Amyotrophic Lateral SclerosisEarly Phase 1SCA202401NCT06889857
Safety and Efficacy of Intravenous Administration of SHED-CM for ALS
A Early Phase 1 study testing The study drug is SHED-CM manufactured by U-Factor in people with Amyotrophic Lateral Sclerosis, run by Hitonowa Medical at 1 site in Japan.
Trial summary
This study evaluates the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media (SHED-CM) in patients with Amyotrophic Lateral Sclerosis (ALS), using the Japanese version of the revised ALS Functional Rating Scale (ALSFRS-R) as an indicator. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- The study drug is SHED-CM manufactured by U-Factor
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteJapan
- Study dates
- 5 Apr 2024to 31 Dec 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients who meet the following inclusion criteria (1) to (6) at the time of Informed consent
- Patients who have provided written informed consent to participate in the study.
- Patients who are at least 20 years of age at the time of obtaining informed consent.
- Patients diagnosed with isolated or familial ALS and diagnosed as definite, the probable, or the probable laboratory-supported by updated Awaji criteria.
- Patients with severity 1 or 2 on ALS severity criteria.
- Outpatients.
- Patients residing in Japan who can communicate in Japanese.
Who cannot join
- Patients who do not meet any of the exclusion criteria (1) to (15) at the time of Informed consent
- Patients with a tracheostomy
- Patients with a history of non-invasive respiratory support
- Patients with a percent FVC of 60 or less
- Patients with chronic obstructive pulmonary disease (COPD)
- Patients newly treated with edaravone or riluzole (oral) within 4 weeks prior to Informed consent
Show all 16Show fewer
- Patients receiving HAL medical leg type treatment
- Patients receiving intravenous edaravone
- Patients with cognitive impairment
- Pregnant women or patients who may be pregnant
- Patients with serious respiratory, cardiovascular, hepatic, or renal disease
- Patients with malignant tumors
- Patients with uncontrolled infection
- Patients who have participated in other clinical trials within 12 weeks prior to obtaining consent
- Patients with a history of drug allergy or severe allergic disease (e.g., anaphylactic shock) or concomitant history
- Patients who are deemed inappropriate to participate in the study by the principal investigator or research coordinator.
Trial locations and contacts
1 site in 1 country.
About this study
This study evaluates the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media (SHED-CM) in patients with Amyotrophic Lateral Sclerosis (ALS), using the Japanese version of the revised ALS Functional Rating Scale (ALSFRS-R) as an indicator.
Read the detailed description
The main objective of this study is to evaluate the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media in patients with Amyotrophic Lateral Sclerosis (ALS), Particular focus will be placed on improving neurological function, slowing the rate of symptom progression, and improving the patient's quality of life.
What are the advantages of this therapy over standard therapy? and for which patients it is most effective ?
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- The study drug is SHED-CM manufactured by U-Factor
- Keywords
- ALS - Amyotrophic Lateral Sclerosis, Stem Cells, Conditioned Medium, Therapy, Safety, Regenerative Medicine
- 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.
The study drug is SHED-CM manufactured by U-FactorBiological
This study will involve informed consent, a 12-week observation period, a 12-week study drug administration period, and a 4-week follow-up period.
| Study group | Role | What they receive |
|---|---|---|
| Receiving the study drug | Experimental | The study drug will be administered intravenously at 120 ml once a week for 12 week. Receives: Biological: The study drug is SHED-CM manufactured by U-Factor |
What is being measured
The primary outcome is the main question the study is designed to answer.
All Adverse Events
This measure will check the total number of adverse events (AEs) reported during the study, categorized by CTCAE Ver 5.0 .
Measured: Immediately after each administration and up to 4 weeks post-treatment
Number of Clinically Significant Changes in Laboratory Test Results
This measure will track the number of events where clinically significant abnormalities are detected in laboratory test parameters, including blood count, biochemistry, and coagulation function tests. Changes from baseline to follow-up are evaluated based on predefined clinical thresholds.
