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Trial record 3 of 315 for:    Ataxia

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

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ClinicalTrials.gov Identifier: NCT03347344
Recruitment Status : Not yet recruiting
First Posted : November 20, 2017
Last Update Posted : November 20, 2017
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

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.


Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxia Type 2 Drug: Riluzole Drug: Placebo Phase 3

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  (Clinical Trial)
Estimated Enrollment : 42 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: 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
Masking: Triple (Participant, Care Provider, Investigator)
Masking Description:

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

Primary Purpose: Treatment
Official Title: Multicenter, Randomized, Double Blind, Placebo Controlled Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2
Estimated Study Start Date : November 2017
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : November 2019


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.
Drug: Riluzole
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
Drug: Placebo
50 mg will be administered (per os) twice a day




Primary Outcome Measures :
  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 Outcome Measures :
  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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03347344


Contacts
Contact: DURR Alexandra, PU-PH 01 57 27 46 87 ext 0033 alexandra.durr@icm-institute.org
Contact: PETIT Elodie 01 57 27 45 31 ext 0033 elodie.petit@icm-institute.org

Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: DURR Alexandra, PU-PH ASSISTANCE PUBLIQUE HÖPITAUX DE PARIS

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT03347344     History of Changes
Other Study ID Numbers: P160927J
2017-001481-23 ( EudraCT Number )
First Posted: November 20, 2017    Key Record Dates
Last Update Posted: November 20, 2017
Last Verified: September 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Spinocerebellar ataxia-SCA2
SARA (Scale for the Assessment and Rating of Ataxia) score
Riluzole (Rilutek)

Additional relevant MeSH terms:
Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Riluzole
Anticonvulsants
Excitatory Amino Acid Antagonists
Excitatory Amino Acid Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Neuroprotective Agents
Protective Agents