RILUZOLE for Spinocerebellar ataxia type 7

Inclusion criteria

• {"criterion_text":"- Male and female of any race and > 6 years old"}
• {"criterion_text":"- Positive genetic test for SCA7"}
• {"criterion_text":"- Signed Informed Consent (in case of minors, written informed consent must be obtained by parents or legal representative)"}

Exclusion criteria

• {"criterion_text":"- Female subjects: pregnant or lactating women cannot participate in the study. Women of childbearing potential cannot participate unless willing to use highly effective contraception methods as combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomised partner; sexual abstinence. In case of use of oral contraception, women should have been stable on the same pill for a minimum of 3 months before taking study drug. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception."}
• {"criterion_text":"- Subjects with a clinically significant or unstable medical or surgical condition that would preclude safe and complete study participation. Such conditions may include cardiovascular, pulmonary, hepatic, renal, severe systemic mycotic infections, metabolic diseases or malignancies"}
• {"criterion_text":"- Hepatic diseases with serum values of alanine aminotransferase, aspartate aminotransferase or bilirubin > 1·5 times above normal limit"}
• {"criterion_text":"- Any medical or psychiatric condition that may affect the subject ability to give informed consent, or to complete the study, or if the subject is considered by the treating neurologist to be, for any other reason, an unsuitable candidate for this study"}
• {"criterion_text":"- Known hypersensitivity to any component of riluzole (Glentek)"}

Primary endpoints

• {"endpoint_text":"- The primary endpoints will be the proportion of patients with stable SARA score and visual acuity expressed as log MAR units at 18 months, in comparison with the same parameters as mean of t0-t3-t6 evaluations .","definition_or_measurement_approach":"Proportion of patients with stability on SARA score and visual acuity measured in logMAR at 18 months compared to the mean of baseline/run-in evaluations at t0, t3, and t6."}

Secondary endpoints

• {"endpoint_text":"- The secondary endpoint will be quantitative ophthalmologic assessments (via a Farnsworth D15 Arrangement Test, Visual evoked, Electroretinography, Optical Coherence tomography, Computerized visual field examination) and SARA score as continuous values at 18 months, in comparison with the same parameters calculated for each patient as mean of t0-t3-t6 evaluations","definition_or_measurement_approach":"Quantitative ophthalmologic assessments including Farnsworth D15 Arrangement Test, Visual evoked potentials, Electroretinography, Optical Coherence Tomography (OCT), and computerized visual field; and SARA score treated as continuous variables at 18 months versus mean of t0, t3, t6."}
• {"endpoint_text":"- The safety profile will be assessed through the recording, reporting and analyzing of baseline medical conditions, adverse events, physical examination findings including laboratory tests.","definition_or_measurement_approach":"Safety assessed by recording and analysis of baseline medical conditions, adverse events, physical exam findings and laboratory test results."}

Methods

• Site-based recruitment through participating neurology and pediatric clinics at listed hospitals/universities in Italy (University Hospital Of Ferrara; Universita Degli Studi Di Roma La Sapienza; Azienda Ospedaliera Universitaria Gaetano Martino Messina; Azienda Unita Sanitaria Locale Di Bologna).
• GP outreach: L2_1 RISCA7 GP Letter (document present) to inform general practitioners about the study (title indicates targeted recruitment via GPs).
• Pediatrician outreach: L2_2 RISCA7 Letter for pediatrician (document present) to inform pediatricians about the study (targeted recruitment via pediatricians).

Italy

Earliest CTIS Part Ii Submission Date

22-10-2024

Latest Decision Or Authorization Date

27-10-2025

Processing Time Days

370

Number Of Sites

4

Number Of Participants

24

Primary sponsor

Full Name

Universita Degli Studi Di Roma La Sapienza

Organisation Type

Educational Institution

Country Of Registered Address

Italy