AROATXN2-1001
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
The purpose of this study is to evaluate ARO-ATXN2, a potential treatment for SCA2 developed by Arrowhead Pharmaceuticals and Sarepta Therapeutics, Inc. ARO-ATXN2 is a medication designed to reduce the level of the Ataxin-2 protein, which has been implicated in the development of SCA2. This study will evaluate the safety of ARO-ATXN2, its metabolism/elimination, and its effects on the body.
ClinicalTrials.gov Link: https://clinicaltrials.gov/study/NCT06672445
Disclaimer: This research opportunity is not affiliated with NAF. We are sharing this information as a resource for the Ataxia community. Sharing this information does not imply endorsement by NAF.
About the Study
Eligible Ataxia Types
Adult patients with a diagnosis of symptomatic SCA2 and a ≥33 CAG repeats in the ATXN2 gene.
Type of Study
Research Study
Clinical Trial Phase
Phase 1
Age Range
Adult 18+
Study Start Date
12/17/2024
Estimated Completion Date
11/11/2026
IRB Approval #
N/A
Study Sponsor
Sarepta Therapeutics, Inc
Contact Information
ClinicalTrials.gov Link: https://clinicaltrials.gov/study/NCT06672445
What does participation in the study entail?
Eligible participants will receive a single intrathecal injection of ARO-ATXN2 or placebo. This is an injection into the cerebrospinal fluid via a lumbar puncture procedure. An overnight stay at the study center for 1 night is required. Additionally, there will be 12 study center visits for study-related assessments, including additional lumbar punctures. The study will last approximately 44 weeks.
What are the potential benefits for participants?
This is the first time ARO-ATXN2 is being tested in humans. It is hoped that ARO-ATXN2 will reduce the levels of the Ataxin-2 protein and that this may help slow the progression of SCA2, however, there is no guarantee of benefit, and you may or may not receive any direct benefit from your participation.
What are the potential risks for participants?
The risks of participation will be discussed in detail by your study physician during the Informed Consent process.
Is there financial compensation?
Yes. Varies by site/region.
Is there travel reimbursement?
Yes. Varies by site/region.
Who is eligible?
Adult patients with a diagnosis of symptomatic SCA2 and a ≥33 CAG repeats in the ATXN2 gene.
Additional Information or Resources
Learn more: https://clinicaltrials.gov/study/NCT06672445
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