A Phase 3 interventional study of Lenzumestrocel and Riluzole in Amyotrophic Lateral Sclerosis, sponsored by Corestemchemon, Inc.. Completed at 5 sites in South Korea. Open to participants aged 25 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-04-30.
Sponsored by Corestemchemon, Inc. · Phase 3, Interventional, and Treatment
ALSUMMIT is a double-blind, randomized, placebo-controlled, multi-center, parallel, phase III clinical trial to evaluate and confirm the efficacy and long-term safety of repeated Lenzumestrocel (Neuronata-R® inj.) treatment.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by selective and progressive loss of motor neurons. Disease progression leads to death within 2-4 years, but there exists no definite treatment so far.
Based on phase I/II clinical trial(NCT01363401), twice intrathecal autologous bone marrow-derived mesenchymal stem cells (Lenzumestrocel) injections showed significant therapeutic benefit lasting at least six months with safety in patients with ALS.
Additionally, the switch from pro- to anti-inflammatory conditions, which was indicated from the inverse correlation between TGF-β1 and MCP-1 levels after Lenzumestrocel injections in the good responder, has been considered a plausible beneficial action mechanism.
This study is designed to investigate the following. First, to reconfirm and evaluate the long-term efficacy of twice injections (single cycle) of Lenzumestrocel, group 1 will receive a single cycle injection with a 26-day interval.
Second, to evaluate the long-term safety and efficacy of Lenzumestrocel repeated injections, group 2 will receive a single cycle injection a 26-day apart followed by three times injections every three-month interval.
Group 3 will receive comparator injections.
981 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 283 are open to participants now.
This study's enrollment of 123 is above the median of 36 across 667 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →Corestemchemon, Inc. is the lead sponsor of 7 studies on the registry; 1 is open to participants now.
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[Inclusion Criteria]
[Exclusion Criteria]
Study drug injections twice in a 26-day interval followed by three times comparator injections every three months.
Biological: Lenzumestrocel · Drug: Riluzole · Drug: Placebo Comparator
Study drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months.
Biological: Lenzumestrocel · Drug: Riluzole
Comparator injections twice in a 26-day interval followed by three times comparator injections every three months.
Drug: Riluzole · Drug: Placebo Comparator
Single cycle administration group : injections twice in a 26-day interval Multiple administration group : injections twice in a 26-day interval followed by repeated three times injections every three months
Also known as: Neuronata-R inj, Autologous Bone Marrow derived Mesenchymal Stem Cell
concomitant administration of Riluzole to all groups, except subjects to whom Riluzole administration is deemed impossible owing to adverse events as determined by medical experts
Also known as: Rilutek
Single cycle administration group: Placebo comparator is injected three times every three months after injection of Lenzumestrocel twice in a 26-day interval. Control group: Placebo comparator is injected twice in a 26-day interval followed by repeated three times injcections every three months
Also known as: Normal Saline Inj.
Joint rank scores (CAFS, Combined Assessment of Functional and Survival)
Joint rank score is derived from Combined Assessment of Function and Survival. Functional assessment is based on ALSFRS-R scores and survival assessment is based on the period from randomization to physical death. Joint rank score will be calculated with ALSFRS-R score data and survival. For each pairwise comparison, a study participant is assigned a score and then the summed scores are ranked for all participants. The higher ranking, the higher score, and the lower ranking, the lower score. And the average rank score is then calculated for each treatment group. A higher mean rank score indicates that participants in that treatment group, on average, fared better. 1. The difference in joint rank scores between multiple administration group and control group at 12 months. 2. The difference in joint rank scores between single cycle administration group and control group at 6 months
Time frame: at 12 months, and 6 months
Joint rank scores (CAFS, Combined Assessment of Functional and Survival)
Joint rank score is derived from Combined Assessment of Function and Survival. Functional assessment is based on ALSFRS-R scores and survival assessment is based on the period from randomization to physical death. Joint rank score will be calculated with ALSFRS-R score data and survival. For each pairwise comparison, a study participant is assigned a score and then the summed scores are ranked for all participants. The higher ranking, the higher score, and the lower ranking, the lower score. And the average rank score is then calculated for each treatment group. A higher mean rank score indicates that participants in that treatment group, on average, fared better. The difference in joint rank scores between multiple administration group and control group at 6 months.
Time frame: at 6 months
Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score
ALSFRS-R is an instrument designed to evaluate functional status of subjects with amyotrophic lateral sclerosis (Lou Gehrig's disease), such as gross motor activity, fine motor activity, bulbar function and respiration function. It consists of 12 items; 3 items for mouth functions, 4 items for upper limb and overall fine motor functions, 2 items for lower limb functions and 3 items for respiration functions. Each item is evaluated in 5-point scale (0\~4). A higher score means a better functional status. 1. Change of ALSFRS-R score measured at baseline and 12 months (multiple administration group and control group) 2. Change of ALSFRS-R score measured at baseline and 6 months (single cycle administration and control group)
Time frame: at 12 months, 6 months
Time to event
Time to Event is defined as physical death, tracheostomy recognized as the point where disease progression functionally stops or chronic use of ventilator, whichever comes earlier. Chronic use of ventilator means that ventilator is used for more than 20 hours in a day and such use is continued for more than 30 days and that a subject in vegetative state requires full supports from other persons to maintain living. 1. Time to event for 12 months (multiple administration group and control group) 2. Time to event for 6 months (single cycle administration group and control group)
Time frame: at 12 months, 6 months
Slow Vital Capacity (SVC)
Change to SVC measured at 6, 12 months and 36 months
Time frame: 6 months, 12 months, 36 months
Muscular strength
Change of Muscular strength which is measured by Hand Held Dynamometer (HHD)
Time frame: 6 months, 12 months, 36 months
Time to event
Time to event for 36 months
Time frame: 36 months
Time to death
Time to death means the period from randomization of a subject to physical death. The comparison with the time to death between treatment groups and control group.
Time frame: 6 months, 12 months, 36 months
EuroQol Short Form (EQ-5D-5L)
EQ-5D-5L is designed to measure health conditions and it consists of 5 questions relating to mobility, self-care, usual activities, pain/discomfort and anxiety/depression. 1\~5 points are given for each question and a higher score means worse condition. Change of EQ-5D-5L from baseline to 6 month, 12 months, and 36 months.
Time frame: 6 month, 12 months, 36 months
Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40)
ALSAQ-40 is designed to evaluate disease-specific health conditions of subjects with ALS/motor neural disease. It consists of 40 questions to evaluate 5 aspects of health conditions affected by the disease. Subjects are asked to think about the difficulties they may have experienced during the last 2 weeks. Subjects are asked to indicate the frequency of each event by selecting one of 5 options (0\~4): never/rarely/sometimes/often/always or cannot do at all.
Time frame: 6 month, 12 months, 36 months
Biological test
Tests on blood samples and cerebrospinal fluid samples for exploratory investigation of biological markers in plasma, blood and CSF. Comparison of change before and after treatment. * Measurement cytokines : TGF-β1, IL-10, IL-6, TNF-α, MCP-1, IL-8, IL-1RA, MIP-1β, RANTES and IP-10 etc. * Units of Measure: pg/mL * Comparative Analysis of how much each cytokine increases or decreases after treatment compared to before treatment.
Time frame: up to 12 months after administration
This study is completed, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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Amyotrophic Lateral Sclerosis→
Corestemchemon, Inc.