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

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.

KEY INCLUSION CRITERIA: 1. 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). 2. Index stroke occurred ≥ 6 months and ≤ 84 months prior to providing informed consent. 3. Age ≥ 18 years and ≤ 75 years at the time of providing informed consent. 4. Chronic motor neurological deficit due to the index stroke. 5. Total Fugl-Meyer Motor Scale (FMMS) \> 25 and ≤ 75 at baseline. 6. Modified Rankin Score (mRS) of 2-4 at baseline. 7. 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. KEY EXCLUSION CRITERIA: 1. Stroke lesion \100 cm3 as measured by MRI. 2. Previous history of symptomatic stroke(s) with incomplete motor recovery (NOT including index stroke). 3. 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. 4. History of any neuromuscular, rheumatologic, autoimmune, orthopedic, or other disease or condition that limits motor function. 5. 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. 6. Intracoronary and/or other intra-arterial stent placement within 12 months of study Screening/Baseline visits requiring uninterrupted anticoagulant and/or anti-platelet therapy. 7. Contracture involving a shoulder, elbow, wrist, finger, hip, knee, and/or ankle. 8. Any contraindication to stereotactic brain surgery. 9. 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.