Randomized Clinical Trial of Endovascular Treatment for Progressive Stroke With Vertebrobasilar Artery Occlusion

Safety and Efficacy of Endovascular Treatment for Progressive Stroke Due to Vertebrobasilar Artery Occlusion: A Multicenter, Prospective, Open-Label Randomized Controlled Trial With Blinded Endpoint Assessment

This multicenter, prospective, open-label randomized controlled trial with blinded assessment was designed to assess the efficacy and safety of endovascular treatment for progressive stroke due to vertebrobasilar artery occlusion.

Study Overview

• Status: Not yet recruiting
• Conditions: Endovascular Treatment; Vertebrobasilar Artery Occlusion
• Intervention / Treatment: Procedure: Endovascular Recanalization; Drug: Medical Therapy Alone

Detailed Description

This multicenter, prospective, open-label, randomized controlled trial with blinded endpoint assessment (PROBE design) was designed to evaluate endovascular treatment (EVT) in patients with progressive stroke due to vertebrobasilar artery occlusion (VBAO). Progressive stroke due to VBAO was defined as vertebrobasilar artery occlusion confirmed by CTA, MRA, or DSA, accompanied by neurological deterioration occurring between 24 hours and 14 days after symptom onset, defined as an increase of ≥4 points in the National Institutes of Health Stroke Scale (NIHSS) score resulting in a total NIHSS score ≥10 in patients with an initial NIHSS score <10. Participants will be randomly assigned in a 1:1 ratio to receive EVT plus medical therapy or medical therapy alone. The primary endpoint is the proportion of patients with good functional outcome at 90 days, defined as a modified Rankin Scale (mRS) score of 0 to 3. Secondary outcomes include the distribution of mRS scores at 90 days, all-cause mortality at 90 days, and the incidence of symptomatic intracranial hemorrhage (sICH).

• Study Type: Interventional
• Enrollment (Estimated): 240
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Feng Gao, MD; Phone Number: 13581936066; Email: gaofengletter@sina.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100070
      • Beijing Tiantan Hospital, Capital Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥18 years.
2. Acute ischemic stroke, with a National Institutes of Health Stroke Scale (NIHSS) score of <10 at initial symptom onset, and no prior endovascular treatment.
3. Stroke progression occurring from 24 hours to 14 days after initial symptom onset, defined as an NIHSS score of ≥10 with an increase of ≥4 points from baseline.
4. Acute basilar artery occlusion or dominant vertebral artery occlusion with contralateral occlusion or hypoplasia, confirmed by CTA, MRA or DSA.
5. Posterior circulation Alberta Stroke Program Early CT Score (pc-ASPECTS) of ≥6 and Pons-Midbrain Index (PMI) of ≤3 on CT or diffusion-weighted imaging (DWI).
6. Randomization within 24 hours of stroke progression.
7. Pre-stroke mRS score of 0-2.
8. Provision of signed informed consent by the patient or their legal representative.

Exclusion Criteria:

