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Amyotrophic Lateral SclerosisPhase 3ALSummitNCT04745299

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

A Phase 3 study testing Lenzumestrocel and Riluzole in people with Amyotrophic Lateral Sclerosis, run by Corestemchemon, Inc. at 5 sites in South Korea.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 25 to 75 years
Conditions the study is for
Amyotrophic Lateral Sclerosis
What is being tested?
Lenzumestrocel and Riluzole
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
123 participants
Where
5 sitesSouth Korea
Study dates
24 Feb 2021to 13 Nov 2024
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 25 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

[Inclusion Criteria]

  1. Subjects who show both upper motor neuron signs and lower motor neuron signs at the same time in neurological tests.
  2. 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).
  3. For subjects who are under Riluzole treatment, those who have received stable dose of Riluzole for more than 28 days before screening visit.
  4. Subjects with duration of disease of no more than 2 years from the first diagnosis date.
  5. Subjects whose ALSFRS-R scores are in the range of 31~46 at the time of screening (P-V0).

[Exclusion Criteria]

  1. 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).
  2. Subjects who received gastrotomy at the time of screening (P-V0).
  3. 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.
  4. 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.
  5. 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).
  6. Subjects who have experienced epileptic seizure.
  7. Subjects with severe renal disorder (serum creatinine: not less than 2.0 mg/dL).
  8. Subjects with severe hepatic disorder (ALT, AST, or bilirubin: over 2.0 times of the normal upper limit).
  9. Subjects who show hemorrhagic tendency at the time of screening (PT and aPTT > 1.5 x ULN)
  10. 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.
  11. Subjects with hypersensitivity to antibiotics (penicillin or streptomycin).
  12. Subjects who have ever received any cell therapy product for the same disease.
  13. Subjects with any malignant tumor in the past 5 years before screening, except malignant tumors with very low risk of metastasis or death.

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

South Korea5 sites

  • Hanyang university hospital

    Seoul, Seoul, 04763

  • Korea University Anam Hospital

    Seoul, 02841

  • Samsung Medical Center

    Seoul, 06351

  • Seoul National University Hospital

    Seoul

  • Pusan National University Yangsan Hospital

    Yangsan, Kyungsangnam-do, 50612

Other trials in South Korea

About this study

Official title: A Double-blind, Randomized, Multicenter, Placebo-Controlled, Parallel, Phase III Clinical Trial to Evaluate the Efficacy and Safety of Lenzumestrocel(Neuronata-R® Inj.) in Patients With Amyotrophic Lateral Sclerosis

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.

Read the detailed description

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.

Being tested
Lenzumestrocel, Riluzole, Placebo Comparator
Keywords
Amyotrophic lateral sclerosis, Mesenchymal stem cell, cell therapy, ALS, neuroprotection, Phase 3 clinical trial
Medical subject headings
Amyotrophic Lateral Sclerosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • LenzumestrocelBiological

    Also called Neuronata-R inj, Autologous Bone Marrow derived Mesenchymal Stem Cell

    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

  • RiluzoleDrug

    Also called Rilutek

    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

  • Placebo ComparatorDrug

    Also called Normal Saline Inj.

    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

Study groups in NCT04745299
Study groupRoleWhat they receive
Single cycle administration groupExperimentalStudy drug injections twice in a 26-day interval followed by three times comparator injections every three months.
Receives: Biological: Lenzumestrocel; Drug: Riluzole; Drug: Placebo Comparator
Multiple administation groupExperimentalStudy drug injections twice in a 26-day interval followed by repeated three times study drug injections every three months.
Receives: Biological: Lenzumestrocel; Drug: Riluzole
Control groupPlacebo comparatorComparator injections twice in a 26-day interval followed by three times comparator injections every three months.
Receives: Drug: Riluzole; Drug: Placebo Comparator

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: at 12 months, and 6 months

Secondary outcomes (3)

  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.

    The difference in joint rank scores between multiple administration group and control group at 6 months.

    Measured: at 6 months

  2. 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)

    Measured: at 12 months, 6 months

  3. 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)

    Measured: at 12 months, 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Feb 2021
  2. Study start24 Feb 2021
  3. Primary completion3 Oct 2024
  4. Study completion13 Nov 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
123 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04745299
Sponsor's protocol ID
NEURONATA-R_ALS301
Acronym
ALSummit
Status last verified
Apr 2026
First submitted
27 Jan 2021
Last updated
30 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 35585556

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Lenzumestrocel and Riluzole in people with Amyotrophic Lateral Sclerosis, run by Corestemchemon, Inc. at 5 sites in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Joint rank scores (CAFS, Combined Assessment of Functional and Survival) (time frame: at 12 months, and 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04745299, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Apr 2026; AzTrials last refreshed its copy on 8 Nov 2026.

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