Measured: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
Number of Clinically Significant Changes in Vital Signs
This measure will evaluate the number of events where clinically significant changes in vital signs occur. Parameters include systolic blood pressure, diastolic blood pressure, pulse rate, body temperature, and SpO2. Each event will be assessed at two time points: immediately after treatment and after follow-up session, comparing values to baseline.
Measured: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
Safety assessment during the study period: Adverse events - Self- and other findings
Medical examination, subjective findings, Other findings
Measured: Immediately after each administration and up to 4 weeks post-treatment
Secondary outcomes (3)
Efficacy assessment: ALSFRS-R
The revised ALS Functional Rating Scale in ALS Patients. This scale is scored from 0 to 48, the higher the score the better.
Measured: Change from baseline ALSFRS-R at follow-up session
Efficacy assessment: %FVC
% forced vital capacity in ALS Patients
Measured: Change from baseline %FVC at follow-up session
Efficacy assessment: grip strength
grip strength in ALS Patients
Measured: Change from baseline grip strength at follow-up session
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2025
- Study start5 Apr 2024
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A group of ALS patients receiving the study drug
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hitonowa Medical
- Sponsor type
- University, hospital or other
- Collaborators
- U-Factor Co.,Ltd.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06889857
- Sponsor's protocol ID
- SHED-CM-ALS-202401
- Acronym
- SCA202401
- Status last verified
- Sep 2025
- First submitted
- 18 Jul 2024
- Last updated
- 15 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Oki R, Izumi Y, Fujita K, Miyamoto R, Nodera H, Sato Y, Sakaguchi S, Nokihara H, Kanai K, Tsunemi T, Hattori N, Hatanaka Y, Sonoo M, Atsuta N, Sobue G, Shimizu T, Shibuya K, Ikeda K, Kano O, Nishinaka K, Kojima Y, Oda M, Komai K, Kikuchi H, Kohara N, Urushitani M, Nakayama Y, Ito H, Nagai M, Nishiyama K, Kuzume D, Shimohama S, Shimohata T, Abe K, Ishihara T, Onodera O, Isose S, Araki N, Morita M, Noda K, Toda T, Maruyama H, Furuya H, Teramukai S, Kagimura T, Noma K, Yanagawa H, Kuwabara S, Kaji R; Japan Early-Stage Trial of Ultrahigh-Dose Methylcobalamin for ALS (JETALS) Collaborators. Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Neurol. 2022 Jun 1;79(6):575-583. doi: 10.1001/jamaneurol.2022.0901.
PubMed 35532908
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Intravenous Administration of SHED-CM for ALS
- ClinicalTrials.gov ID
- NCT06889857
- Acronym / sponsor ID
- SCA202401 / SHED-CM-ALS-202401
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS - Amyotrophic Lateral Sclerosis, Stem Cells, Conditioned Medium, Therapy, Safety, Regenerative Medicine
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Hitonowa MedicalUniversity, hospital or other
- Collaborators
- U-Factor Co.,Ltd.
- Study start
- 5 Apr 2024 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 21 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study evaluates the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media (SHED-CM) in patients with Amyotrophic Lateral Sclerosis (ALS), using the Japanese version of the revised ALS Functional Rating Scale (ALSFRS-R) as an indicator.
Detailed description
The main objective of this study is to evaluate the safety and efficacy of Stem Cell from Human Exfoliated Deciduous teeth Conditioned Media in patients with Amyotrophic Lateral Sclerosis (ALS), Particular focus will be placed on improving neurological function, slowing the rate of symptom progression, and improving the patient's quality of life.
What are the advantages of this therapy over standard therapy? and for which patients it is most effective ?
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A group of ALS patients receiving the study drug
| Arm | Type | Receives |
|---|---|---|
| Receiving the study drug | Experimental | Biological: The study drug is SHED-CM manufactured by U-FactorThe study drug will be administered intravenously at 120 ml once a week for 12 week. |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Patients who meet the following inclusion criteria (1) to (6) at the time of Informed consent
- Patients who have provided written informed consent to participate in the study.