1. Any sign of intracranial hemorrhage (except microbleeds) on baseline brain imaging.
2. Imaging confirms the progression of symptoms caused by intracranial hemorrhage, brain edema, or other clear causes.
3. Extensive cerebellar infarction with significant mass effect or bilateral thalamic infarction on baseline neuroimaging.
4. Presence of untreated intracranial aneurysm, intracranial tumor (except small meningioma and aneurysms <3 mm in diameter), or intracranial arteriovenous malformation.
5. Known or highly suspected chronic responsible artery occlusion.
6. Presence of severe stenosis in the extracranial or intracranial segment of the responsible artery, arterial dissection, or excessive vascular tortuosity that may prevent successful delivery or navigation of endovascular devices.
7. Known contraindication to contrast medium (except mild rash).
8. Refractory hypertension not controlled by medication, defined as systolic blood pressure >185 mmHg or diastolic blood pressure >110 mmHg.
9. Known pregnancy or lactation, or a positive pregnancy test prior to randomization.
10. Known dementia or psychiatric disease precluding completion of neurological assessment and follow-up.
11. Life expectancy <1 year, including patients with malignancy or advanced cardiopulmonary disease.
12. Current participation in any other clinical trial of drugs or medical devices, or anticipated participation in another such trial within 3 months after enrollment.
13. Acute ischemic stroke within 48 hours after cardiovascular or cerebrovascular interventional treatment or major surgery; patients presenting >48 hours after such procedures were eligible.
14. Recent (within 1 month) gastrointestinal or genitourinary bleeding, acute myocardial infarction, or traumatic brain injury.
15. Multivessel severe stenosis or occlusion confirmed by CTA, MRA, or DSA.
16. Known or suspected central nervous system vasculitis.
17. Known hereditary or acquired bleeding diathesis, coagulation factor deficiency, current use of oral anticoagulants with an INR >1.5, or active bleeding.
18. Blood glucose <2.8 or >22.2 mmol/L; platelet count <100 × 10⁹/L; estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m² or serum creatinine ≥177 μmol/L (2.0 mg/dL).
19. Any other condition deemed by the site investigator to make the patient unsuitable for participation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Endovascular Treatment Group; Participants randomized to the endovascular recanalization arm will receive endovascular treatment in addition to medical therapy. The procedure aims to achieve recanalization of the occluded vertebrobasilar artery using mechanical thrombectomy techniques, including stent retriever and/or aspiration-based approaches. When necessary, adjunctive endovascular measures may be performed as rescue therapy at the discretion of the treating neurointerventionalists.	Procedure: Endovascular Recanalization; The endovascular approach is selected by the treating neurointerventionalists based on angiographic findings, occlusion characteristics, and procedural feasibility. Permitted techniques include mechanical thrombectomy using stent retriever and/or aspiration-based methods. Adjunctive endovascular procedures, such as balloon angioplasty, stent deployment, or intraarterial thrombolysis, may be used when deemed necessary to achieve or maintain vessel patency. Angiographic reperfusion is assessed during the procedure, and treatment is terminated once adequate revascularization is obtained. Subsequent medical therapy is individualized according to stroke mechanism, procedural findings, and post-treatment imaging.
Experimental: Medical Therapy Alone Group; Participants randomized to this arm will receive medical therapy alone, consistent with current guideline recommendations for the acute and secondary prevention phases of ischemic stroke. No endovascular recanalization procedures are permitted after randomization.	Drug: Medical Therapy Alone; Medical therapy consists of comprehensive evidence-based medical therapy for acute ischemic stroke, encompassing acute supportive care, neurological and physiological monitoring, etiological evaluation, and secondary prevention strategies. Standard pharmacological treatments are administered as appropriate, together with risk factor modification and supportive care measures, in accordance with current guideline recommendations. Endovascular recanalization procedures are not included in this treatment strategy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The proportion of patients achieving modified Rankin Scale (mRS) score 0-3 at 90 days	The modified Rankin scale (mRS) ranged from 0 to 6, with a score of 0 indicating no disability, 1 no clinically significant disability, 2 slight disability, 3 moderate disability but remaining able to walk unassisted, 4 moderately severe disability,5 severe disability, and 6 death.	90±14 days after randomization

