Using Riluzole to Treat Spinocerebellar Ataxia Type 7
Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in Phase
This study is testing if the medication riluzole can help people with Spinocerebellar Ataxia Type 7 feel better since there are currently no effective treatments for this condition.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 34 (estimated) |
| Ages | 7 Years and up |
| Sex | All |
| Sponsor | S. Andrea Hospital Academic / other |
| Locations | 1 site (Rome) |
| Trial ID | NCT03660917 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the effects of riluzole in patients diagnosed with Spinocerebellar Ataxia Type 7 (SCA7), a rare inherited condition with no current therapeutic options. The study employs a randomized, double-blind, placebo-controlled design, beginning with a 6-month run-in phase to assess baseline ophthalmological metrics and ataxia scores. Following this, participants will be assigned to receive either riluzole or a placebo for 12 months, with evaluations conducted at specified intervals to measure treatment effects and safety. The trial aims to provide insights into the potential efficacy of riluzole for this condition based on previous encouraging data.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals aged 7 years and older with a confirmed genetic diagnosis of SCA7.
Not a fit: Patients with serious systemic illnesses or conditions that may exacerbate the side effects of riluzole, as well as those who are pregnant or breastfeeding, are unlikely to benefit from this study.
Why it matters
Potential benefit: If successful, this trial could provide a new therapeutic option for patients suffering from SCA7, potentially improving their quality of life.
How similar studies have performed: Previous studies have shown promising results with riluzole in other inherited cerebellar ataxias, suggesting potential efficacy in SCA7, although this specific application is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * positive genetic test for SCA7. Exclusion Criteria: * cardiac arrhythmias; * haematologic diseases; * hepatic diseases with serum values of alanine aminotransferase, aspartate aminotransferase or bilirubin \> 1·5 times above normal limit; * pregnancy (women of childbearing potential agreed to use contraception); * breastfeeding.
Where this trial is running
Rome
- Neurological Unit, S. Andrea Hospital, Faculty of Medicine and Psychology, "Sapienza" University of Rome — Rome, Italy (Recruiting)
Study contacts
- Study coordinator: Silvia Romano, MD, PhD
- Email: silvia.romano@uniroma1.it
- Phone: +390633776044
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.