Patient Information
This brochure provides etails about the AROATXN2-1001 Study for individuals with spinocerebellar ataxia type 2 (SCA2)d. It aims to help potential participants decide whether they or someone they know may want to join the study.
Eligibility Criteria
You may be eligible to participate if you:
- Are between 18 and 65 years old.
- Have been diagnosed with SCA2.
- Can walk without assistance for 8 meters.
Considerations
- The study team will explain the potential benefits and risks.
- Participation is voluntary, and you can withdraw at any time.
- Study-related travel costs, medications, and tests will be provided at no cost.
- Your health will be closely monitored by a team of doctors and nurses.
About Clinical Research Studies
Clinical research studies answer questions about investigational drugs, such as their safety and effectiveness. All medications must be researched in people before approval for prescription. An Institutional Review Board (IRB)/Ethics Committee (EC) has reviewed this study to protect participants’ rights, safety, and well-being.
About the AROATXN2-1001 Study
Current therapies for SCA2 are supportive and aim to ease symptoms, but there are no treatments that delay, slow, or reverse the condition. This study will investigate a potential new medication that may lower the amount of a protein called ATXN2 in the body, potentially stopping or slowing disease progression. The study will assess:
- The safety of the study drug.
- Possible side effects.
- The most suitable dose for further research.
Study Design
- Up to 36 participants will be involved.
- Participants will receive either the study drug at one of four dose levels or a placebo.
- The study drug or placebo will be administered once as an injection in the fluid around the spine.
- Participants will undergo five lumbar punctures and two MRI brain scans.
- The study includes an overnight stay at the study center and 12 visits for assessments.
Study Periods
- Screening Period (up to 2 months): At least one visit to determine eligibility and willingness to participate.
- Study Treatment Period (about 24 hours, overnight): Administration of the study medication and assessments.
- Follow-up Period (about 8 months): Nine visits for assessments, with potential extension for further visits every two months.
Contact Information
For more details or to express interest in participating, contact the study team. There is no obligation to join the study by reaching out.
Investigator: Dr Antoine Duquette, CHUM
Study coordinator: Martine Comeau
E-mail address: martine.comeau.chum@ssss.gouv.qc.ca
Telephone: 514-890-8000, ext. 30154
Investigator Dr Massimo Pandolfo, Neuro, McGill
Study coordinator: Romina Perrotti romina.perrotti@mcgill.ca
Téléphone:514-398-2964.