Evaluation of novel thrombolytic TGD001 in patients with acute ischemic stroke
| ISRCTN | ISRCTN97585476 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN97585476 |
| Integrated Research Application System (IRAS) | 1013913 |
| Sponsor's protocol code number | TG1-CL-201 |
| Sponsor | TargED Biopharmaceuticals B.V. |
| Funder | TargED Biopharmaceuticals B.V. |
- Submission date
- 25/07/2026
- Registration date
- 28/09/2026
- Last edited
- 28/09/2026
- Recruitment status
- Recruiting
- Overall study status
- Ongoing
- Condition category
- Circulatory System
Plain English summary of protocol
Background and study aims
An acute ischaemic stroke happens when a blood clot blocks blood flow to part of the brain. This can damage brain tissue and may cause long-term disability. Current treatments include medicines that dissolve blood clots and a procedure to remove the clot, called thrombectomy. However, these treatments do not always work well enough, so better treatments are needed.
This study is testing a new clot-dissolving medicine called TGD001. TGD001 targets a part of blood clots called von Willebrand factor, which is different from the main target of currently used clot-dissolving medicines. The main aim of the study is to find out whether TGD001 is safe and well tolerated in people who have had an acute ischaemic stroke. The study will also look at how TGD001 affects blood flow in the brain and recovery after stroke.
Who can participate?
Adults aged 18 years or older who have had an acute ischaemic stroke confirmed by a brain scan may be able to take part. This includes some people who have had a thrombectomy and some people who are not suitable for standard clot treatments. Men and women can take part, but women who are pregnant or breastfeeding cannot participate.
What does the study involve?
About 130 people will take part at specialist stroke hospitals.
The study has two parts. In the first part, small groups of participants receive a single dose of TGD001 into a vein. Different doses are studied to help identify a suitable dose for further testing. In the second part, participants are randomly assigned to receive either TGD001 or a placebo treatment. Neither the participant nor most of the study team will know which treatment was given.
Participants will continue to receive the standard stroke care that is appropriate for them. During the study, doctors will monitor participants for side effects. Participants will also have blood tests, brain scans and assessments of their stroke symptoms and ability to carry out everyday activities. Each participant will be followed for about 90 days.
What are the possible benefits and risks of participating?
Participants may or may not benefit directly from taking part. TGD001 may help dissolve blood clots and improve blood flow to the brain, but it is not yet known whether it will improve recovery from stroke. Participants will also be closely monitored during the study. The information collected may help researchers better understand TGD001 and may contribute to better treatments for people with stroke in the future.
The main potential risk of TGD001 is bleeding, which may occur in the brain or elsewhere in the body. There is also a possibility of an allergic or treatment-related reaction. Blood tests, brain scans and other study procedures may also cause discomfort or carry small risks. Participants will be monitored and treated if any problems occur.
Where is the study run from?
The study is managed by TargED Biopharmaceuticals B.V. in Utrecht, the Netherlands.
When is the study starting and how long is it expected to run for?
March 2026 to December 2027
Who is funding the study?
TargED Biopharmaceuticals B.V. (Netherlands)
Who is the main contact?
