Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Authorised, recruitment pending
Participants planned
30
Countries
1
Sites
1
Ischemic Stroke
To evaluate the effect of CD34+ cells administered intra-arterially in the three months infarct volume of patients with acute ischemic stroke in early (7 days) or late (20 days) subacute stage.
Key facts
- Sponsor
- Unidade Local De Saude De Coimbra E.P.E.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Decision date (initial)
- 2025-01-30
- Transition trial
- Yes
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
- Funding sources
- COMPETE 2020 (POCI-01- 0247-FEDER-003386)
External identifiers
- EU CT number
- 2025-520850-13-00
- EudraCT number
- 2017-002456-88
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy, Therapy
To evaluate the effect of CD34+ cells administered intra-arterially in the three months infarct volume of patients with acute ischemic stroke in early (7 days) or late (20 days) subacute stage.
Secondary objectives 7
- Functional outcome (modified Rankin scale and Stroke Impact Scale)
- Cognitive performance (Montreal Cognitive Assessment scale)
- Functional Independence and performance (Barthel Scale; Upper limb Capacity - Stroke Upper Limb Capacity Scale – SULCS; Evaluation of gait speed (TEST 10 METERS); Evaluation of temporospacial gait (GAIT RIte speed)
- Depression (Hospital Anxiety and Depression Scale- HADS Scale)
- Qualitiy of life assessment (EuroQol Scale EQ-5D)
- Deglutition (Teste Rápido de Identificação da Disfagia -TRIDIS; Functional Oral Intake de Identificação da Disfagia -FOIS)
- Aphasia (Bateria de Avaliação de Afasia de Lisboa - BAAL; The Aphasia Rapid Test- ART)
Conditions and MedDRA coding
Ischemic Stroke
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 22.1 | PT | 10061256 | Ischaemic stroke | 100000004852 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | STROKE34 Phase IIa randomized sham-controlled clinical trial, unicentre, superiority design, with blind outcome assessment evaluating the efficacy of intra-arterially administered CD34+ cells, at 7±2 and 20±5 days after ischemic stroke (PROBE design) by comparison of imaging outcome
|
Randomised Controlled | Single | [{"id":106397,"code":1,"name":"Subject"}] |
Regulatory references
- Plan to share IPD
- Yes
- IPD plan description
- The datasets generated during the study will be available on reasonable request by scientific researchers.
| EU CT number | Title | Sponsor |
|---|---|---|
| 2017-002456-88 | STROKE34: randomized controlled phase IIa trial of intra-arterial CD34+ cells in acute ischemic stroke. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 7
- Age 18-80 years
- Have suffered an acute hemispheric ischemic stroke attributable to injury within the territory supplied by the Middle Cerebral Artery (MCA)
- Symptomatic arterial territory is recanalyzed at the time of randomization
- Onset of an acute ischemic stroke that can have full clinical, imagiological and bone marrow collection within 7 days after the onset of symptoms. Onset is defined as the time that the subject was last seen in a normal state, or bedtime for unwitnessed strokes occurring during sleep
- Have readily accessible peripheral venous access blood sampling
- Have the ability to understand the requirements of the study and be willing to provide written informed consent, as evidenced by signature on an informed consent document (which has been submitted and approved by the local Ethical Committee),and agree to perform the required assessments. In the event of incapacitated subjects, informed consent will be sought from a legally acceptable representative
- NIHSS of at least 6 at the time of study inclusion
Exclusion criteria 13
- Patients found delirious, comatose, demented or having any mental impairment other than the neurological deficits related to the index stroke that in the investigator’s opinion renders the subject incapable to participate in the study
- Presence of high-grade (>70%) internal carotid artery stenosis or occlusion ipsilateral to the current stroke
- Have inflammatory disease at baseline (chronic systemic inflammatory disease active at the time of inclusion or acute inflammatory disease such as an infection)
