Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Riluzole in Patients With Spinocerebellar Ataxia Type 7

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03660917
Recruitment Status : Not yet recruiting
First Posted : September 7, 2018
Last Update Posted : December 19, 2018
Sponsor:
Information provided by (Responsible Party):
Giovanni Ristori, S. Andrea Hospital

Brief Summary:
Spinocerebellar ataxia type 7 (SCA7) belongs to the dominant forms of inherited cerebellar ataxias (CA), being one of the rarest form. SCA7 has no therapeutic options, so that the relentless course, the important visual deficit that accompanies CA, and the possibility of disease development in childhood are pressing unmet needs. The investigators published encouraging data on riluzole in inherited CA other than SCA7. These results prompted off-label use of riluzole in single cases of SCA7 in Italy and United States, suggesting possible efficacy of the drug in this condition.

Condition or disease Intervention/treatment Phase
SCA7 Drug: Riluzole Drug: Placebo Phase 2 Phase 3

  Show Detailed Description

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 34 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: We opted for a randomized, double-blind, placebo-controlled pilot trial with a lead-in phase. Moreover, the patients to be included in the placebo arm will receive riluzole during the last 6 months of study, so that all patients will undergo the active drug in the last phase of the study.
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Riluzole in Patients With Spinocerebellar Ataxia Type 7: a Randomized , Double-blind, Placebo-controlled Pilot Trial With a Lead in Phase
Estimated Study Start Date : January 1, 2019
Estimated Primary Completion Date : October 1, 2020
Estimated Study Completion Date : January 1, 2021


Arm Intervention/treatment
Experimental: Riluzole
Riluzole 50 mg twice daily for 12 months in the treated group. In pre-pubertal subjects the dosage will be adjusted on a mg/m2 basis according to the recommended human daily dose (RHDD; 100 mg).
Drug: Riluzole
Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months in the treated group.

Placebo Comparator: Placebo + riluzole
Placebo twice daily for 6 months and riluzole 50 mg twice daily for the following 6 months in the comparison group
Drug: Riluzole
Study drug will be orally dispensed in doses of 50 mg twice daily for 12 months in the treated group.

Drug: Placebo
Placebo drug for 6 months, however they will receive riluzole during the last 6 months of study, so that all patients will undergo the active drug in the last phase of the study.




Primary Outcome Measures :
  1. visual acuity expressed as log MAR units [ Time Frame: 18 months ]
    the metric to quantify the best corrected visual acuity, by applying the ETDRS chart (either back-illuminated or projected) with the patient's correction for distance

  2. the proportion of patients with stable Scale for the assessment and rating of ataxia (SARA) score [ Time Frame: 18 months ]

    neurological assessment for ataxia. It has eight categories with accumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia). When completing the outcome measure each category is assessed and scored accordingly. Scores for the eight items range as follows:

    Gait (0-8 points), Stance (0-6 points), Sitting (0-4 points) Speech disturbance (0-6 points) Finger chase (0-4 points) Nose-finger test (0-4 points) Fast alternating hand movement (0-4 points) Heel-shin slide (0-4 points) Once each of the 8 categories have been assessed, the total is calculated to determine the severity of ataxia.

    For motor activities of the four extremities (items 5-8), assessments are performed bilaterally, and the mean values are used to obtain the total score.



Secondary Outcome Measures :
  1. Farnsworth D15 Arrangement Test [ Time Frame: 18 months ]
    quantitative ophthalmologic assessments

  2. Visual evoked potentials [ Time Frame: 18 months ]
    quantitative ophthalmologic assessments

  3. Electroretinography [ Time Frame: 18 months ]
    quantitative ophthalmologic assessments

  4. Optical Coherence tomography [ Time Frame: 18 months ]
    quantitative ophthalmologic assessments

  5. Scale for the assessment and rating of ataxia (SARA) score [ Time Frame: 18 months ]

    neurological assessment for ataxia. It has eight categories with accumulative score ranging from 0 (no ataxia) to 40 (most severe ataxia). When completing the outcome measure each category is assessed and scored accordingly. Scores for the eight items range as follows:

    Gait (0-8 points), Stance (0-6 points), Sitting (0-4 points) Speech disturbance (0-6 points) Finger chase (0-4 points) Nose-finger test (0-4 points) Fast alternating hand movement (0-4 points) Heel-shin slide (0-4 points) Once each of the 8 categories have been assessed, the total is calculated to determine the severity of ataxia.

    For motor activities of the four extremities (items 5-8), assessments are performed bilaterally, and the mean values are used to obtain the total score.




Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   7 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • positive genetic test for SCA7.

Exclusion Criteria:

  • cardiac arrhythmias;
  • haematologic diseases;
  • hepatic diseases with serum values of alanine aminotransferase, aspartate aminotransferase or bilirubin > 1·5 times above normal limit;
  • pregnancy (women of childbearing potential agreed to use contraception);
  • breastfeeding.

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): NCT03660917


Locations
Layout table for location information
Italy
Center for Experimental Neurological Therapies (CENTERS), S. Andrea Hospital, Faculty of Medicine and Psychology, "Sapienza" University of Rome
Rome, Italy, 00139
Sponsors and Collaborators
S. Andrea Hospital

Layout table for additonal information
Responsible Party: Giovanni Ristori, Principal Investigator, S. Andrea Hospital
ClinicalTrials.gov Identifier: NCT03660917     History of Changes
Other Study ID Numbers: AIFA-2016-02365063
First Posted: September 7, 2018    Key Record Dates
Last Update Posted: December 19, 2018
Last Verified: December 2018

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Layout table for MeSH terms
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Cerebellar Ataxia
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Ataxia
Dyskinesias
Neurologic Manifestations
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