Sonja Visscher, clinops@targedbio.com
Contact information
Scientific, Public
Princetonlaan 6
Utrecht
3584 CB
Netherlands
| Phone | +31 (0)30 2028079 ext 00 |
|---|---|
| regulatory@Targedbio.com |
Scientific, Public
Princetonlaan 6
Utrecht
3584 CB
Netherlands
| Phone | +31 (0)30 2028079 ext 00 |
|---|---|
| clinops@targedbio.com |
Principal investigator
Guy Hilton Research Centre
1 Thornburrow Drive
Stoke-on-Trent
ST4 7QB
United Kingdom
| Phone | +44 (0)1782 679484 |
|---|---|
| Christine.Roffe@uhnm.nhs.uk |
Study information
| Primary study design | Interventional |
|---|---|
| Allocation | Randomized controlled trial |
| Masking | Blinded (masking used) |
| Control | Placebo |
| Assignment | Parallel |
| Purpose | Treatment, Safety |
| Scientific title | A Phase Ib/IIa open-label dose-finding followed by randomized, double-blind, placebo-controlled expansion study to evaluate the safety, tolerability, and preliminary efficacy of TGD001 in patients with acute ischemic stroke |
| Study acronym | TG1-CL-201 |
| Study objectives | Primary objective: To assess the safety and tolerability of single intravenous (IV) doses of TGD001 in participants with an acute ischemic stroke (AIS) diagnosis, supported by neuroimaging, who either receive endovascular thrombectomy (EVT) or do not receive EVT or standard of care (SOC) intravenous thrombolysis (IVT). Secondary objectives: 1. To assess the rates of asymptomatic intracranial hemorrhage (aICH) 2. To determine the pharmacokinetic (PK) profile of single IV doses of TGD001 in participants with AIS 3. To assess recanalization post-intervention 4. To assess reperfusion post-intervention 5. To assess neurological outcome at 90 days post-intervention 6. To determine the functional status at 90 days post-intervention |
| Ethics approval(s) |
Not yet submitted |
| Health condition(s) or problem(s) studied | Acute ischemic stroke |
| Intervention | The trial consists of two parts and includes prespecified groups of participants with acute ischemic stroke. Phase 1b is an open-label, single-dose escalation and expansion phase. Participants receive one intravenous administration of TGD001. Dose-escalation and de-escalation decisions are overseen by the Data Safety Monitoring Committee (DMC), which will select the dose or doses for further evaluation. Three dose levels are planned for each patient group. Additional dose levels may be established by the DMC. Phase 2a is a randomized, double-blind, placebo-controlled expansion phase. Participants receive one intravenous administration of either TGD001 or placebo. Randomisation occurs before treatment according to a prespecified randomisation schedule. All participants are followed for approximately 90 days. Follow-up includes safety assessments, laboratory and pharmacokinetic/pharmacodynamic sampling, brain imaging, and neurological and functional outcome assessments. |
| Intervention type | Drug |
| Phase | Phase II |
| Drug / device / biological / vaccine name(s) | TGD001 |
| Primary outcome measure(s) |
1. Symptomatic intracranial haemorrhage (sICH) is assessed according to the Heidelberg Bleeding Classification during the post-intervention follow-up (90 days) |
| Key secondary outcome measure(s) |
1. Asymptomatic intracranial haemorrhage (aICH) is assessed by brain imaging during post-intervention follow-up (90 days) |
| Completion date | 31/12/2027 |
Eligibility
| Participant type(s) | |
|---|---|
| Age group | Mixed |
| Lower age limit | 18 Years |
| Upper age limit | 100 Years |
| Sex | All |
| Target sample size at registration | 130 |
| Key inclusion criteria | 1. Age ≥18 years 2. Informed consent obtained from the participant or a participant representative in accordance with local legal and institutional review board (IRB) and independent ethics committee (IEC) requirements 3. Functionally independent (Modified Rankin Scale [mRS] 0-2) prior to stroke onset 4. Diagnosis of AIS supported by neuroimaging: 4.1. A visible occlusion on computed tomography angiography (CTA) or magnetic resonance angiography (MRA) imaging; and/or 4.2. An ischemic lesion ( Alberta stroke program early CT score [ASPECTS] 6 - 10) on non-contrast computed tomography (NCCT) or magnetic resonance imaging (MRI), with target mismatch on computed tomography perfusion (CTP) or magnetic resonance perfusion (MRP) imaging, if performed 5. National Institutes of Health Stroke Scale (NIHSS) ≥5 and that remains ≥5 immediately before enrollment 6. Participants who: 6.1. Meet the local endovascular thrombectomy (EVT) eligibility requirements and undergo EVT (Groups 1 and 2), or 6.2. Do not receive EVT or SOC IVT (Group 3) |
