Phase Ib/IIa Clinical Study of ApTOLL for the Treatment of Acute Ischemic Stroke

NCT ID: NCT04734548

Last Updated: 2022-10-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

151 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-10-28

Study Completion Date

2022-09-07

Brief Summary

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This is a prospective, multicenter, double-blind, randomized, placebo-controlled, Phase Ib/IIa clinical study to assess the administration of ApTOLL together with endovascular therapy in acute ischemic stroke patients who are candidates to receive reperfusion therapies.

Detailed Description

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This is a prospective, multicenter, double-blind, randomized, placebo-controlled, Phase Ib/IIa clinical study to assess the administration of ApTOLL together with endovascular therapy in acute ischemic stroke (AIS) patients with confirmed Large Vessel Occlusion (LVO) who are candidates to receive reperfusion therapies including endovascular treatment with or without i.v. rt-PA (recombinant tissue Plasminogen Activator).

The study will be a Phase Ib/IIa trial where 2 doses selected, based on safety criteria, on Phase Ib will be administered in the following Phase IIa.The objective of the study is to evaluate if administration of ApTOLL at different doses is safe and well tolerated compared to placebo when administered with endovascular therapy (EVT), with or without i.v. rt-PA, in the AIS target population.

Conditions

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Stroke Acute Stroke Ischemic Stroke

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Phase Ib ApTOLL

ApTOLL is administered intravenously in a single ascending dose pattern in four dose levels (0.025mg/kg - 0.2mg/kg). All levels include six patients.

Group Type ACTIVE_COMPARATOR

ApTOLL

Intervention Type DRUG

ApTOLL is a Toll-like receptor 4 (TLR4) antagonist, a receptor that is involved in innate immune responses but also responds to tissue damage, and therefore it is directly involved in a large number of diseases where the inflammatory response is involved. ApTOLL has demonstrated specific binding to human TLR4 as well as a TLR4 antagonistic effect, reducing inflammation and improving outcome after different disease models.

Phase Ib Placebo

Placebo is administered intravenously in a single ascending dose pattern in four dose levels (0.025mg/kg - 0.2mg/kg). All levels include two patients.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

White freeze-dried powder which is indistinguishable to ApTOLL for taste, color, texture and size.

Phase IIa ApTOLL

ApTOLL is administered intravenously (two doses selected in Phase Ib). The two dose levels include 35 patients each one.

Group Type ACTIVE_COMPARATOR

ApTOLL

Intervention Type DRUG

ApTOLL is a Toll-like receptor 4 (TLR4) antagonist, a receptor that is involved in innate immune responses but also responds to tissue damage, and therefore it is directly involved in a large number of diseases where the inflammatory response is involved. ApTOLL has demonstrated specific binding to human TLR4 as well as a TLR4 antagonistic effect, reducing inflammation and improving outcome after different disease models.

Phase IIa Placebo

Placebo is administered intravenously in one arm which includes 49 patients.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type OTHER

White freeze-dried powder which is indistinguishable to ApTOLL for taste, color, texture and size.

Interventions

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ApTOLL

ApTOLL is a Toll-like receptor 4 (TLR4) antagonist, a receptor that is involved in innate immune responses but also responds to tissue damage, and therefore it is directly involved in a large number of diseases where the inflammatory response is involved. ApTOLL has demonstrated specific binding to human TLR4 as well as a TLR4 antagonistic effect, reducing inflammation and improving outcome after different disease models.

Intervention Type DRUG

Placebo

White freeze-dried powder which is indistinguishable to ApTOLL for taste, color, texture and size.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

1. Age ≥18 and ≤90 years.
2. Informed consent obtained from subject or acceptable subject surrogate (i.e. next of kin, or legal representative).
3. A new focal disabling neurologic deficit consistent with acute cerebral ischemia.
4. Baseline NIHSS obtained prior to randomization ≥ 8 points and ≤ 25 points.
5. Pre-stroke mRS score of 0 - 2.
6. Treatable as soon as possible and at least within 6 hours of symptom onset, defined as point in time when the subject was last seen well (at baseline).
7. Patients should be candidates to receive EVT treatment with or without i.v. rt-PA.
8. Occlusion (TICI 0 or TICI 1 flow), of the terminal internal carotid artery (TICA), M1 or M2 segments of the middle cerebral artery, suitable for mechanical embolectomy, confirmed on Computed Tomography Angiography.
9. The following imaging criteria should also be met on admission neuroimaging:

1. MRI criterion: volume of DWI (Diffusion-weighted Imaging) restriction ≥5 mL and ≤70 mL OR
2. CT criterion: Alberta Stroke program early CT score (ASPECTS) 6 to 10 on baseline CT AND infarct core determined on admission CTPerfusion by Cerebral Blood Flow\<30%: ≥5 mL and ≤70 mL.
10. The subject has an indication and is planned to receive endovascular treatment of stroke according to the European Stroke Organization Guidelines.

