Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)

NCT03347344 | Completed Phase 3

• 2 other identifiers: P160927J2017-001481-23
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Spinocerebellar Ataxia Type 2

Keywords

Spinocerebellar ataxia-SCA2; SARA (Scale for the Assessment and Rating of Ataxia) score; Riluzole (Rilutek)

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (3)

• Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score)

  at 12 months

• Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS))

  at 12 months

• 12 months survival

  at 12 months

Study Arms (2)

RILUZOLE

EXPERIMENTAL

Riluzole PMCS 50 mg is presented as a round, biconvex, 8 mm diameter nearly white film-coated tablet. The tablets will be held under a blister of 20 tablets.

Drug: Riluzole

PLACEBO

PLACEBO COMPARATOR

The placebo PMCS 50 mg is presented as a round, biconvex, 8 mm diameter nearly white film-coated tablet matching the appearance of the Riluzole used in this study

Drug: Placebo

Interventions

Riluzole DRUG

50 mg will be administered (per os) twice a day

RILUZOLE

Placebo DRUG

50 mg will be administered (per os) twice a day

PLACEBO

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Durr

Paris, 75013, France

Location

Related Publications (1)

• Coarelli G, Heinzmann A, Ewenczyk C, Fischer C, Chupin M, Monin ML, Hurmic H, Calvas F, Calvas P, Goizet C, Thobois S, Anheim M, Nguyen K, Devos D, Verny C, Ricigliano VAG, Mangin JF, Brice A, Tezenas du Montcel S, Durr A. Safety and efficacy of riluzole in spinocerebellar ataxia type 2 in France (ATRIL): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2022 Mar;21(3):225-233. doi: 10.1016/S1474-4422(21)00457-9. Epub 2022 Jan 18.

  PMID: 35063116 DERIVED

MeSH Terms

Conditions

Spinocerebellar Ataxias

Interventions

Riluzole

Condition Hierarchy (Ancestors)

Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• DURR Alexandra, PU-PH

  ASSISTANCE PUBLIQUE HÖPITAUX DE PARIS

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Treatments will be presented in numbered boxes, labeled for this study according to the Good Manufacturing Practices by the General Agency of Equipment and Health Products (AGEPS). Each numbered box will consist of 6 months of treatment: 20 blister packs of 20 active or placebo tablets
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Multi-centric, double-blind randomized, two-arm controlled study (one group with 50 mg riluzole twice a day versus one group with placebo), during one year

Study Record Dates

• First Submitted: September 21, 2017
• First Posted: November 20, 2017
• Study Start: January 17, 2018
• Primary Completion: December 14, 2020
• Study Completion: December 14, 2020
• Last Updated: June 21, 2021

Record last verified: 2021-06

Locations

FR (1)