Spinocerebellar ataxia type 7 (SCA7) MedDRA version: 20.0 Level: SOC Classification code 10029205 Term: Nervous system disorders System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Glentek 50 mg film-coated tablets
Product Name: Glentek 50 mg compresse rivestite con film
Product Code: [Glentek 50 mg compresse rivestite con film]
Pharmaceutical Form: Coated tablet
INN
Sponsors
DIPARTIMENTO DI NEUROSCIENZE SALUTE MENTALE E ORGANI DI SENSO - NESMOS - SAPIENZA UNIVERSITÀ DI ROMA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Male and female of any race and > 6 years old; 2) Positive genetic test for SCA7; 3) Signed Informed Consent (in case of minors, written informed consent must be obtained by parents or legal representative). Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 29 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1) 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. 2.) 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; 3) Hepatic diseases with serum values of alanine aminotransferase, aspartate aminotransferase or bilirubin > 1·5 times above normal limit 4) 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; 5)Known hypersensitivity to any component of riluzole (Glentek).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To compare the two study arms for the proportion of patients who remain stable at SARA score and visual acuity at 18 months respect to run-in.;Secondary Objective: To evaluate the effect of riluzole on visual function using quantitative ophthalmologic assessments and on SARA score, as continuous values, assessing changes at 18 months compared to run-in. To investigate the safety and tolerability of riluzole administered in SCA 7 patients.;Primary end point(s): 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 .;Timepoint(s) of evaluation of this end point: Months: 0, 3, 6, 18 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 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; The safety profile will be assessed through the recording, reporting and analyzing of baseline medical conditions, adverse events, physical examination findings including laboratory tests.;Timepoint(s) of evaluation of this end point: Months: 0, 3, 6, 18; Months: 3, 6, 9, 12, 15, 18 | — |
Countries
Italy, United States
Contacts
Public ContactDipartimento NESMOS
Università La Sapienza
Outcome results
None listed