Amyotrophic Lateral SclerosisNCT06608719
Retrospective Study on the Safety and Therapeutic/Improvement Effects of Intravenous Administration of SHED-CM for ALS (SHED-CAH2023)
An observational study of Amyotrophic Lateral Sclerosis, run by Hitonowa Medical at 1 site in Japan.
Trial summary
In this study, we will retrospectively evaluate the safety and efficacy of administering SHED-CM for the treatment of ALS. Read more about this study
- Who is it for?
- Ages 38 to 81 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- The study drug is SHED-CM manufactured by U-Factor
- How many people
- 24 participants
- Where
- 1 siteJapan
- Study dates
- 25 Dec 2023to 1 Jul 2024
- Phase
- Observational
- 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 38 to 81 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Amyotrophic Lateral Sclerosis
Who may be able to join
- patients who visited our clinic between January 1, 2022 and November 30, 2023 and have been diagnosed with ALS
- male and female patients aged between 16 and 90 years old
- ALSFRS-R score of 12 or more at the time of initial visit
Who cannot join
- Patients who have a tracheostomy at the time of visit.
- Patients who have expressed their refusal to participate in this study.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Retrospective Study on the Safety and Therapeutic/Improvement Effects of Intravenous Administration of SHED-CM for ALS
In this study, we will retrospectively evaluate the safety and efficacy of administering SHED-CM for the treatment of ALS.
Read the detailed description
Amyotrophic Lateral Sclerosis (ALS) is a progressive and irreversible neurodegenerative disease with limited treatment options. Advances in regenerative medicine have opened new avenues for therapeutic interventions. This retrospective cohort study evaluated the safety and efficacy of stem cells from human exfoliated deciduous teeth-conditioned media (SHED-CM) in 24 patients with ALS treated at a single facility between January 1, 2022, and November 30, 2023. Safety assessments included adverse events, vital signs, and laboratory test changes before and after administration, whereas efficacy was measured using the ALS Functional Rating Scale-Revised (ALSFRS-R), grip strength, and forced vital capacity. while ALSFRS-R scores typically decline over time, the progression rate in this cohort was slower, suggesting a potential delay in disease progression. Alternatively, improvements in muscle strength and mobility were observed in some patients. Although adverse events were reported in only 3% of cases (no serious allergic reactions), the treatment-induced changes in vital signs and laboratory results were not clinically significant. The SHED-CM treatment appears to be a safe and potentially effective therapeutic option for patients with ALS. Further research is needed to optimize the SHED-CM treatment; however, this study lays the groundwork for future exploration of regenerative therapies for ALS.
- 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
Patients diagnosed with ALS were administered SHED-CM
| Study group | Role | What they receive |
|---|---|---|
| Receiving the study drug | Patients diagnosed with ALS who visited our hospital and were administered SHED-CM between January 1, 2022, and November 30, 2023. 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: after the 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 after the 4th treatment session will be evaluated based on predefined clinical thresholds.
Measured: Baseline (pre-treatment) and after 4th treatment
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 the 4th treatment session, comparing values to baseline.
Measured: Baseline (pre-treatment), post-treatment, and after 4th treatment
Safety assessment during the study period: Adverse events - Self- and other findings
Medical examination, subjective findings, Other findings
Measured: after the treatment
Secondary outcomes (3)
Efficacy assessment: Changes in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised version
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 at 16 weeks
Efficacy assessment: Changes in %FVC
% forced vital capacity in percent
Measured: Change from baseline at 16 weeks
Efficacy assessment: Changes in grip strength
grip strength in kilograms
Measured: Change from baseline at 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2024
- Study start25 Dec 2023
- Primary completion1 Jul 2024
- Study completion1 Jul 2024
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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hitonowa Medical
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06608719
- Sponsor's protocol ID
- SHED-CM-ALS-H-2023
- Status last verified
- Sep 2024
- First submitted
- 18 Jul 2024
- Last updated
- 23 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: This was a retrospective cohort study including patients diagnosed with ALS who visited our hospital between January 1, 2022, and November 30, 2023. We enrolled pa-tients with ALS who received SHED-CM within this period, had a score of 12 or higher on the ALS Functional Rating Scale-Revised (ALSFRS-R), and did not undergo trache-ostomy. All administered data were evaluated for safety. Several patients experienced difficulties presenting to the hospital due to the progression of their ALS; therefore, we evaluated the efficacy of the drug from the first to the 12th dose, which were adminis-tered consecutively, and for which all patients presented to the hospital.
