Trial Review

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06672445




Registration number
NCT06672445
Ethics application status
Date submitted
1/11/2024
Date registered
4/11/2024
Date last updated
27/11/2024

Titles & IDs
Public title
Study of ARO-ATXN2 Injection in Adults With Spinocerebellar Ataxia Type 2
Scientific 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
Secondary ID [1] 0 0
UTN U111-1308 5875
Secondary ID [2] 0 0
AROATXN2-1001
Universal Trial Number (UTN)
UTN U111-1308 5875
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Spinocerebellar Ataxia Type 2 0 0
Condition category
Condition code
Neurological 0 0 0 0
Other neurological disorders
Neurological 0 0 0 0
Neurodegenerative diseases
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ARO-ATXN2 Injection
Treatment: Drugs - Placebo

Experimental: ARO-ATXN2 - ARO-ATXN2 Injection

Placebo comparator: Placebo - (0.9% NaCl)


Treatment: Drugs: ARO-ATXN2 Injection
single dose of ARO-ATXN2 by intrathecal (IT) administration

Treatment: Drugs: Placebo
calculated volume to match active treatment by IT administration
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) Over Time
Timepoint [1] 0 0
Through End of Study (EOS), Day 253
Secondary outcome [1] 0 0
Pharmacokinetics (PK) of ARO-ATXN2: Maximum Observed Plasma Concentration (Cmax)
Timepoint [1] 0 0
Through 24 hours post-dose
Secondary outcome [2] 0 0
PK of ARO-ATXN2: Time to Maximum Observed Plasma Concentration (Tmax)
Timepoint [2] 0 0
Through 24 hours post-dose
Secondary outcome [3] 0 0
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Timepoint [3] 0 0
Through 24 hours post-dose
Secondary outcome [4] 0 0
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quatifiable Plasma Concentration (AUClast)
Timepoint [4] 0 0
Through 24 hours post-dose
Secondary outcome [5] 0 0
PK of ARO-ATXN2: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Timepoint [5] 0 0
Through 24 hours post-dose
Secondary outcome [6] 0 0
PK of ARO-ATXN2: Elimination Half-life (t1/2)
Timepoint [6] 0 0
Through 24 hours post-dose
Secondary outcome [7] 0 0
PK of ARO-ATXN2: Apparent Systemic Clearance (CL/F)
Timepoint [7] 0 0
Through 24 hours post-dose
Secondary outcome [8] 0 0
PK of ARO-ATXN2: Recovery of Unchanged Drug Excreted in Urine (Ae)
Timepoint [8] 0 0
Through 24 hours post-dose
Secondary outcome [9] 0 0
PK of ARO-ATXN2: Renal Clearance (CLr)
Timepoint [9] 0 0
Through 24 hours post-dose
Secondary outcome [10] 0 0
Percentage of Administered Drug Recovered in Urine
Timepoint [10] 0 0
Through 24 hours post-dose
Secondary outcome [11] 0 0
Change from Baseline in Total Protein in Cerebral Spinal Fluid (CSF) Over Time
Timepoint [11] 0 0
Baseline through End of Study (EOS), Day 253 or until ATXN2 protein levels are = 50% of predose Baseline
Secondary outcome [12] 0 0
Change from Baseline in Glucose in CSF Over Time
Timepoint [12] 0 0
Baseline through End of Study (EOS), Day 253 or until ATXN2 protein levels are = 50% of predose Baseline
Secondary outcome [13] 0 0
Change from Baseline in Cell Count in CSF Over Time
Timepoint [13] 0 0
Baseline through End of Study (EOS), Day 253 or until ATXN2 protein levels are = 50% of predose Baseline

Eligibility
Key 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
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/11/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2026
Actual
Sample size
Target
36
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Arrowhead Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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) and pharmacodynamic (PD) parameters.
Trial website
https://clinicaltrials.gov/study/NCT06672445
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Monitor
Address 0 0
Country 0 0
Phone 0 0
626-304-3400
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT06672445