Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
34
Countries
1
Sites
4
Spinocerebellar ataxia type 7 (SCA7)
To compare the two study arms for the proportion of patients who remain stable at SARA score and visual acuity at 18 months respect to run-in.
Key facts
- Sponsor
- Universita Degli Studi Di Roma La Sapienza
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years, 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 7 Dec 2021 → ongoing
- Decision date (initial)
- 2024-11-27
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- AIFA (bandi per la ricerca indipendente)
External identifiers
- EU CT number
- 2024-518962-29-00
- EudraCT number
- 2018-000282-37
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
To compare the two study arms for the proportion of patients who remain stable at SARA score and visual acuity at 18 months respect to run-in.
Secondary objectives 1
- To evaluate the effect of riluzole on visual function using quantitative ophthalmologic assessments and on SARA score, as continuous values, assessing changes at 18 months compared to run-in. To investigate the safety and tolerability of riluzole administered in SCA 7 patients
Conditions and MedDRA coding
Spinocerebellar ataxia type 7 (SCA7)
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | SOC | 10029205 | Nervous system disorders | 8 |
Study design 7 periods
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Visit 1 – from day -3 to day +1 Baseline Baseline evaluation will include history, electrocardiogram, clinical and neurological assessment using SARA and ophthalmological exams (Best corrected visual acuity, Color vision, Visual evoked potentials, Electroretinography,Optical Coherence tomography, Computerized visual field examination). Blood will be sampled for routine laboratory evaluation.
|
Not Applicable | None | ||
| 2 | Visit 2 – 3 months (±7 days) Follow-up (lead-in period) The following assessments will be done at visits:
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Collection of the medical history
• Physical examination and neurological examination
• Ophthalmological assessment
• SARA scale
• Hematology and Blood Chemistry (and pregnancy test if female)
• Electrocardiogram
|
Not Applicable | None | ||
| 3 | Visit 3 - 6 months (±7 days) Randomization, treatment start The following assessments will be done at visits:
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Collection of the medical history
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Ophthalmological assessment
• SARA scale
• Hematology and Blood Chemistry (and pregnancy test if female)
• Electrocardiogram
• Randomization
• Treatment start
|
Randomised Controlled | Double | [{"id":147811,"code":2,"name":"Investigator"},{"id":147810,"code":1,"name":"Subject"}] | Placebo: Placebo will be given orally every 12 h for 6 months in the comparison group Treatment: Riluzole (Glentek) 50 mg will be given orally every 12 h for 12 months in the treated group and 6 months in the comparison group |
| 4 | Visit 4 – 9 months (±7 days) Follow-up visit The following assessments will be done at visits:
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Collection of the medical history
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Ophthalmological assessment
• SARA scale
• Hematology and Blood Chemistry (and pregnancy test if female)
• Electrocardiogram
|
Randomised Controlled | Double | [{"id":147814,"code":1,"name":"Subject"},{"id":147813,"code":2,"name":"Investigator"}] | Placebo: Placebo will be given orally every 12 h for 6 months in the comparison group Treatment: Riluzole (Glentek) 50 mg will be given orally every 12 h for 12 months in the treated group and 6 months in the comparison group |
| 5 | Visit 5 – 12 months (±7 days) Follow-up visit The following assessments will be done at visits:
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Collection of the medical history
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Ophthalmological assessment
• SARA scale
• Hematology and Blood Chemistry (and pregnancy test if female)
• Electrocardiogram
|
Randomised Controlled | Double | [{"id":147817,"code":2,"name":"Investigator"},{"id":147816,"code":1,"name":"Subject"}] | Placebo: Placebo will be given orally every 12 h for 6 months in the comparison group Treatment: Riluzole (Glentek) 50 mg will be given orally every 12 h for 12 months in the treated group and 6 months in the comparison group |
| 6 | Visit 6 – 15 months (±7 days) Follow-up visit The following assessments will be done at visits:
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Collection of the medical history
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Ophthalmological assessment
• SARA scale
• Hematology and Blood Chemistry (and pregnancy test if female)
• Electrocardiogram
|
Randomised Controlled | Double | [{"id":147819,"code":2,"name":"Investigator"},{"id":147820,"code":1,"name":"Subject"}] | Placebo: Placebo will be given orally every 12 h for 6 months in the comparison group Treatment: Riluzole (Glentek) 50 mg will be given orally every 12 h for 12 months in the treated group and 6 months in the comparison group |
| 7 | Visit 7 – 18 months (±7 days) End of study visit The following assessments will be done at visits:
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Collection of the medical history
• Evaluation of concomitant medications
• Physical examination and neurological examination
• Ophthalmological assessment
• SARA scale
• Hematology and Blood Chemistry (and pregnancy test if female)
• Electrocardiogram
|
Randomised Controlled | Double | [{"id":147822,"code":1,"name":"Subject"},{"id":147823,"code":2,"name":"Investigator"}] | Placebo: Placebo will be given orally every 12 h for 6 months in the comparison group Treatment: Riluzole (Glentek) 50 mg will be given orally every 12 h for 12 months in the treated group and 6 months in the comparison group |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- Male and female of any race and > 6 years old
- Positive genetic test for SCA7
- Signed Informed Consent (in case of minors, written informed consent must be obtained by parents or legal representative)
Exclusion criteria 5
- Female subjects: pregnant or lactating women cannot participate in the study. Women of childbearing potential cannot participate unless willing to use highly effective contraception methods as combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable; intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomised partner; sexual abstinence. In case of use of oral contraception, women should have been stable on the same pill for a minimum of 3 months before taking study drug. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
- Subjects with a clinically significant or unstable medical or surgical condition that would preclude safe and complete study participation. Such conditions may include cardiovascular, pulmonary, hepatic, renal, severe systemic mycotic infections, metabolic diseases or malignancies
- Hepatic diseases with serum values of alanine aminotransferase, aspartate aminotransferase or bilirubin > 1·5 times above normal limit
- Any medical or psychiatric condition that may affect the subject ability to give informed consent, or to complete the study, or if the subject is considered by the treating neurologist to be, for any other reason, an unsuitable candidate for this study
- Known hypersensitivity to any component of riluzole (Glentek)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The primary endpoints will be the proportion of patients with stable SARA score and visual acuity expressed as log MAR units at 18 months, in comparison with the same parameters as mean of t0-t3-t6 evaluations .