| Key exclusion criteria | 1. Unable to undergo both MRI and CT imaging 2. Intracranial hemorrhage on pre-intervention imaging 3. Demarcated infarct or early ischemic changes resulting in ASPECTS ≤5 on pre-intervention imaging 4. Active internal bleeding 5. Known hereditary or acquired hemorrhagic diathesis, coagulation factor deficiency 6. Use of an anticoagulant, including but not limited to direct oral anticoagulants (DOACs) or warfarin, within the last 48 hours or recent oral anticoagulant therapy with international normalized ratio (INR) ≥1.7. If the DOAC level is measured and found to be below the therapeutic threshold (Factor Xa inhibitor levels of less than 50 ng/ml or anti-Xa levels of less than 0.5 IU/ml), the participant can be enrolled regardless of the recency of the last dose. 7. Use of any medication and/or recent procedure that is known to significantly increase the bleeding risk (at the discretion of the investigator) 8. More than four recanalization attempts of target lesion with stent retriever or aspiration device during EVT prior to the use of the trial intervention 9. Coma, and/or NIHSS >25 10. Seizure(s) at stroke onset if this precludes obtaining an accurate baseline NIHSS 11. Severe, uncontrolled hypertension (systolic blood pressure >180 mmHg or diastolic blood pressure >110 mmHg). NOTE: If the blood pressure can be successfully reduced and maintained at an acceptable level using European Stroke Organization (ESO) guidelines-recommended medication (including i.v. antihypertensive drips), the participant can be enrolled. 12. Platelet count <100.000/µl 13. Blood glucose <50 mg/dl (2.8 mmol/l) or >400 mg/dL (22.20 mmol/l) 14. Impaired renal function as indicated by estimated glomerular filtration rate (GFR) below 30 ml/min/1.73 m² or requiring hemodialysis/peritoneal dialysis 15. Severe liver fibrosis or portal hypertension 16. Female who is pregnant, lactating, or has a positive pregnancy test at the time of admission 17. Evidence of active systemic infection that could have led to presumed septic embolus, suspicion of bacterial endocarditis 18. Surgery or trauma affecting intracranial or intraspinal areas within the last 2 months 19. Pre-existing severe medical, neurological, or psychiatric disease that would significantly confound the neurological or functional evaluations (investigator judgment) 20. History of cerebrovascular incident in the last 3 months 21. Intracranial neoplasm, arteriovenous malformation, or aneurysm 22. Other serious, advanced, or terminal illness in the last 3 months (investigator judgment) or life expectancy is less than 6 months 23. Known hypersensitivity to any of the trial treatments or their excipients or to drugs of similar chemical classes 24. Participant participating in a trial involving an investigational drug or device that would impact this trial within the last 30 days |
| Date of first enrolment | 11/03/2026 |
| Date of final enrolment | 06/12/2027 |
Locations
Countries of recruitment
- United Kingdom
- England
- Belgium
- France
- Germany
- Netherlands
- Poland
- Serbia
- Spain
- United States of America
Study participating centres
Stoke-on-trent
ST4 6QG
England
London
SE5 9RS
England
Newcastle upon Tyne
NE1 4LP
England
London
NW1 2PG
England
Hamburg
20246
Germany
Altenburg
4600
Germany
Heidelberg
69120
Germany
Potsdam
14467
Germany
Dresden
1307
Germany
Bonn
53127
Germany
Tübingen
72076
Germany
Berlin
12203
Germany
Sevilla
41008
Spain
Barcelona
Badalona
Spain
Murcia
30120
Spain
Pontevedra
36312
Spain
Sevilla
41013
Spain
Barcelona
8035
Spain
Valladolid
47003
Spain
Girona
17007
Spain
Krakow
30-688
Poland
Katowicach ul. Ziołowa 45-47
Katowice
40-635
Poland
Belgrade
11000
Serbia
Nis
18000
Serbia
Kragujevac
34000
Serbia
Novi Sad
21137
Serbia
Results and Publications
| Individual participant data (IPD) Intention to share | No |
|---|
Editorial Notes
26/09/2026: ISRCTN received notification of combined HRA/MHRA approval for this trial on 26/09/2026.
27/07/2026: Study's existence confirmed by the HRA.
