Spinocerebellar Ataxia Type 2 Clinical Trial
— ATRILOfficial title:
Multicenter, Randomized, Double Blind, Placebo Controlled Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2
Verified date | June 2021 |
Source | Assistance Publique - Hôpitaux de Paris |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
ATRIL is a multi-centric, double-blind randomized, two-arm controlled study. 42 SpinoCerebellar Ataxia type 2 (SCA2) patients, both gender, at least 18 years of age will be included. Riluzole 50 mg will be administered (per os) twice a day, versus one group with placebo for 12 months. Riluzole (Rilutek®) is a benzothiazole drug, market approved, for Amyotrophic Lateral Sclerosis (ALS). It delays the onset of ventilator-dependence or tracheostomy in selected patients and may increase survival. Scale for the Assessment and Rating of Ataxia (SARA) will be used at M0, M6 and M12. To assess primary criterion, the percentage of patients with a decrease of at least 1 point of the SARA score between the inclusion visit, and Visit 3 (Months 12) will be calculated.
Status | Completed |
Enrollment | 42 |
Est. completion date | December 14, 2020 |
Est. primary completion date | December 14, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Genetically diagnosed SCA2 (CAG triplet in ATXN2 = 33) - At least 18 years of age - Signature of informed consent - Covered by social security - SARA score = 5 and = 26 - Age at onset = 50 years old Exclusion Criteria: - Treated with riluzole prior to the study - Hepatotoxic medication - Hypersensitivity to the active substance or to any of the excipients - Serious systemic illnesses or conditions known for enhancing the side effects of riluzole - Contraindications for MRI examination - Participation in another therapeutic trial (3 months exclusion period) - Pregnancy or breastfeeding - Non abstinence or absence of effective contraception for women - Inability to understand information about the protocol - Persons deprived of their liberty by judicial or administrative decision - Adult subject under legal protection or unable to consent - Other ataxic syndromes than SCA2 |
Country | Name | City | State |
---|---|---|---|
France | Durr | Paris |
Lead Sponsor | Collaborator |
---|---|
Assistance Publique - Hôpitaux de Paris |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA)) | To compare the proportion of patients with Scale for the Assessment and Rating of Ataxia (SARA) improvement (decrease) of at least one point from baseline to 12 months | at 12 months. | |
Secondary | Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score) | To compare the difference of the CCFS score (Composite Cerebellar Functional Severity Score) from baseline at 12 months. A decrease is expected in the intervention group. | at 12 months | |
Secondary | Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS)) | To compare the difference of the extracerebellar symptoms (INAS, Inventory of Non-Ataxia Signs) by showing decrease in the INAS count from baseline at 12 months | at 12 months | |
Secondary | 12 months survival | To compare survival of the patients between the two treatment groups at 12 months | at 12 months |
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