurttemberg, Germany
RECRUITINGResearch Site 14
Ulm, Baden-Wurttemberg, Germany
RECRUITINGResearch Site 16
Milan, Italy
RECRUITINGResearch Site 4
Auckland, New Zealand
RECRUITING...and 6 more locations
Percentage of Administered Drug Recovered in Urine
Time frame: 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
Change from Baseline in Glucose in CSF Over Time
Time frame: 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