Evaluation the Efficacy and Safety of Mutiple Lenzumestrocel (Neuronata-R® Inj.) Treatment in Patients With ALS

NCT04745299 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: NEURONATA-R_ALS301
• Study Type: interventional
• Target: 123
• Locations: 1 country
• Sites: 5

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic lateral sclerosis; Mesenchymal stem cell; cell therapy; ALS; neuroprotection; Phase 3 clinical trial

Outcome Measures

Primary Outcomes (1)

• 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

  at 12 months, and 6 months

Secondary Outcomes (3)

• Joint rank scores (CAFS, Combined Assessment of Functional and Survival)

  at 6 months

• Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score

  at 12 months, 6 months

• Time to event

  at 12 months, 6 months

Other Outcomes (7)

• Slow Vital Capacity (SVC)

  6 months, 12 months, 36 months

• Muscular strength

  6 months, 12 months, 36 months

• Time to event

  36 months

Study Arms (3)

Single cycle administration group

EXPERIMENTAL

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

Multiple administation group

EXPERIMENTAL

Study drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months.

Biological: Lenzumestrocel; Drug: Riluzole

Control group

PLACEBO COMPARATOR

Comparator injections twice in a 26-day interval followed by three times comparator injections every three months.

Drug: Riluzole; Drug: Placebo Comparator

Interventions

Lenzumestrocel BIOLOGICAL

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

Multiple administation group; Single cycle administration group

Riluzole DRUG

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

Control group; Multiple administation group; Single cycle administration group

Placebo Comparator DRUG

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.

Control group; Single cycle administration group

Eligibility Criteria

• Age: 25 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who show both upper motor neuron signs and lower motor neuron signs at the same time in neurological tests.
• Among subjects diagnosed with familial or sporadic ALS, subjects falling into clinically definite ALS, probable ALS and probable ALS-lab supported according to The revised World Federation of Neurology El Escorial Criteria\[Rix Brooks, 2000\], during 17-weeks period prior to the administration and ALSFRS-R score (progression rate) of 1.03 ± 50%/month (meaning mean value in that total period).
• For subjects who are under Riluzole treatment, those who have received stable dose of Riluzole for more than 28 days before screening visit.
• Subjects with duration of disease of no more than 2 years from the first diagnosis date.
• Subjects whose ALSFRS-R scores are in the range of 31\~46 at the time of screening (P-V0).

You may not qualify if:

• Subjects who received tracheostomy or use ventilators (including positive pressure ventilators; subjects who use non-invasive ventilation for sleep apnea may be allowed after review) at the time of screening (P-V0).
• Subjects who received gastrotomy at the time of screening (P-V0).
• Subjects for whom clinical efficacy evaluation is not possible because pulmonary functional tests cannot be conducted at the time of screening (P-V0) or subjects whose forced vital capacity is found to be not greater than 40%of the expected value.
• Subjects who fall into above Class II according to the New York Heart Association's functional classification, who have showed myocardial infarction, unstable arrhythmia and/or other significant cardiovascular diseases such as unstable angina in the past 3 months, or who show electrocardiographic signs of myocardial infarction or angina at the time of screening (P-V0) or who received stent insertion or coronary artery bypass grafting.
• Subjects who have received other investigational products or edaravone within 3 month or 5 half-lives at the time of screening (P-V0) and lead in period visit L-V1 (evaluated by whichever is longer).
• Subjects who have experienced epileptic seizure.
• Subjects with severe renal disorder (serum creatinine: not less than 2.0 mg/dL).
• Subjects with severe hepatic disorder (ALT, AST, or bilirubin: over 2.0 times of the normal upper limit).
• Subjects who show hemorrhagic tendency at the time of screening (PT and aPTT \> 1.5 x ULN)
• Subjects who are found to have active viral infections (HBsAg, HCV Ab, HIV Ab, CMV IgM, EBV IgM, HSV IgM and Treponema pallidum) at the time of screening.
• Subjects with hypersensitivity to antibiotics (penicillin or streptomycin).
• Subjects who have ever received any cell therapy product for the same disease.
• Subjects with any malignant tumor in the past 5 years before screening, except malignant tumors with very low risk of metastasis or death.

Sponsors & Collaborators

• Corestemchemon, Inc.: lead

Study Sites (5)

Pusan National University Yangsan Hospital

Yangsan, Kyungsangnam-do, 50612, South Korea

Location

Hanyang university hospital

Seoul, Seoul, 04763, South Korea

Location

Korea University Anam Hospital

Seoul, 02841, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

Related Publications (1)

• Nam JY, Lee TY, Kim K, Chun S, Kim MS, Shin JH, Sung JJ, Kim BJ, Kim BJ, Oh KW, Kim KS, Kim SH. Efficacy and safety of Lenzumestrocel (Neuronata-R(R) inj.) in patients with amyotrophic lateral sclerosis (ALSUMMIT study): study protocol for a multicentre, randomized, double-blind, parallel-group, sham procedure-controlled, phase III trial. Trials. 2022 May 18;23(1):415. doi: 10.1186/s13063-022-06327-4.

  PMID: 35585556 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Riluzole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Thiazoles; Sulfur Compounds; Organic Chemicals; Benzothiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Seung Hyun Kim, MD, PhD

  Hanyang University Seoul Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• 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: January 27, 2021
• First Posted: February 9, 2021
• Study Start: February 24, 2021
• Primary Completion: October 3, 2024
• Study Completion: November 13, 2024
• Last Updated: April 30, 2026

Record last verified: 2026-04

Locations

KR (5)