A Phase 1 Dose-escalation, Single-Center, Open-labeled Study to Evaluate the Safety and Tolerability of Human Induced Pluripotent Stem Cell-derived Human Forebrain Neural Progenitor Cell Injection (hNPC01) in Chronic Ischemic Stroke

Hopstem Biotechnology Inc.1 site in 1 country21 target enrollmentNovember 9, 2023

ConditionsChronic Ischemic Stroke

InterventionshNPC01

DrugshNPC01

Overview

Phase: Phase 1
Intervention: hNPC01
Conditions: Chronic Ischemic Stroke
Sponsor: Hopstem Biotechnology Inc.
Enrollment: 21
Locations: 1
Primary Endpoint: Incidence of Treatment-Emergent Adverse Events as assessed by physiological parameters during 6 months after intracerebral injection of hNPC01
Status: Recruiting
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

The principal aims of the clinical investigation involve assessing the safety profile and MTD of human forebrain neural progenitor cells (hNPC01) administered at escalated doses via single-dose intracerebral injection to subjects with stable chronic ischemic stroke.

Detailed Description

The primary aims of this open-label single-armed study is to evaluate the safety and tolerability up to 4 cohorts of escalating doses of hNPC01 administered at a single dose via intracerebral injection to subjects with chronic cerebral ischemic stroke, and to determine MTD. The exploratory aim is to explore the evaluation tools with preliminary efficacy assessment and the potential of hNPC01 to mitigate the symptoms, especially motor symptoms associated with chronic stroke. Eligible participants for this study are individuals who have experienced a single ischemic stroke 6 to 60 months prior of enrollment and have exhibited no significant improvement following standard physical therapy interventions.

Registry

clinicaltrials.gov

Start Date

November 9, 2023

End Date

December 30, 2025

Last Updated

2 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Hopstem Biotechnology Inc.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Subject or his/her legal guardian must understand and voluntarily sign an Informed Consent Form (ICF) prior to any study-related assessments/procedure being conducted;
•Age 30-65 years old, male or female; at the time of signing the ICF;
•Female patients with negative blood pregnancy test and women in the reproductive period need to adopt effective contraceptive methods; Or has become infertile (menopausal for at least 2 years or has undergone hysterectomy, oophorectomy, or sterilization surgery); Male patients who have no plans to donate sperm or have children within 30 days before the trial and six months after the trial end need to adopt effective contraceptive methods;
•Documented history of completed ischemic stroke in the subcortical and/or cortical regions of the middle cerebral artery (MCA) or lenticulostriate arteries confirmed by head CT or magnetic resonance imaging;
•6 - 60 months since the acute onset of ischemic stroke, accompanied by unilateral limb motor dysfunction, with/without aphasia;
•Modified Rankin Score 3, or 4 points at time of screening;
•FMMS scores lower than 55 and score changes no greater than 5 points in two assessments at least 3 weeks apart before surgery;
•NIHSS score change is no greater than 4 points in two assessments at least 3 weeks apart before surgery;
•Subject must have maintained regular physical therapy and rehabilitation prior to enrollment and be able to continue physical therapy and rehabilitation after treatment.

Exclusion Criteria

•Has any psychological or psychiatric condition that would significantly interfere with the study;
•History of more than one symptomatic stroke \[transient ischemic attacks (TIAs) are not exclusionary\];
•Has signs and symptoms of intracerebral hernia or elevated intracerebral pressure;
•History of epilepsy or current use of antiepileptic drugs, including any seizures in the 3 months prior to screening;
•Intracerebral aneurysm or arteriovenous malformation; hemorrhagic transformation of ischemic loci within 2 weeks of screening; moyamoya disease (MMD); autosomal dominant cerebrovascular disease with subcortical infarction and leukoencephalopathy; patients with giant cerebral infarction (MRI confirms that the infarct size is greater than 150 cm\^3); CT angiography (CTA) shows severe stenosis in vital vessels in head or neck;
•Patients with severely impaired motor function of one limb caused by previous neurological diseases (such as PD, motor neuron disease, arthritis, or anatomical deformity, etc.); Patients who have undergone any major surgery 8 weeks prior to screening;
•Severe muscular atrophy or muscular dystrophy;
•Patients with severe cardiovascular diseases within 3 months of screening, such as malignant arrhythmia, atrial fibrillation, acute myocardial infarction, congestive heart failure, etc. (cardiac function grade III-IV using the New York Heart Association (NYHA) classification for heart failure);
•Patients participating in other drug or device clinical trials within the last 3 months or 5 half-lives of the drug, whichever is longer;
•Receiving other cell transfusions or alike other than transfusion in the past 5 years;

Arms & Interventions

hNPC01 therapy

1.5×10\^7 hNPC01 cells; 3.0×10\^7 hNPC01 cells; 6.0×10\^7 hNPC01 cells

Intervention: hNPC01

Outcomes

Primary Outcomes

Incidence of Treatment-Emergent Adverse Events as assessed by physiological parameters during 6 months after intracerebral injection of hNPC01

Time Frame: during 6 months after intracerebral hNPC01 injection

Secondary Outcomes

Incidence of Treatment-Emergent Adverse Events as assessed by physiological parameters during 12 months after intracerebral injection of hNPC01(during 12 months after intracerebral hNPC01 injection)