Study of Riluzole for Patients with Spinocerebellar Ataxia Type 7

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What is this study about?

This clinical trial is focused on studying a condition called Spinocerebellar Ataxia type 7 (SCA7), which is a rare genetic disorder that affects movement and coordination. The study is testing a medication called Riluzole, which is available in a form known as Glentek 50 mg film-coated tablets. The purpose of the study is to compare the effects of Riluzole with a placebo, which is a substance with no active medication, to see if Riluzole can help patients maintain stability in their movement and vision over time.

Participants in the study will be randomly assigned to receive either Riluzole or the placebo. The study will be conducted in a double-blind manner, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are not influenced by expectations. The trial will last for a period of 18 months, during which participants will be monitored for changes in their condition, specifically looking at their movement abilities and vision clarity.

Throughout the study, participants will undergo various assessments to track their progress. These assessments will include tests for movement coordination and vision, such as the SARA score, which measures ataxia symptoms, and visual acuity tests. The safety of Riluzole will also be evaluated by monitoring any side effects or changes in health. The goal is to determine if Riluzole can help stabilize the symptoms of SCA7 and improve the quality of life for those affected by this condition.

1 joining the study

Upon joining the study, you will be required to provide a signed informed consent. If you are a minor, your parent or legal representative must provide this consent on your behalf.

You must have a positive genetic test for spinocerebellar ataxia type 7 (SCA7) and be over 6 years old to participate.

2 lead-in phase

During the lead-in phase, you will be monitored to establish baseline measurements. This includes assessments of your SARA score, which evaluates your movement abilities, and your visual acuity, which measures how well you see.

These assessments will be used to compare your progress throughout the study.

3 medication administration

You will be randomly assigned to receive either the medication riluzole (Glentek 50 mg film-coated tablets) or a placebo. A placebo is a substance that looks like the medication but does not contain the active ingredient.

The medication or placebo will be taken orally. The specific dosage and frequency will be provided by the study team.

4 regular assessments

Throughout the study, you will undergo regular assessments to monitor your condition. These assessments will occur at specified intervals, such as at the start (t0), and then at 3 months (t3) and 6 months (t6).

The primary focus will be on your SARA score and visual acuity, with additional ophthalmologic tests to evaluate your eye health.

5 final evaluation

At the end of the 18-month study period, a final evaluation will be conducted to determine the stability of your SARA score and visual acuity.

The results will be compared to your baseline measurements to assess the effectiveness of the treatment.

6 safety monitoring

Your safety will be closely monitored throughout the study. This includes recording any adverse events or side effects you may experience.

Regular physical examinations and laboratory tests will be conducted to ensure your well-being.

Who Can Join the Study?

Both males and females can participate.
Participants must be older than 6 years.
A positive genetic test for Spinocerebellar ataxia type 7 (SCA7) is required.
A signed informed consent is necessary. For minors, this must be obtained from parents or a legal representative.

Who Cannot Join the Study?

Patients with any other serious health condition that could interfere with the study.
Patients who are pregnant or breastfeeding.
Patients who have participated in another clinical trial within the last 30 days.
Patients who have a history of drug or alcohol abuse within the past year.
Patients who are unable to comply with the study procedures.
Patients who have a known allergy to any of the study medications.
Patients with a history of certain eye diseases that could affect the study results.
Patients who have had a major surgery within the last 3 months.
Patients with uncontrolled high blood pressure.
Patients with severe liver or kidney disease.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Italy
A.O.U. Policlinico G. Martino Di Messina	Messina	Italy
Universita’ Degli Studi Di Ferrara	Ferrara	Italy
Uxddarrppb Dinyn Szvaq Du Rqgf Lu Ssagpkof	Rome	Italy

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	07.12.2021

Trial locations

Investigated drugs:

Riluzole

Riluzole is a medication that is being tested in this clinical trial for its potential benefits in patients with SpinoCerebellar Ataxia type 7. This condition affects the brain and can lead to problems with movement and coordination. Riluzole is already used to treat another neurological condition called Amyotrophic Lateral Sclerosis (ALS), where it helps to slow down the progression of symptoms. In this trial, researchers want to see if Riluzole can help patients with SpinoCerebellar Ataxia type 7 by keeping their symptoms stable, particularly focusing on their movement abilities and vision over an 18-month period.

Spinocerebellar ataxia type 7 (SCA7) – Spinocerebellar ataxia type 7 is a genetic disorder characterized by progressive problems with movement and coordination. It primarily affects the cerebellum, the part of the brain that controls balance and coordination, leading to symptoms such as unsteady walking, difficulty with fine motor tasks, and slurred speech. Over time, individuals with SCA7 may experience worsening of these symptoms, along with vision problems due to retinal degeneration. The disease is caused by a mutation in the ATXN7 gene, which leads to the production of an abnormal protein that accumulates in cells, causing damage. As the condition progresses, individuals may also develop muscle weakness and difficulty swallowing. SCA7 is inherited in an autosomal dominant pattern, meaning one copy of the altered gene in each cell is sufficient to cause the disorder.

Trial ID:

2024-518962-29-00

Protocol code:

AIFA-2016-02365063

Trial Phase:

Therapeutic exploratory (Phase II)

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Study of Riluzole for Patients with Spinocerebellar Ataxia Type 7

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?