Spinocerebellar Ataxia Type 2Phase 3ATRILNCT03347344
Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2 (ATRIL)
A Phase 3 study testing Riluzole in people with Spinocerebellar Ataxia Type 2, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Spinocerebellar Ataxia Type 2
- What is being tested?
- Riluzole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 42 participants
- Where
- 1 siteFrance
- Study dates
- 17 Jan 2018to 14 Dec 2020
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
Show all 12Show fewer
- 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 locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read more
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.
Read the 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.
- Conditions
- Spinocerebellar Ataxia Type 2
- Being tested
- Riluzole, Placebo
- Keywords
- Spinocerebellar ataxia-SCA2, SARA (Scale for the Assessment and Rating of Ataxia) score, Riluzole (Rilutek)
- Medical subject headings
- Spinocerebellar Ataxias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RiluzoleDrug
50 mg will be administered (per os) twice a day
PlaceboDrug
50 mg will be administered (per os) twice a day
| Study group | Role | What they receive |
|---|---|---|
| RILUZOLE | Experimental | 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. Receives: Drug: Riluzole |
| PLACEBO | Placebo comparator | 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 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA))
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
Measured: at 12 months.
Secondary outcomes (3)
Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score)
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.
Measured: at 12 months
Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS))
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
Measured: at 12 months
12 months survival
To compare survival of the patients between the two treatment groups at 12 months
Measured: at 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2017
- Study start17 Jan 2018
- Primary completion14 Dec 2020
- Study completion14 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinocerebellar Ataxia Type 2Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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 blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- DURR Alexandra, PU-PH, Principal investigator, ASSISTANCE PUBLIQUE HÖPITAUX DE PARIS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03347344
- Sponsor's protocol ID
- P160927J
- Other IDs
- 2017-001481-23, 2017
- Acronym
- ATRIL
- Status last verified
- Jun 2021
- First submitted
- 21 Sep 2017
- Last updated
- 21 Jun 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Coarelli G, Heinzmann A, Ewenczyk C, Fischer C, Chupin M, Monin ML, Hurmic H, Calvas F, Calvas P, Goizet C, Thobois S, Anheim M, Nguyen K, Devos D, Verny C, Ricigliano VAG, Mangin JF, Brice A, Tezenas du Montcel S, Durr A. Safety and efficacy of riluzole in spinocerebellar ataxia type 2 in France (ATRIL): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2022 Mar;21(3):225-233. doi: 10.1016/S1474-4422(21)00457-9. Epub 2022 Jan 18.
PubMed 35063116
Study at a glance
From the registry record, last updated by the sponsor on 21 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter, Randomized, Double Blind, Placebo Controlled Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2
- ClinicalTrials.gov ID
- NCT03347344
- Acronym / sponsor ID
- ATRIL / P160927J
- Conditions
- Spinocerebellar Ataxia Type 2
- Keywords
- Spinocerebellar ataxia-SCA2, SARA (Scale for the Assessment and Rating of Ataxia) score, Riluzole (Rilutek)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 17 Jan 2018 (actual)
- Primary completion
- 14 Dec 2020 (actual)
- Study completion
- 14 Dec 2020 (actual)
- First posted
- 20 Nov 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- 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
| Arm | Type | Receives |
|---|---|---|
| RILUZOLE | Experimental | Drug: RiluzoleRiluzole 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. |
| PLACEBO | Placebo comparator | Drug: PlaceboThe 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Riluzole50 mg will be administered (per os) twice a day | Drug | RILUZOLE |
| Placebo50 mg will be administered (per os) twice a day | Drug | PLACEBO |
Primary outcome
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
3 secondary outcomes
Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score)
Time frame: at 12 months
Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS))
Time frame: at 12 months
12 months survival
Time frame: at 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jun 2021.
- Derived from this study: PubMed 35063116Coarelli G, Heinzmann A, Ewenczyk C, Fischer C, Chupin M, Monin ML, Hurmic H, Calvas F, Calvas P, Goizet C, Thobois S, Anheim M, Nguyen K, Devos D, Verny C, Ricigliano VAG, Mangin JF, Brice A, Tezenas du Montcel S, Durr A. Safety and efficacy of riluzole in spinocerebellar ataxia type 2 in France (ATRIL): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2022 Mar;21(3):225-233. doi: 10.1016/S1474-4422(21)00457-9. Epub 2022 Jan 18.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
DURR Alexandra, PU-PH principal investigator
ASSISTANCE PUBLIQUE HÖPITAUX DE PARIS
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Riluzole in people with Spinocerebellar Ataxia Type 2, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Jun 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA)) (time frame: at 12 months.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03347344, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Jun 2021.