Secondary endpoints 2
- The secondary endpoint will be quantitative ophthalmologic assessments (via a Farnsworth D15 Arrangement Test, Visual evoked, Electroretinography, Optical Coherence tomography, Computerized visual field examination) and SARA score as continuous values at 18 months, in comparison with the same parameters calculated for each patient as mean of t0-t3-t6 evaluations
- The safety profile will be assessed through the recording, reporting and analyzing of baseline medical conditions, adverse events, physical examination findings including laboratory tests.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD525388 · Product
- Active substance
- Riluzole
- Pharmaceutical form
- FILM-COATED TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 100 mg milligram(s)
- Max total dose
- 100 mg milligram(s)
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- N07XX02 — RILUZOLE
- Marketing authorisation
- 85173.00.00
- MA holder
- GLENMARK ARZNEIMITTEL GMBH
- MA country
- Germany
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- Yes
- Modification description
- Over-encapsulation for masking
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Universita Degli Studi Di Roma La Sapienza
- Sponsor organisation
- Universita Degli Studi Di Roma La Sapienza
- Address
- Via Di Grottarossa 1035-1039
- City
- Rome
- Postcode
- 00189
- Country
- Italy
Scientific contact point
- Organisation
- Universita Degli Studi Di Roma La Sapienza
- Contact name
- Giovanni Ristori
Public contact point
- Organisation
- Universita Degli Studi Di Roma La Sapienza
- Contact name
- Silvia Romano
Locations
1 EU/EEA country · 4 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Italy | Ongoing, recruiting | 24 | 4 |
| Rest of world
United States
|
— | 10 | — |
Investigational sites
Universita Degli Studi Di Roma La Sapienza
AO S. Andrea - UOC Neurologia, Via Di Grottarossa 1035-1039, 00189, Rome
University Hospital Of Ferrara
Azienda Ospedaliera-Universitaria Sant’Anna - Clinica Pediatrica, Cona, Via Aldo Moro 8, Ferrara
Azienda Ospedaliera Universitaria Gaetano Martino Messina
UOC Neurologia e malattie neuromuscolari, Via Consolare Valeria N 1, 98124, Messina
Azienda Unita Sanitaria Locale Di Bologna
IRCCS Istituto delle Scienze Neurologiche di Bologna - UOC Clinica Neurologica, Via Altura 3, 40139, Bologna
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Italy | 2021-12-07 | 2021-12-07 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 15 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-518962-29-00_Redacted | 3.0 |
| Protocol (for publication) | D2_5 RISCA7 D2_Protocol modification n 2 2024-518962-29-00_Redacted | 2 |
| Recruitment arrangements (for publication) | K1. Recruitment arrangement | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 14-18 yr | 3.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF 7-13 yr | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF adults_Redacted | 4.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF parents_Redacted | 4.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Privacy adults_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Privacy parents_Redacted | 3.0 |
| Subject information and informed consent form (for publication) | L2_1 RISCA7 GP Letter | 3.0 |
| Subject information and informed consent form (for publication) | L2_2 RISCA7 Letter for pediatrician | 3.0 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Glentek 50mg Film coated Tablets | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol Lay Summary EN 2024-518962-29-00 | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Lay Summary ITA 2024-518962-29-00 | 1.0 |
| Synopsis of the protocol (for publication) | D1_Protocol Synopsis ITA 2024-518962-29-00_Clean | 3.0 |
Application history
2 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-10-15 | Italy | Acceptable 2024-11-18
|
2024-11-27 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2025-08-05 | Italy | Acceptable 2025-10-27
|
2025-10-27 |