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Amyotrophic Lateral SclerosisPhase 1NCT06676423

Evaluate the Safety of Neuronata-R® Inj. Suspended With HypoTHermosol® FRS (HTS-FRS) in Patients With ALS

A Phase 1 study testing Lenzumestrocel and Riluzole in people with Amyotrophic Lateral Sclerosis, run by Corestemchemon, Inc. at 1 site 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

This clinical trial is a single-center, open-label, and phase I clinical trial to Evaluate the Safety of Neuronata-R® Inj. suspended with HypoTHermosol® FRS (HTS-FRS) in Patients with Amyotrophic Lateral Sclerosis. 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
Could I receive a placebo?
No, everyone receives an active treatment
How many people
8 participants
Where
1 siteSouth Korea
Study dates
9 Nov 2022to 27 Nov 2023
Phase
Phase 1
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

Who may be able to join

  • Among subjects diagnosed with familial or sporadic amyotrophic lateral sclerosis
  • Subjects whose ALSFRS-R scores are in the range of 25~46 at the time of screening (Visit 1).
  • Subjects who are able to visit the site by themselves or with other's support.
  • When subjects and/or their legal guardians consent to participating in this clinical study.
  • Subjects deemed, by the investigator, capable of complying with the clinical study protocol
  • For child-bearing aged female subjects: Subjects who consent to sexual abstinence (refraining from sexual intercourse) or use of contraception method with annual failure rate of < 1% during the study period.
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  • A female subject who has experienced the menarche, does not reach the menopause (or 12-month or longer amenorrhea for unknown reasons except menopause) and does not receive surgical sterilization (ovariectomy and/or hysterectomy) is regarded as the child-bearing aged woman.
  • Examples of contraception methods with annual failure rate of < 1% include bilateral tubal ligation, vasectomy, appropriate use of hormonal contraceptives that inhibit ovulation (supplemented by barrier method and spermicide), hormone-releasing intrauterine device and copper intrauterine device.
  • Based on clinical study period and subject's preferred lifestyle, the reliability of sexual abstinence should be evaluated. Periodic abstinence (e.g., calendar method, ovulation and post-ovulation symptothermal method) and extravaginal ejaculation are not acceptable contraception methods.
  • For male subjects: Subjects who, as described below, consent to sexual abstinence (refraining from sexual intercourse) or use of contraception method and consent to refrain from donation of sperm.
  • When a male subject has his child-bearing aged female partner or pregnant female partner, he should maintain sexual abstinence or use condom to avoid exposure to embryo. Such male subject should not donate sperm during this period.
  • Based on clinical study period and subject's preferred lifestyle, the reliability of sexual abstinence should be evaluated. Periodic abstinence and extravaginal ejaculation are not acceptable contraception methods.

Who cannot join

  • Subjects who fail to satisfy ALS diagnosis criteria according to the revised World Federation of Neurology El Escorial Criteria [Rix Brooks, 2000].
  • Subjects expected to have side effects on administration of cell therapy (such as subjects suspected to have malignant tumor, high-risk subjects vulnerable to psychogenic shock and severe hypertension subjects).
  • ALSFRS-R score of less than 25 and 47 or higher during screening (visit 1)
  • Subjects who fall into above Class II according to the New York Heart Association's functional classification (see the attachment), 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 (Visit 1) or who received stent insertion or coronary artery bypass grafting.
  • Subjects who have experienced epileptic seizure.
  • Subjects with severe renal disorder (serum creatinine: not less than 3.0 mg/dL).
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  • 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 (such as 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 with any malignant tumor in the past 5 years before screening, except malignant tumors with very low risk of metastasis or death (such as appropriately treated cervical intraepithelial neoplasia, skin basal or squamous cell carcinoma, localized prostate cancer or ductal carcinoma in situ).
  • Subjects who are receiving any medicinal products that may affect bone marrow functions.
  • Subjects with severe mental disorders (such as schizophrenia and bipolar disorder. However, exception applies to mild ALS-related cognitive impairment and secondary emotional trauma).
  • Subjects for whom administration of investigational product is prohibited, subjects with conditions that may affect interpretation of results or subjects with conditions that may result in high risk of complications, such as the rest diseases, metabolic disorders, physical examination results and/or laboratory test results as diseases or such conditions are reasonably suspected.

Trial locations and contacts

1 site in 1 country.

South Korea1 site

  • Hanyang university hospital

    Seoul, 04763

Other trials in South Korea

About this study

Official title: A Phase I Clinical Trial to Evaluate the Safety of Neuronata-R® Inj. Suspended With HypoTHermosol® FRS (HTS-FRS) in Patients With Amyotrophic Lateral Sclerosis

This clinical trial is a single-center, open-label, and phase I clinical trial to Evaluate the Safety of Neuronata-R® Inj. suspended with HypoTHermosol® FRS (HTS-FRS) in Patients with Amyotrophic Lateral Sclerosis.

