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

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

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.

Study Overview

Status

Completed

Intervention / Treatment

Detailed Description

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.

Study Type

Interventional

Enrollment (Actual)

42

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Paris, France, 75013
        • Durr

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RILUZOLE
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.
50 mg will be administered (per os) twice a day
Placebo Comparator: PLACEBO
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
50 mg will be administered (per os) twice a day

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
at 12 months.

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
at 12 months
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
at 12 months
12 months survival
Time Frame: at 12 months
To compare survival of the patients between the two treatment groups at 12 months
at 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: DURR Alexandra, PU-PH, Assistance Publique Hopitaux de Paris

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 17, 2018

Primary Completion (Actual)

December 14, 2020

Study Completion (Actual)

December 14, 2020

Study Registration Dates

First Submitted

September 21, 2017

First Submitted That Met QC Criteria

November 17, 2017

First Posted (Actual)

November 20, 2017

Study Record Updates

Last Update Posted (Actual)

June 21, 2021

Last Update Submitted That Met QC Criteria

June 18, 2021

Last Verified

June 1, 2021

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Spinocerebellar Ataxia Type 2

  • Cadent Therapeutics
    Withdrawn
    Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8 | Spinocerebellar Ataxia Type 17 | ARCA1 - Autosomal Recessive...
    United States
  • Biohaven Pharmaceuticals, Inc.
    Active, not recruiting
    Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxias | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 10 | Spinocerebellar Ataxia Type 7 | Spinocerebellar Ataxia Type 8
    United States, China
  • University of Florida
    Acorda Therapeutics
    Completed
    Spinocerebellar Ataxias Type 1 | Spinocerebellar Ataxias Type 2 | Spinocerebellar Ataxias Type 3 | Spinocerebellar Ataxias Type 6
    United States
  • Biohaven Pharmaceuticals, Inc.
    Active, not recruiting
    Spinocerebellar Ataxias | Spinocerebellar Ataxia Genotype Type 1 | Spinocerebellar Ataxia Genotype Type 2 | Spinocerebellar Ataxia Genotype Type 3 | Spinocerebellar Ataxia Genotype Type 6 | Spinocerebellar Ataxia Genotype Type 7 | Spinocerebellar Ataxia Genotype Type 8 | Spinocerebellar Ataxia Genotype...
    United States
  • Sclnow Biotechnology Co., Ltd.
    Not yet recruiting
    Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6
  • Teachers College, Columbia University
    Active, not recruiting
    Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | Spinocerebellar Ataxia Type 7
    United States
  • University of California, Los Angeles
    Active, not recruiting
    Spinocerebellar Ataxias | Spinocerebellar Ataxia 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6 | MSA-C
    United States
  • University of Chicago
    Pfizer; Biogen; APDM Wearable Technologies
    Active, not recruiting
    Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6
    United States
  • University of Florida
    University of California, Los Angeles; National Ataxia Foundation
    Recruiting
    Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia Type 6
    United States
  • Assistance Publique - Hôpitaux de Paris
    Completed
    Spinocerebellar Ataxia Type 1 | Spinocerebellar Ataxia Type 2 | Spinocerebellar Ataxia, Autosomal Recessive 3 | Episodic Ataxia, Type 7
    France

Clinical Trials on Riluzole

Subscribe