- Patients who are at least 20 years of age at the time of obtaining informed consent.
- Patients diagnosed with isolated or familial ALS and diagnosed as definite, the probable, or the probable laboratory-supported by updated Awaji criteria.
- Patients with severity 1 or 2 on ALS severity criteria.
Key exclusion criteria (16 listed)
- Patients who do not meet any of the exclusion criteria (1) to (15) at the time of Informed consent
- Patients with a tracheostomy
- Patients with a history of non-invasive respiratory support
- Patients with a percent FVC of 60 or less
- Patients with chronic obstructive pulmonary disease (COPD)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| The study drug is SHED-CM manufactured by U-FactorThis study will involve informed consent, a 12-week observation period, a 12-week study drug administration period, and a 4-week follow-up period. | Biological | Receiving the study drug |
Primary outcome
All Adverse Events
Time frame: Immediately after each administration and up to 4 weeks post-treatment
This measure will check the total number of adverse events (AEs) reported during the study, categorized by CTCAE Ver 5.0 .
Number of Clinically Significant Changes in Laboratory Test Results
Time frame: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
This measure will track the number of events where clinically significant abnormalities are detected in laboratory test parameters, including blood count, biochemistry, and coagulation function tests. Changes from baseline to follow-up are evaluated based on predefined clinical thresholds.
Number of Clinically Significant Changes in Vital Signs
Time frame: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
This measure will evaluate the number of events where clinically significant changes in vital signs occur. Parameters include systolic blood pressure, diastolic blood pressure, pulse rate, body temperature, and SpO2. Each event will be assessed at two time points: immediately after treatment and after follow-up session, comparing values to baseline.
Safety assessment during the study period: Adverse events - Self- and other findings
Time frame: Immediately after each administration and up to 4 weeks post-treatment
Medical examination, subjective findings, Other findings
3 secondary outcomes
Efficacy assessment: ALSFRS-R
Time frame: Change from baseline ALSFRS-R at follow-up session
Efficacy assessment: %FVC
Time frame: Change from baseline %FVC at follow-up session
Efficacy assessment: grip strength
Time frame: Change from baseline grip strength at follow-up session
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2025.
- Results publication: PubMed 35532908Oki R, Izumi Y, Fujita K, Miyamoto R, Nodera H, Sato Y, Sakaguchi S, Nokihara H, Kanai K, Tsunemi T, Hattori N, Hatanaka Y, Sonoo M, Atsuta N, Sobue G, Shimizu T, Shibuya K, Ikeda K, Kano O, Nishinaka K, Kojima Y, Oda M, Komai K, Kikuchi H, Kohara N, Urushitani M, Nakayama Y, Ito H, Nagai M, Nishiyama K, Kuzume D, Shimohama S, Shimohata T, Abe K, Ishihara T, Onodera O, Isose S, Araki N, Morita M, Noda K, Toda T, Maruyama H, Furuya H, Teramukai S, Kagimura T, Noma K, Yanagawa H, Kuwabara S, Kaji R; Japan Early-Stage Trial of Ultrahigh-Dose Methylcobalamin for ALS (JETALS) Collaborators. Efficacy and Safety of Ultrahigh-Dose Methylcobalamin in Early-Stage Amyotrophic Lateral Sclerosis: A Randomized Clinical Trial. JAMA Neurol. 2022 Jun 1;79(6):575-583. doi: 10.1001/jamaneurol.2022.0901.
- We referenced this paper because of the Phase III trial in ALS.Link provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing The study drug is SHED-CM manufactured by U-Factor in people with Amyotrophic Lateral Sclerosis, run by Hitonowa Medical at 1 site in Japan.
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 7 inclusion and 16 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: under way, not looking for participants, as of the sponsor's last update on 15 Sep 2025. 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 Japan. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
All Adverse Events (time frame: Immediately after each administration and up to 4 weeks post-treatment). 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 NCT06889857, 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 15 Sep 2025.