Read the detailed description

Neuronata-R is produced by mixing the patient's own cerebrospinal fluid with cultured mesenchymal stem cells the day before administration. Therefore, as autologous cerebrospinal fluid is collected from the patient for drug production at each administration, the patient must endure the occurrence of adverse events (headache, pain, etc.) and pain caused by collection, and problems such as fatigue and time consumption of the medical staff's procedure have continuously emerged.

Therefore, a comparative equivalence tests and stability tests were conducted with HypoThermosol® FRS (HTS-FRS) as a suspension agent for Neuronata-R, and it was confirmed that there was no difference in the quality of the finished product depending on the type of additive and Neuronata-R using autologous cerebrospinal fluid as a suspension agent, and it was confirmed that it was safe as an additive through non-clinical trials.

The dose determination for the safety evaluation of HypoThermosol® FRS (HTS-FRS), which will be used as a suspension, was determined in consultation with the MFDS to use a 1:1 mixture of autologous CSF and HypoThermosol® FRS (HTS-FRS) as a suspension in the first stage dose, and only HypoThermosol® FRS (HTS-FRS) was used as a suspension in the second stage dose without cerebrospinal fluid. And, the administration period is follow-up for 4 weeks after administration twice every 26 days.

Being tested
Lenzumestrocel, Riluzole
Keywords
Amyotrophic Lateral Sclerosis, Mesenchymal stem cell, cell theraphy, ALS, HypoThermosol(HTS-FRS), Phase 1 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, Autologous Bone Marrow derived Mesenchymal Stem cell

    1.0 ⅹ 10^6 cells/kg of Neuronata-R mixed with HTS-FRS suspension are administered twice in the cerebrospinal fluid at intervals of 26 days

  • RiluzoleDrug

    Also called Rilutek

    In the case of a subject who is not taking Riluzole at the time of screening (visit 1), Riluzole should be taken except when discontinued due to Adverse events. The dose can be changed within the authorization according to the medical expert's judgement

Study groups in NCT06676423
Study groupRoleWhat they receive
Groups/CohortsExperimentalIn the first-stage dose, a 1:1 mixture of autologous CSF and HypoThermosol® FRS (HTS-FRS) is used as a suspension, and in the second-stage dose, only HypoThermosol® FRS (HTS-FRS) is used as a suspension without cerebrospinal fluid. Mix 1.0 ⅹ 10^6 cells per kg of body weight with the suspension at the dose level below 1. First stage dose: HTS-FRS 0.5mL/10kg + cerebrospinal fluid 0.5mL/10kg 2. Second stage dose: HTS-FRS 1.0mL/10kg
Receives: Biological: Lenzumestrocel; Drug: Riluzole

What is being measured

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

  1. tolerability Assessment

    HTS-FRS DLT expression for each dose step

    Measured: through study period, an average of 8week

  2. Safety Assessment

    Adverse Events(including DLT)

    Measured: through study period, an average of 8week

  3. Safety Assessment

    Clinical Laboratory Analysis(Change in laboratory test results at the time of visit after IP administration compared to baseline)

    Measured: Baseline and 26day, 8week

  4. Safety Assessment

    Cerebrospinal Fluid Analysis(Changes in cerebrospinal fluid results at 26day compared to 0day)

    Measured: 0 Day, 26 Day

Secondary outcomes (2)

  1. Exploratory Assessment

    Change in total ALSFRS-R score from before treatment (screening) to after administration

    Measured: baseline and 26day , 8week

  2. Exploratory Assessment

    Change rate in ALSFRS-R score from before treatment (screening) to after administration

    Measured: within -8week and 0day, 8week

Timeline

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

  1. First posted6 Nov 2024
  2. Study start9 Nov 2022
  3. Primary completion27 Nov 2023
  4. Study completion27 Nov 2023

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 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
8 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06676423
Sponsor's protocol ID
NEURONATA-ALSHT122
Status last verified
Nov 2024
First submitted
16 Jun 2024
Last updated
6 Nov 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Lenzumestrocel and Riluzole in people with Amyotrophic Lateral Sclerosis, run by Corestemchemon, Inc. at 1 site 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?

The sponsor lists 12 inclusion and 14 exclusion criteria. 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 6 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

tolerability Assessment (time frame: through study period, an average of 8week). The study lists 4 primary outcomes in all. 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 NCT06676423.

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 6 Nov 2024.

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