Treatment Evaluation of Acute Stroke for Using in Regenerative Cell Elements

NCT02961504 | Completed Phase 2

• 1 other identifier: B01-03
• Study Type: interventional
• Target: 206
• Locations: 1 country
• Sites: 1

Brief Summary

The primary objectives of this study is to evaluate the efficacy of HLCM051 on functional outcome in subjects with acute ischemic stroke and to evaluate the safety of HLCM051 in subjects with acute ischemic stroke.

Conditions

Stroke, Acute

Outcome Measures

Primary Outcomes (2)

• Proportion of subjects with an excellent outcome defined by the functional assessments

  \<Excellent outcome\> is defined as mRS score of ≤1 (scale, 0 to 6), NIHSS score of ≤1 (scale, 0 to 42), and BI score of ≥95 (scale, 0 to 100).

  Day 90

• Comparison between the HLCM051 and the placebo groups in key adverse events

  within Day90

Secondary Outcomes (12)

• Proportion of subjects with an excellent outcome defined by the functional assessments

  Day 365

• Proportion of subjects exhibiting functional outcome throughout the range of mRS scores by shift analysis

  Day 90

• Proportion of subjects exhibiting functional outcome throughout the range of mRS scores by shift analysis

  Day 365

• Proportion of subjects who meet all of a mRS score of ≤2, NIHSS score improvement of ≥75% from baseline and a BI score of ≥95

  Day 90

• Proportion of subjects with a mRS score of ≤1 and a mRS score of ≤2

  Day 90

Other Outcomes (1)

• Changes in biomarkers (white blood cell populations and inflammatory markers) from baseline

  Day 2, Day 7, Day 30.

Study Arms (2)

HLCM051 (MultiStem)

EXPERIMENTAL

single dose of 1.2 billion HLCM051 cells

Biological: HLCM051

Placebo

PLACEBO COMPARATOR

a single dose of placebo

Biological: Placebo

Interventions

HLCM051 BIOLOGICAL

Within 18 to 36 hours after the onset of ischemic stroke, subjects will receive a single dose of 1.2 billion HLCM051 cells to be intravenously administered

HLCM051 (MultiStem)

Placebo BIOLOGICAL

Within 18 to 36 hours after the onset of ischemic stroke, subjects will receive a single dose of placebo to be intravenously administered

Placebo

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Japanese male or female patients 20 years of age or older;
• Clinical diagnosis of cerebral cortical ischemic stroke;
• Occurrence of an ischemic stroke with clear motor or speech deficit documented by National Institutes of Health Stroke Scale (NIHSS) score of 8 to 20 (at the baseline assessment) that did not change by ≥4 points from the screening to the baseline assessment;
• Onset of ischemic stroke must have occurred within 18 to 36 hours prior to the start of administration of the investigational product;
• Confirmation of hemispheric cortical infarct with brain magnetic resonance imaging (MRI) including diffusion-weighted imaging with b-value of 1,000 demonstrating an acute lesion measuring ≥ 2.0 cm of longest diameter;
• A modified Rankin Scale (mRS) of 0 or 1, by either self-report or family report, prior to the onset of ischemic stroke;
• Female patients who meet either:
• Not pregnant, not breastfeeding/ interrupting breastfeeding, and not planning on becoming pregnant during the trial;
• Not of childbearing potential, defined as one who has been postmenopausal for at least 1 year, or has been surgically sterilized, or has had a hysterectomy at least 3 months prior to the start of this trial; or
• If of childbearing potential, one who has agreed to follow investigator's advice and use an effective contraceptive method up to the end of the trial. Effective contraceptive methods include contraceptive methods used consistently and correctly (oral contraceptives, intrauterine devices, diaphragm, or male or female condoms), abstinence, and a sterile sexual partner;
• Male patients with female partners of childbearing potential must agree to follow investigator's advice and use adequate contraceptive methods (a combination of a condom and another form of contraception) up to the end of the trial if engaging in sexual intercourse;
• Patients or legal representatives must freely sign the informed consent form after the nature of the trial and the disclosure of his/her data have been explained;
• Willing and able to comply with all aspects of the treatment and testing schedule; and
• Willing and able to return to the trial site for the post-treatment evaluations.

You may not qualify if:

• Presence of a lacunar, a lesion of ≤ 2.0 cm of longest diameter, or a brainstem infarct on MRI as the etiology of symptoms of ischemic stroke;
• Reduced level of consciousness (score of 3 for item 1a of NIHSS);
• Occurrence of a hemorrhagic transformation as evidenced by computerized tomography (CT) or brain MRI scan that is clinically significant in the opinion of the investigator;
• Ipsilateral focal neurological deficits from prior lesions in the brain that would complicate evaluation;
• Experienced seizures since the onset of ischemic stroke;
• History of a neurological event such as stroke or clinically significant head trauma within 6 months prior to the start of screening;
• Patients who both received tPA treatment and underwent mechanical reperfusion (patients are eligible for the trial if they had only one of them, tPA treatment or mechanical reperfusion);
• Uncontrolled hypertension, defined as persistent systolic blood pressure \>220 mmHg or diastolic blood pressure \>120 mmHg, despite antihypertensive therapy;
• Blood glucose level \<50 mg/dL or \>350 mg/dL at baseline;
• Patients who have a significant comorbid medical condition(s), including, but not limited to:
• Severe kidney disease requiring hemodialysis or peritoneal dialysis;
• Advanced liver disease such as hepatitis or liver cirrhosis;
• Severe congestive heart failure or history of ejection fraction \<30%;
• Severe lung disease requiring home oxygen; or
• Active unstable angina requiring daily treatment with nitrates or other medications;

Sponsors & Collaborators

• Healios K.K.: lead

Study Sites (1)

Hokkaido University Hospital

Sapporo, 060-8648, Japan

Location

Related Publications (2)

• Houkin K, Osanai T, Uchiyama S, Minematsu K, Taguchi A, Maruichi K, Niiya Y, Asaoka K, Kuga Y, Takizawa K, Haraguchi K, Yoshimura S, Kimura K, Tokunaga K, Aoyama A, Ikawa F, Inenaga C, Abe T, Tominaga A, Takahashi S, Kudo K, Fujimura M, Sugiyama T, Ito M, Kawabori M, Hess DC, Savitz SI, Hirano T; TREASURE Study Investigators. Allogeneic Stem Cell Therapy for Acute Ischemic Stroke: The Phase 2/3 TREASURE Randomized Clinical Trial. JAMA Neurol. 2024 Feb 1;81(2):154-162. doi: 10.1001/jamaneurol.2023.5200.

  PMID: 38227308 DERIVED

• Osanai T, Houkin K, Uchiyama S, Minematsu K, Taguchi A, Terasaka S. Treatment evaluation of acute stroke for using in regenerative cell elements (TREASURE) trial: Rationale and design. Int J Stroke. 2018 Jun;13(4):444-448. doi: 10.1177/1747493017743057. Epub 2017 Nov 14.

  PMID: 29134924 DERIVED

MeSH Terms

Conditions

Stroke

Condition Hierarchy (Ancestors)

Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Vascular Diseases; Cardiovascular Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 7, 2016
• First Posted: November 11, 2016
• Study Start: November 15, 2017
• Primary Completion: March 31, 2021
• Study Completion: February 28, 2023
• Last Updated: January 17, 2024

Record last verified: 2024-01

Locations

JP (1)