Exclusion Criteria

1. Subject has suffered a stroke in the past 1 year.
2. Occlusion (TICI 0 or TICI 1 flow) of the basilar or vertebral or posterior or anterior cerebral arteries.
3. Clinical symptoms suggestive of bilateral stroke or stroke in multiple territories.
4. Known hemorrhagic diathesis, coagulation factor deficiency, or oral anticoagulant therapy with INR (international normalized ratio)\>3.0.
5. Baseline platelet count \<50,000/μL.
6. Baseline blood glucose of \<50 mg/dL or \>400 mg/dL.
7. Severe, sustained hypertension (systolic blood pressure \>185 mmHg or diastolic blood pressure \>110 mmHg).
8. Serious, advanced, or terminal illness with anticipated life expectancy of less than 1 year.
9. Subjects with identifiable intracranial tumors.
10. History of life-threatening allergy (more than rash) to contrast medium.
11. Known renal insufficiency with creatinine ≥3 mg/dL or Glomerular Filtration Rate (GFR) \<30 mL/min.
12. Cerebral vasculitis.
13. Evidence of active systemic infection.
14. Known current use of cocaine at time of treatment.
15. Patient participating in a study involving an investigational drug or device that would impact this study.
16. Patients that are unlikely to be available for a 90-day follow-up (e.g. no fixed home address, visitor from overseas).
17. Female who is pregnant or lactating or has a positive pregnancy test at time of admission.
18. CT or MRI evidence of hemorrhage (the presence of microbleeds is allowed).
19. Significant mass effect with midline shift.
20. Suspicion of aortic dissection presumed septic embolus, or suspicion of bacterial endocarditis.
Minimum Eligible Age

18 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ministry of Science and Innovation, Spain

OTHER_GOV

Sponsor Role collaborator

Anagram

UNKNOWN

Sponsor Role collaborator

aptaTargets S.L.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Marc Ribó, MD, PhD

Role: STUDY_DIRECTOR

aptaTargets S.L.

Macarena Hernández, PhD

Role: STUDY_DIRECTOR

aptaTargets S.L.

Locations

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Centre Hospitalier Régional Universitaire de Lille

Lille, , France

Site Status

Foundation Adolphe de Rothschild

Paris, , France

Site Status

Centre Hospitalier Universitaire de Toulouse

Toulouse, , France

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Hospital Universitario A Coruña

A Coruña, , Spain

Site Status

Hospital Universitario Central de Asturias

Asturias, , Spain

Site Status

Hospital Bellvitge

Barcelona, , Spain

Site Status

Hospital Germans Trias i Pujol

Barcelona, , Spain

Site Status

Hospital Universitario Vall d´Hebron

Barcelona, , Spain

Site Status

Hospital Universitario de Gerona Dr. Josep Trueta

Girona, , Spain

Site Status

Hospital Universitario 12 de Octubre

Madrid, , Spain

Site Status

Hospital Universitario La Princesa

Madrid, , Spain

Site Status

Hospital Universitario Ramón y Cajal

Madrid, , Spain

Site Status

Hospital Virgen del Rocío

Seville, , Spain

Site Status

Hospital Universitario y Politécnico La Fe

Valencia, , Spain

Site Status

Hospital Clínico Valladolid

Valladolid, , Spain

Site Status

Countries

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France Germany Spain

References

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Hernandez-Jimenez M, Martin-Vilchez S, Ochoa D, Mejia-Abril G, Roman M, Camargo-Mamani P, Luquero-Bueno S, Jilma B, Moro MA, Fernandez G, Pineiro D, Ribo M, Gonzalez VM, Lizasoain I, Abad-Santos F. First-in-human phase I clinical trial of a TLR4-binding DNA aptamer, ApTOLL: Safety and pharmacokinetics in healthy volunteers. Mol Ther Nucleic Acids. 2022 Mar 9;28:124-135. doi: 10.1016/j.omtn.2022.03.005. eCollection 2022 Jun 14.

Reference Type BACKGROUND
PMID: 35402075 (View on PubMed)

Hernandez-Jimenez M, Abad-Santos F, Cotgreave I, Gallego J, Jilma B, Flores A, Jovin TG, Vivancos J, Hernandez-Perez M, Molina CA, Montaner J, Casariego J, Dalsgaard M, Liebeskind DS, Cobo E, Castellanos M, Portela PC, Masjuan J, Moniche F, Tembl JI, Terceno Izaga M, Arenillas JF, Callejas P, Olivot JM, Calviere L, Henon H, Mazighi M, Pineiro D, Pugliese M, Gonzalez VM, Moro MA, Garcia-Tornel A, Lizasoain I, Ribo M. Safety and Efficacy of ApTOLL in Patients With Ischemic Stroke Undergoing Endovascular Treatment: A Phase 1/2 Randomized Clinical Trial. JAMA Neurol. 2023 Aug 1;80(8):779-788. doi: 10.1001/jamaneurol.2023.1660.

Reference Type DERIVED
PMID: 37338893 (View on PubMed)

Hernandez-Jimenez M, Abad-Santos F, Cotgreave I, Gallego J, Jilma B, Flores A, Jovin TG, Vivancos J, Molina CA, Montaner J, Casariego J, Dalsgaard M, Hernandez-Perez M, Liebeskind DS, Cobo E, Ribo M. APRIL: A double-blind, placebo-controlled, randomized, Phase Ib/IIa clinical study of ApTOLL for the treatment of acute ischemic stroke. Front Neurol. 2023 Feb 24;14:1127585. doi: 10.3389/fneur.2023.1127585. eCollection 2023.

Reference Type DERIVED
PMID: 36908619 (View on PubMed)

Duran-Laforet V, Pena-Martinez C, Garcia-Culebras A, Alzamora L, Moro MA, Lizasoain I. Pathophysiological and pharmacological relevance of TLR4 in peripheral immune cells after stroke. Pharmacol Ther. 2021 Dec;228:107933. doi: 10.1016/j.pharmthera.2021.107933. Epub 2021 Jun 24.

Reference Type DERIVED
PMID: 34174279 (View on PubMed)

Related Links

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http://aptatargets.com

Biotechnology company specialized in therapeutic aptamers

Other Identifiers

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APRIL

Identifier Type: -

Identifier Source: org_study_id

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