Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
30
Countries
1
Sites
1
Cerebral Autosomal Dominant Arteriopathy with subcortical Infarcts and Leukoencephalopathy (CADASIL)
The objective of this trial is the global risk-benefit assessment of Cerebrolysin as compared to Placebo in patients with genetically proven CADASIL.
Key facts
- Sponsor
- Ever Neuro Pharma GmbH
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 29 Nov 2023 → ongoing
- Decision date (initial)
- 2024-09-19
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
External identifiers
- EU CT number
- 2024-513828-42-00
- EudraCT number
- 2022-002394-29
- ClinicalTrials.gov
- NCT05755997
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety, Efficacy
The objective of this trial is the global risk-benefit assessment of Cerebrolysin as compared to Placebo in patients with genetically proven CADASIL.
Conditions and MedDRA coding
Cerebral Autosomal Dominant Arteriopathy with subcortical Infarcts and Leukoencephalopathy (CADASIL)
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 6
- Patients of ≥18 years of age, all genders
- Diagnosis of CADASIL based on clinical symptoms, MRI, and genetic analysis
- MoCA >11
- Adequate visual, auditory, and language skills (no language interpreter required) to follow study procedures
- Patient is not of childbearing potential (i.e. women are post-menopausal for two years, surgically sterile, or using adequate method of contraception such as hormonal contraception in combination with a barrier method, the use of an intrauterine device or hormone-releasing system, bilateral tubal occlusion, vasectomized partner, sexual abstinence)
- Patient participates voluntarily and gave written informed consent
Exclusion criteria 7
- Any significant neurological disease/conditions other than CADASIL
- Focal lesions that may be responsible for the cognitive status of the patient (e.g. infectious disease, space-occupying lesion, normal pressure hydrocephalus)
- Any other diseases/conditions that may affect compliance with the protocol, such as: a. severe psychiatric disorders within the last three months b. delusional symptoms c. history of schizophrenia, schizoaffective disorder, bipolar affective disorder d. major depressive disorder newly identified within eight weeks before screening e. history of alcohol or substance abuse or dependence within the past two years
- Any circumstances that -in the investigator’s opinion- may result in the patient’s non-compliance with study procedures, e.g. fragile or thin veins that prevent many i.v. infusions
- Any other disease/conditions that may affect the safety assessment, such as: a. history of systemic cancer within the past two years b. history of myocardial infarction in the past year or unstable or severe cardiovascular disease (including uncontrolled hypertension and/or history of unstable hypertension not compensated by antihypertensive therapy) c. any clinically significant laboratory abnormalities at screening d. uncontrolled insulin-requiring diabetes or non-insulin dependent diabetes mellitus (HbA1c >87 mmol/mol)
- Use of concomitant medication with neuroprotective/neurotrophic/nootropic effects (e.g. ginkgo biloba, erythropoietin, citicoline, amantadine, piracetam)
- Any condition that would represent a contraindication for Cerebrolysin administration: a. hypersensitivity to one of the components of the drug b. epilepsy c. severe renal impairment (estimated Glomerular Filtration Rate [eGFR] <30 ml/min/1.73 m2 as assessed at local laboratory within one month before screening)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- The primary multidimensional outcome ensemble comprises 10 single analysis variables of three dimensions (cognition, mood, imaging characteristics) assessed during and at the end of treatment phases I and II (at months 6, 12, 21, and 27). Month 12 (end of phase I) and Month 27 (end of phase II) are the primary endpoints of the formal cross-over analysis.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Cerebrolysin 215,2mg/ml Injekční roztok
PRD526497 · Product
- Active substance
- Cerebrolysin Concentrate
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 40 ml millilitre(s)
- Max total dose
- 1920 ml millilitre(s)
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- N07X — OTHER NERVOUS SYSTEM DRUGS
- Marketing authorisation
- 04/127/71-C
- MA holder
- EVER NEURO PHARMA GMBH
- MA country
- Czech Republic
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Placebo 1
Sodium chloride Fresenius Kabi 0,9% infuzní roztok
PRD2128237 · Product
- Active substance
- Sodium Chloride
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS INFUSION
- Max daily dose
- 100 ml millilitre(s)
- Max total dose
- 4800 ml millilitre(s)
- Max treatment duration
- 12 Month(s)
- Authorisation status
- Authorised
- ATC code
- B05XA03 — SODIUM CHLORIDE
- Marketing authorisation
- 76/365/96-C
- MA holder
- FRESENIUS KABI S.R.O.
- MA country
- Czech Republic
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Ever Neuro Pharma GmbH
- Sponsor organisation
- Ever Neuro Pharma GmbH
- Address
- Oberburgau 3, Oberburgau Oberburgau
- City
- Sankt Gilgen
- Postcode
- 4866
- Country
- Austria
Scientific contact point
- Organisation
- Ever Neuro Pharma GmbH
- Contact name
- Stefan Winter
Public contact point
- Organisation
- Ever Neuro Pharma GmbH
- Contact name
- Stefan Winter
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Czechia | Ongoing, recruiting | 30 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
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 |
|---|---|---|---|---|---|
| Czechia | 2023-11-29 | 2023-11-29 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 7 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2024-513828-42-00 redacted | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements placeholder | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF biomarker redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF data protection redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF main redacted | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF MRI redacted | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Cerebrolysin | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-08-22 | Czechia | Acceptable with conditions 2024-09-19
|
2024-09-19 |