- Have active malignancy, or recent surgery (within the previous 3 months)
- Have premorbid neurological deficits and functional limitations assessed by a premorbid Modified Rankin Scale (mRS) score >2
- Have severe co-existing diseases that may interfere with the conduct of the study, irrespective of stroke outcome
- Have known pregnancy. Females of childbearing potential will be screened at baseline with urine pregnancy test and positive results will be excluded (the choice of excluding pregnancies is due to the relative contra-indication to MRI in these patients)
- Contra-indication to MRI
- Allergy to contrast agents
- Non compliance of portuguese national law 12/2009 of march 26th (including but not limited to donor laboratorial analysis not compliant with HIV 1 and 2 (anti-HIV-1,2); hepatitis B (HBsAg; anti-HBc); hepatitisC (anti-HCV-Ab); syphilis and Mycoplasm
- Hematological causes of stroke
- Arterial dissection as cause of stroke or complication of previous angiography
- Known defect of clotting or platelet function
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 2
- Infarct volume at three months after ischemic stroke
- Volume de enfarte aos três meses após AVC isquémico
Secondary endpoints 7
- Functional outcome: modified Rankin Scale (mRS), Stroke Impact Scale (SIS)
- Cognitive performance: Montreal Cognitive Assessment scale (MoCA)
- Functional Independence and performance : Barthel Scale; Upper limb Capacity - Stroke Upper Limb Capacity Scale – SULCS; Evaluation of gait speed (TEST 10 METERS); Evaluation of temporospacial gait (GAIT RIte speed)
- Depression - Hospital Anxiety and Depression Scale- (HADS Scale)
- Qualitiy of life assessment - EuroQol Scale( EQ-5D)
- Deglutition: Teste Rápido de Identificação da Disfagia (TRIDIS); Functional Oral Intake de Identificação da Disfagia (FOIS)
- Aphasia: Bateria de Avaliação de Afasia de Lisboa - BAAL; The Aphasia Rapid Test (ART)
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
Strimvelis 1-10 x 106 cells/mL dispersion for infusion
PRD10726364 · Product
- Active substance
- Autologous CD34 Enriched Cell Fraction That Contains CD34 Cells Transduced with Retroviral Vector That Encodes for the Human Ada Cdna Sequence
- Substance synonyms
- Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene, GSK2696273, AUTOLOGOUS CD34+ ENRICHED CELL FRACTION THAT CONTAINS CD34+ CELLS TRANSDUCED WITH RETROVIRAL VECTOR THAT ENCODES FOR THE HUMAN ADENOSINE DEAMINASE CDNA SEQUENCE
- Pharmaceutical form
- DISPERSION FOR INFUSION
- Route of administration
- INTRAVENIOUS INFUSION
- Max daily dose
- 20 U unit(s)
- Max total dose
- 1000000 U unit(s)
- Max treatment duration
- 1 Week(s)
- Authorisation status
- Authorised
- ATC code
- L03AX — OTHER CYTOKINES AND IMMUNOMODULATORS
- Marketing authorisation
- EU/1/16/1097/001
- MA holder
- FONDAZIONE TELETHON ETS
- MA country
- Liechtenstein
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Unidade Local De Saude De Coimbra E.P.E.
- Sponsor organisation
- Unidade Local De Saude De Coimbra E.P.E.
- Address
- Praceta Professor Mota Pinto
- City
- Coimbra
- Postcode
- 3004-561
- Country
- Portugal
Scientific contact point
- Organisation
- Unidade Local De Saude De Coimbra E.P.E.
- Contact name
- João Sargento Freitas
Public contact point
- Organisation
- Unidade Local De Saude De Coimbra E.P.E.
- Contact name
- João Sargento Freitas
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Portugal | Authorised, recruitment pending | 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 |
|---|---|---|---|---|---|
| Portugal |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 5 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | 3 STROKE34_Protocolo_V3 | 3 |
| Recruitment arrangements (for publication) | Recruitment and Informed consent procedure_STROKE34 | 1 |
| Subject information and informed consent form (for publication) | Folheto_Inform_Consentimento_Informado_V2_24052021_Clean | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | IB_STROKE34_v2_21052021_clean | 1 |
| Synopsis of the protocol (for publication) | STROKE34 Sinopse Protocolo V3 | 3 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-01-30 | Portugal | Acceptable with conditions 2025-01-30
|
2025-01-30 |