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

Assistance Publique - Hôpitaux de Paris

Status and phase: Completed; Phase 3

Conditions: Spinocerebellar Ataxia Type 2

Treatments: Drug: Placebo; Drug: Riluzole

Study type: Interventional

Funder types: Other

Identifiers

NCT03347344

2017-001481-23 (EudraCT Number)

P160927J

Details and patient eligibility

About

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.

Full 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.

Enrollment: 42 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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42 participants in 2 patient groups, including a placebo group

RILUZOLE

Experimental group

Description:

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.

Treatment:

Drug: Riluzole

PLACEBO

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo