CC-101 (f/k/a NR1) Neural Stem Cell Transplantation for Adults With Chronic Ischemic Stroke

NCT07709845 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: NR1-03
• Study Type: interventional
• Target: 36
• Locations: 0 countries
• Sites: N/A

Brief Summary

The suNR1se II Study is a Phase 2b randomized, controlled, multi-center clinical trial evaluating the efficacy and safety of stereotactic intracerebral administration of CC-101 (f/k/a NR1), an investigational allogeneic neural stem cell therapy, in adults with chronic ischemic stroke and persistent motor impairment. The study is designed to assess whether administration of CC-101 immediately adjacent to the region of prior stroke injury may improve motor function recovery compared with a sham surgical control procedure. The study will also evaluate the role of short-term anti-rejection therapy with tacrolimus in subjects receiving CC-101. Approximately 36 participants will be randomized in a 1:1:1 ration to receive CC-101 plus tacrolimus, sham surgery (no CC-101) plus tacrolimus-matched placebo, or CC-101 plus tacrolimus-matched placebo. Participants will be followed for safety and functional outcomes for at least 12 months following the study procedure.

Conditions

Chronic Ischemic Stroke

Keywords

Ischemic Stroke; Neural Stem Cells; Stroke; Allogeneic Cell Therapy; Stroke Rehabilitation; Neuroregeneration; Intracerebral Transplantation; Motor Recovery; Chronic Stroke; Stroke Impairment; Stroke Mobility Impairment; Regenerative Medicine; Cell Therapy

Outcome Measures

Primary Outcomes (1)

• Total Fugl-Meyer Motor Scale (FMMS) Score on Day +365 Following Neurosurgical Intervention (CC-101 transplantation or sham surgery) on Day 0.

  The total Fugl-Meyer Motor Scale (FMMS) score (0-100; the larger the better) reflects the sum of the upper extremity (0-66) and lower extremity (0-34) FMMS scores. For each study subject, this clinical assessment of motor function on Day +365 will be compared with the baseline total FMMS score to determine the interval change in motor function.

  Baseline to Day +365.

Secondary Outcomes (7)

• Total Fugl-Meyer Motor Scale (FMMS) Score on Day +365 Following Neurosurgical Intervention (CC-101 transplantation or sham surgery) on Day 0.

  Baseline to Day +365.

• Total Fugl-Meyer Motor Scale (FMMS) Score on Day +90 and Day +182 Following Neurosurgical Intervention (CC-101 transplantation or sham surgery) on Day 0.

  Baseline to Day +90 and baseline to Day +182

• Total Fugl-Meyer Motor Scale (FMMS) Score on Day +90 and Day +182 Following Neurosurgical Intervention (CC-101 transplantation or sham surgery) on Day 0.

  Baseline to Day +90 and baseline to Day +182.

• Upper Extremity Motor Function as Assessed by the Action Research Arm Test (ARAT) on Day +365 Following Neurosurgical Intervention (CC-101 transplantation or sham surgery) on Day 0.

  Baseline to Day +365.

• Barthel Index on Day +90 and Day +182 Following Neurosurgical Intervention (CC-101 transplantation or sham surgery) on Day 0.

  Baseline to Day +90 and baseline to Day +182.

Study Arms (3)

CC-101 (f/k/a NR1) + oral Tacrolimus [ARM A]

EXPERIMENTAL

Participants will receive stereotactic intracerebral administration of investigational CC-101 allogeneic neural stem cells on Day 0 plus twice-daily oral tacrolimus anti-rejection therapy from Day -2 to Day +60.

Biological: CC-101 (f/k/a NR1) Intracerebral Transplantation; Drug: Tacrolimus

Partial thickness burr hole without any cells + oral Tacrolimus-matched placebo [ARM B]

SHAM COMPARATOR

Participants will undergo a sham surgical control procedure on Day 0 designed to preserve study blinding along with twice-daily oral tacrolimus-matched placebo from Day -2 to Day +60.

Procedure: Sham Surgical Control

CC-101 (f/k/a NR1) + oral Tacrolimus-matched placebo [ARM C]

EXPERIMENTAL

Participants will receive stereotactic intracerebral administration of investigational CC-101 allogeneic neural stem cells on Day 0 plus twice-daily oral tacrolimus-matched placebo therapy from Day -2 to Day +60.

Biological: CC-101 (f/k/a NR1) Intracerebral Transplantation

Interventions

Tacrolimus DRUG

Oral tacrolimus or tacrolimus-matched placebo initiated prior to study intervention and continued for approximately 60 days following study intervention per protocol-defined dosing and taper schedule.

CC-101 (f/k/a NR1) + oral Tacrolimus [ARM A]

Sham Surgical Control PROCEDURE

Participants will undergo a partial thickness burr hole craniotomy without disruption of the meninges or transplantation of any cells as a means to maintain treatment masking.

Partial thickness burr hole without any cells + oral Tacrolimus-matched placebo [ARM B]

CC-101 (f/k/a NR1) Intracerebral Transplantation BIOLOGICAL

Investigational allogeneic neural stem cell product administered stereotactically via intracerebral injection immediately adjacent to the prior stroke lesion.

CC-101 (f/k/a NR1) + oral Tacrolimus [ARM A]; CC-101 (f/k/a NR1) + oral Tacrolimus-matched placebo [ARM C]

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of completed ischemic subcortical stroke in the middle cerebral artery (MCA) distribution with or without cortical involvement based on magnetic resonance imaging (MRI) and/or computed tomography (CT) scan(s).
• Index stroke occurred ≥ 6 months and ≤ 84 months prior to providing informed consent.
• Age ≥ 18 years and ≤ 75 years at the time of providing informed consent.
• Chronic motor neurological deficit due to the index stroke.
• Total Fugl-Meyer Motor Scale (FMMS) \> 25 and ≤ 75 at baseline.
• Modified Rankin Score (mRS) of 2-4 at baseline.
• Subjects taking oral anti-spasticity agent (such as tizanidine hydrochloride or baclofen) must be on a stable dose for 90 days prior to a Screening/Baseline visit.

You may not qualify if:

• Stroke lesion \<1 cm3 or \>100 cm3 as measured by MRI.
• Previous history of symptomatic stroke(s) with incomplete motor recovery (NOT including index stroke).
• Complete or near complete lack of right and/or left upper extremity and/or right and/or left lower extremity movement as defined by the MRC Scale for Muscle Strength scores.
• History of any neuromuscular, rheumatologic, autoimmune, orthopedic, or other disease or condition that limits motor function.
• Acute myocardial infarction (heart attack), acute pulmonary embolism (blood clot to the lungs), acute proximal deep vein thrombosis (blood clot in the leg or arm), acute peripheral arterial occlusion, or any other type of acute blood clotting episode within six (6) months of study screening.
• Intracoronary and/or other intra-arterial stent placement within 12 months of study Screening/Baseline visits requiring uninterrupted anticoagulant and/or anti-platelet therapy.
• Contracture involving a shoulder, elbow, wrist, finger, hip, knee, and/or ankle.
• Any contraindication to stereotactic brain surgery.
• Presence of serum anti-Human Leukocyte Antigen (HLA) Class I and/or Class II antibodies to donor NR1 cells with a Luminex assay value greater than the larger of the central lab's reference ULN or 3,000 Maximum Fluorescence Intensity.

Sponsors & Collaborators

• Clarion Cells, Inc.: lead

MeSH Terms

Conditions

Ischemic Stroke; Stroke

Interventions

Tacrolimus

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Gary K Steinberg, MD, PhD

  Stanford University School of Medicine, Department of Neurosurgery

  STUDY CHAIR

Central Study Contacts

Ruth Antoine, MS, MBA

CONTACT

732-551-6611; rantoine@clarioncells.com

Steven R Deitcher, MD

CONTACT

srdeitcher@clarioncells.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Participants; non-neurosurgeon investigators; study staff responsible for safety assessments; outcome raters; Sponsor personnel involved in study conduct, data review, and study management; and other Sponsor representatives with responsibility for trial oversight will remain blinded to treatment assignment throughout the study. Neurosurgical personnel performing the study procedures and other designated personnel with responsibilities requiring knowledge of treatment assignment (e.g., preparation and administration of investigational product) will be unblinded. Procedures will be implemented to maintain the blind.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants will be randomized in a 1:1:1 allocation ratio to one of three parallel treatment groups evaluating CC-101 (f/k/a NR1) allogeneic neural stem cell therapy with or without active, short-term, oral anti-rejection drug therapy, compared with a sham surgical control. About two-thirds of study participants will receive intracerebral CC-101 transplantation, while one-third will receive sham surgery.

Study Record Dates

• First Submitted: July 12, 2026
• First Posted: July 17, 2026
• Study Start (Estimated): January 1, 2027
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2029
• Last Updated: July 17, 2026

Record last verified: 2026-07