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Completed

NCT Number: NCT03347344

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

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.

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Key information

Conditions

Spinocerebellar Ataxia Type 2 Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Ataxia Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Nervous System Diseases Neurodegenerative Diseases Neurologic Manifestations Spinal Cord Diseases Spinocerebellar Ataxias Spinocerebellar Degenerations

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Durr

Paris, 75013, France

About this study

Inherited cerebellar ataxias are genetically heterogeneous neurological disorders. They are characterized by ataxic gait and cerebellar dysarthria that progresses over time with loss of ambulation and speech. The mutations by expansions of CAG triplets in the genes ATXN1 (SCA1), ATXN 2 (SCA2), 3 (SCA3), CACNA1A (SCA6), ATXN 7 (SCA7), and TBP (SCA17) are responsible for 50% of hereditary forms There is no curative or preventive treatment. This phase III study is a multi-centric, double-blind randomized, two-arm controlled study (one group with 50 mg Riluzole twice a day versus one group with placebo), to measure the efficacy of treatment with riluzole in SCA2 patients during 12 months. Amelioration is defined by a 1 point decrease of the SARA score.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Riluzole

Drug

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

Placebo

Drug

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

Primary outcomes

  1. Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA))

    Time frame: at 12 months.

    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

Secondary outcomes

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

    Time frame: at 12 months

    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.

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

    Time frame: at 12 months

    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

  3. 12 months survival

    Time frame: at 12 months

    To compare survival of the patients between the two treatment groups at 12 months

Sponsors and collaborators

Lead sponsor

Assistance Publique - Hôpitaux de Paris

Other

Registry information

Official study title

Multicenter, Randomized, Double Blind, Placebo Controlled Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2

Acronym: ATRIL

Important dates

Study start
2018
Primary completion
2020
Study completion
2020
First posted
Nov 20, 2017
Registry last updated
Jun 21, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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