Overview
Sponsor-declared trial summary
Phase
Human pharmacology (Phase I) - First administration to humans
Status
Ongoing, recruiting
Participants planned
10
Countries
1
Sites
1
Autoimmune refractory epilepsy and Rasmussen Encephalitis
To assess the safety and tolerability of intra-arterial administration of autologous mesenchymal stem cells in patients with immune-mediated refractory epilepsies.
Key facts
- Sponsor
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Princesa
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 24 Jan 2025 → ongoing
- Decision date (initial)
- 2025-01-24
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
External identifiers
- EU CT number
- 2024-520427-86-00
- EudraCT number
- 2021-003607-17
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Safety, Therapy, Dose response
To assess the safety and tolerability of intra-arterial administration of autologous mesenchymal stem cells in patients with immune-mediated refractory epilepsies.
Secondary objectives 3
- To assess the clinical response to intra-arterial administration of autologous bone marrow mesenchymal cells by detecting any changes in the neurological assessments, neuropsychological evaluations, structural and functional neuroimaging tests, and neurophysiological tests.
- Determine the effect of therapy on the patient's immune system and mRNA profile, both peripherally (peripheral blood) and locally in the primary sample obtained during surgery.
- To study the molecular profile of the mesenchymal stem cells used in the trial and correlate them with the clinical outcome.
Conditions and MedDRA coding
Autoimmune refractory epilepsy and Rasmussen Encephalitis
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 21.0 | LLT | 10071145 | Rasmussen syndrome | 10029205 |
| 20.0 | PT | 10071141 | Rasmussen encephalitis | 100000004852 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Acceptance of informed consent.
- Paediatric population under 16 years of age.
- Epilepsy of immune etiology refractory to first or second line treatment (antiepileptic drugs and conventional immunomodulatory therapy) in which motor and/or cognitive impairment is observed. A special group will be those undergoing epilepsy surgery in the context of Rasmussen's Encephalitis.
- Possibility of performing CMR, electrophysiological tests, PET and neuropsychological evaluations.
Exclusion criteria 4
- Concomitant illnesses that may affect patient safety or outcome such as: - Active CNS or systemic active infections - Active tumour - Active chemotherapy - Cardiac disease - Dementia or other degenerative CNS disease
- Any condition in which catheterisation of the arterial system is contraindicated or not feasible (major behavioural disorders preventing adequate collaboration, difficult arterial access or clinically relevant coagulation parameters).
- Any condition where therapeutic products cannot be administered (history of frequent adverse reactions or thromboembolic complications associated with blood components).
- Refusal of the patient or family members to adequate follow-up.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- To measure dose-limiting toxicities (DLTs, defined in the protocol as MSC infusion-related grade 3 toxicities occurring in the first 28 days of treatment).
Secondary endpoints 1
- To asses clinical response
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD11445375 · Product
- Active substance
- Allogenic Bone Marrow-Derived Mesenchymal Stem Cells Transduced with ICOVIR-5, Ex Vivo Expanded
- Substance synonyms
- ALOCELYVIR
- Pharmaceutical form
- CELL SUSPENSION FOR INJECTION
- Route of administration
- INTRAARTERIAL USE
- Authorisation status
- Not Authorised
- MA holder
- FUNDACIO INSTITUT D'INVESTIGACIO BIOMEDICA DE BELLVITGE IDIBELL
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Princesa
- Sponsor organisation
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Princesa
- Address
- Calle De Diego De Leon 62
- City
- Madrid
- Postcode
- 28006
- Country
- Spain
Scientific contact point
- Organisation
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Princesa
- Contact name
- Gina Paola Mejía Abril
Public contact point
- Organisation
- Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Princesa
- Contact name
- Gina Paola Mejía Abril
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruiting | 10 | 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 |
|---|---|---|---|---|---|
| Spain | 2025-01-24 | 2025-01-24 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-01-24 | Spain | Acceptable with conditions 2025-01-24
|
2025-01-24 |