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 23 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Retrospective Study on the Safety and Therapeutic/Improvement Effects of Intravenous Administration of SHED-CM for ALS
- ClinicalTrials.gov ID
- NCT06608719
- Acronym / sponsor ID
- SHED-CM-ALS-H-2023
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS - Amyotrophic Lateral Sclerosis, Stem Cells, Conditioned Medium, Therapy, Safety, Regenerative Medicine
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Hitonowa MedicalUniversity, hospital or other
- Study start
- 25 Dec 2023 (actual)
- Primary completion
- 1 Jul 2024 (actual)
- Study completion
- 1 Jul 2024 (actual)
- First posted
- 23 Sep 2024 (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.
In this study, we will retrospectively evaluate the safety and efficacy of administering SHED-CM for the treatment of ALS.
Detailed description
Amyotrophic Lateral Sclerosis (ALS) is a progressive and irreversible neurodegenerative disease with limited treatment options. Advances in regenerative medicine have opened new avenues for therapeutic interventions. This retrospective cohort study evaluated the safety and efficacy of stem cells from human exfoliated deciduous teeth-conditioned media (SHED-CM) in 24 patients with ALS treated at a single facility between January 1, 2022, and November 30, 2023. Safety assessments included adverse events, vital signs, and laboratory test changes before and after administration, whereas efficacy was measured using the ALS Functional Rating Scale-Revised (ALSFRS-R), grip strength, and forced vital capacity. while ALSFRS-R scores typically decline over time, the progression rate in this cohort was slower, suggesting a potential delay in disease progression. Alternatively, improvements in muscle strength and mobility were observed in some patients. Although adverse events were reported in only 3% of cases (no serious allergic reactions), the treatment-induced changes in vital signs and laboratory results were not clinically significant. The SHED-CM treatment appears to be a safe and potentially effective therapeutic option for patients with ALS. Further research is needed to optimize the SHED-CM treatment; however, this study lays the groundwork for future exploration of regenerative therapies for ALS.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Receiving the study drug | – | Biological: The study drug is SHED-CM manufactured by U-FactorPatients diagnosed with ALS who visited our hospital and were administered SHED-CM between January 1, 2022, and November 30, 2023. |
Who may qualify
- Ages
- 38 to 81 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Amyotrophic Lateral Sclerosis
Key inclusion criteria (3 listed)
- patients who visited our clinic between January 1, 2022 and November 30, 2023 and have been diagnosed with ALS
- male and female patients aged between 16 and 90 years old
- ALSFRS-R score of 12 or more at the time of initial visit
Key exclusion criteria (2 listed)
- Patients who have a tracheostomy at the time of visit.
- Patients who have expressed their refusal to participate in this study.
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-FactorPatients diagnosed with ALS were administered SHED-CM | Biological | Receiving the study drug |
Primary outcome
All Adverse Events
Time frame: after the 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 (pre-treatment) and after 4th treatment
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 after the 4th treatment session will be evaluated based on predefined clinical thresholds.
Number of Clinically Significant Changes in Vital Signs
Time frame: Baseline (pre-treatment), post-treatment, and after 4th treatment
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 the 4th treatment session, comparing values to baseline.
Safety assessment during the study period: Adverse events - Self- and other findings
Time frame: after the treatment
Medical examination, subjective findings, Other findings
3 secondary outcomes
Efficacy assessment: Changes in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised version
Time frame: Change from baseline at 16 weeks
Efficacy assessment: Changes in %FVC
Time frame: Change from baseline at 16 weeks
Efficacy assessment: Changes in grip strength
Time frame: Change from baseline at 16 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2024.
- Background: 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: Yes. This was a retrospective cohort study including patients diagnosed with ALS who visited our hospital between January 1, 2022, and November 30, 2023. We enrolled pa-tients with ALS who received SHED-CM within this period, had a score of 12 or higher on the ALS Functional Rating Scale-Revised (ALSFRS-R), and did not undergo trache-ostomy. All administered data were evaluated for safety. Several patients experienced difficulties presenting to the hospital due to the progression of their ALS; therefore, we evaluated the efficacy of the drug from the first to the 12th dose, which were adminis-tered consecutively, and for which all patients presented to the hospital.
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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?
An observational study of 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 3 inclusion and 2 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 23 Sep 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 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.
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?
All Adverse Events (time frame: after the 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 NCT06608719, 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 23 Sep 2024.