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ordinal shift analysis of mRS score at 90 days after randomization	The modified Rankin Scale (mRS) ranged from 0 to 6, with a score of 0 indicating no disability, 1 no clinically significant disability, 2 slight disability, 3 moderate disability but remaining able to walk unassisted, 4 moderately severe disability, 5 severe disability, and 6 death.	90±14 days after randomization
The proportion of patients achieving mRS scores 0-1 and 0-2 at 90 days	The modified Rankin Scale (mRS) ranged from 0 to 6, with a score of 0 indicating no disability, 1 no clinically significant disability, 2 slight disability, 3 moderate disability but remaining able to walk unassisted, 4 moderately severe disability, 5 severe disability, and 6 death.	90±14 days after randomization
The National Institutes of Health Stroke Scale (NIHSS) score at 24 hours	National Institutes of Health Stroke Scale (NIHSS) score at 24 hours after randomization The National Institute of Health Stroke Scale (NIHSS) ranged from 0 to 42, with higher scores indicating greater neurologic deficits.	24 (-6/+12) hours after randomization
The NIHSS score at 7 days after randomization or discharge (whichever came first), as well as changes from baseline	NIHSS score at 7 days after randomization or discharge (whichever came first) The National Institute of Health Stroke Scale (NIHSS) ranged from 0 to 42, with higher scores indicating greater neurologic deficits.	7 days after randomization or at discharge
The score on the European Quality of life 5-Dimension 5-Level (EQ-5D-5L) patient-reported questionnaire score at 90 days	European Quality of life 5-Dimension 5-Level (EQ-5D-5L) is a standardized instrument for measuring the general health status. Rated level can be coded as a number 1, 2, 3, 4 or 5, which indicates having no problems for 1, having some problems for 2, having moderate problems for 3, having serious problems for 4 and having extreme problems for 5.	90±14 days after randomization
The proportion of Barthel index 95-100 points at 90 days	The proportion of Barthel Index 95-100 at 90 days after randomization.	90±14 days after randomization
The recanalization of the vertebrobasilar artery at 24 hours after randomization (confirmed by CTA, MRA, DSA, or TCD)	The recanalization of the vertebrobasilar artery at 24 hours after randomization (confirmed by CTA, MRA, DSA or TCD).	24 (-6/+12) hours after randomization
The rate of technical success is defined as the successful recanalization of the target vessel at the end of the procedure (expanded Thrombolysis in Cerebral Infarction scale [eTICI] 2b-3)	Technical success rate, defined as successful recanalization of target vessels at the end of procedure (The expanded Thrombolysis in Cerebral Infarction scale [eTICI] 2b-3).	At the end of the procedure

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
The rate of mortality within 90 days after randomization	All-cause mortality within 90 days after randomization.	90±14 days after randomization
The incidence of any symptomatic intracranial hemorrhage (sICH) defined as the Heidelberg classification within 24 hours	Heidelberg standard was defined as new intracranial hemorrhage detected by brain imaging associated with any of the item below: 4 points of total NIHSS score at the time of diagnosis compared to immediately before worsening. 2 points of NIHSS score in one category. Leading to intubation/hemicraniectomy/ventricular drainage placement or other major medical/surgical intervention. Absence of alternative explanation for deterioration.	24 (-6/+12) hours after randomization
The rate of any intracranial hemorrhage identified by CT or MRI imaging within 24 hours	Any intracranial hemorrhage identified by CT or MRI imaging within 24 hours.	24 hours after randomization
The rate of all-cause mortality within 7 days	The rate of all-cause mortality within 7 days after randomization.	7 days after randomization or at discharge
The incidence of procedure-related complications: arterial perforation, arterial dissection, and embolism in newly developed vascular areas	The incidence of procedure-related complications: arterial perforation, arterial dissection, and embolism in newly developed vascular areas.	At the end of the operation or intraoperative

Collaborators and Investigators

• Sponsor: Feng Gao
• Investigators: Principal Investigator: Feng Gao, MD, Beijing Tiantan Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2029

Study Registration Dates

• First Submitted: April 23, 2026
• First Submitted That Met QC Criteria: April 23, 2026
• First Posted (Actual): April 29, 2026

Study Record Updates

• Last Update Posted (Actual): April 29, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Acute Ischemic Stroke; Endovascular Treatment; Vertebrobasilar Artery Occlusion
• Additional Relevant MeSH Terms: Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases; Stroke; Ischemic Stroke
• Other Study ID Numbers: HX-A-2026012

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No