A Study of NCS-01 in Patients With Acute Ischemic Stroke

A Phase 1/2 Randomized Study to Evaluate the Safety and Tolerability of Intracarotid Artery Administration of NCS-01 in Patients With Acute Ischemic Stroke

This is an initial Phase1/2 dose-finding, randomized, multi-center study to evaluate the safety and tolerability of NCS-01 in patients with acute ischemic stroke. All patients will be randomized within 48 hours of stroke onset. This study will be conducted in 2 stages.

Study Overview

• Status: Active, not recruiting
• Conditions: Ischemic Stroke
• Intervention / Treatment: Biological: NCS-01

• Study Type: Interventional
• Enrollment (Estimated): 16
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 76 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Males and females, age 18 to 85 years inclusive
• Clinical evidence of acute ischemic unilateral cerebral infarction
• - Evidence of neurologic deficits as defined by NIHSS 6 to 18 or NIHSS < 6 with Fugl-Meyer upper extremity subtotal 6 to 40 inclusive
• Women of child bearing potential who agrees to take acceptable birth control as described in the ICF
• Provide written informed consent before participation, either by patient or a legal representative

Exclusion Criteria:

• Progressive neurologic deficit
• An inability to undergo an MRI scan
• Any malignancies within the last 5 years
• Previous organ transplantation
• Participation in another clinical trial with an investigational drug, device or biologic within the preceding 3 months
• Women of childbearing potential with a positive pregnancy test
• Already dependent in activities of daily living (Rankin scale 3 or more) before the present acute stroke
• Known hypersensitivity, allergy or intolerance to the similar biologic interventions
• Any other clinically relevant acute or chronic diseases which could interfere with patients' safety during the trial, or expose them to undue risk, or which could interfere with study objectives judged by Investigator based on medical history, physical examination, laboratory tests and/or ECG

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NCS-01; human bone marrow derived cells	Biological: NCS-01; single infusion
Sham Comparator: sham; sham procedure	Biological: NCS-01; single infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse events -Safety by Incidence of Treatment-Emergent Adverse Events	Number of participants with treatment-related adverse events as assessed by CTCAE v4.0" . Incidence and nature of adverse events; vital signs;	multiple times for the duration of the study (baseline through Month 12)
National Institutes of Health Stroke Scale (NIHSS)	Change from baseline in the NIHSS NIHSS is a systematic assessment tool that provides a quantitative measure of stroke-related neurologic deficit. The score for each ability is a number between 0 and 4, 0 being normal functioning and 4 being completely impaired. The patient's NIHSS score is calculated by adding the number for each element of the scale; 42 is the highest score possible. In the NIHSS, the higher the score, the more impaired a stroke patient is.	1 hour, 3 hours, and 6 hours post infusion
Number of participants with change in in physical examination	physical examination changes General appearance ,Head, eyes, ears, nose, and throat, Respiratory, Cardiovascular, Musculoskeletal, Abdomen, Neurologic, Extremities, Dermatologic, Lymphatic)	multiple times from baseline through Month 12
Number of participants with change in Electrocardiography (ECG)	ECG (standard digital 12-lead in singlicate)	multiple times for the duration of the study (baseline through Month 12)
Number of participants with change in clinical laboratory evaluations	changes in clinical laboratory evaluations (Creatinine, Potassium(K+),Sodium (Na+) , Chloride (Cl-), Magnesium (Mg++), Calcium, Inorganic phosphate, Glucose, Urea,Bilirubin (Total) ,Bilirubin (direct), AST, ALT, GGT, Alkaline phosphatase, Total Protein Albumin,	multiple times for the duration of the study (baseline through Month 12)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with change in Modified Rankin Scale (mRS)	The modified Rankin Scale (mRS) is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered a stroke.Score Description 0 No symptoms at all; No significant disability despite symptoms; able to carry out all usual duties and activities; Slight disability; unable to carry out all previous activities, but able to look after own affairs without assistance; Moderate disability; requiring some help, but able to walk without assistance; Moderately severe disability; unable to walk without assistance and unable to attend to own bodily needs without assistance; Severe disability; bedridden, incontinent and requiring constant nursing care and attention; Dead	multiple times for the duration of the study (baseline through Month 12)
Number of participants with change in Cytokine panel (IL-1, 6, 8, etc.)	blood test for proteins that modulate the inflammatory response	multiple times for the duration of the study (baseline through Month 12)
Number of participants with change in MRI	global white matter and grey matter volumetrics; cortical thinning on MRI	multiple times for the duration of the study (baseline through Month 12)
Number of participants with change in Barthel Index	Barthel Index is a 4 point scale of Activities of Daily Living	multiple times for the duration of the study (baseline through Month 12)

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with change in Domain-specific Scales (Fugl-Meyer)	The Fugl-Meyer Assessment (FMA) is a stroke-specific, performance-based impairment index. It is designed to assess motor functioning, sensation, balance, joint range of motion and joint pain in patients with post-stroke hemiplegia (Fugl-Meyer, Jaasko, Leyman, Olsson, & Steglind, 1975; Gladstone, Danells, & Black, 2002). It is applied clinically and in research to determine disease severity, describe motor recovery, and to plan and assess treatment.	multiple times for the duration of the study (baseline through Month 12)

Collaborators and Investigators

• Sponsor: NC Medial Research Inc
• Investigators: Principal Investigator: Sean Savitz, MD, The University of Texas Health Science Center, Houston

Study record dates

Study Major Dates

• Study Start (Actual): February 27, 2020
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): October 1, 2028

Study Registration Dates

• First Submitted: April 9, 2019
• First Submitted That Met QC Criteria: April 11, 2019
• First Posted (Actual): April 16, 2019

Study Record Updates

• Last Update Posted (Estimated): September 11, 2025
• Last Update Submitted That Met QC Criteria: September 4, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• 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: NCS-01-01

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: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No