Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS - Amyotrophic Lateral Sclerosis, Stem Cells, Conditioned Medium, Therapy, Safety, Regenerative Medicine
Brief 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.
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 ?
Interventions
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.
Sponsors
U-Factor Co.,Ltd.
Hitonowa Medical
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
A group of ALS patients receiving the study drug
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Patients who meet the following inclusion criteria (1) to (6) at the time of Informed consent 1. Patients who have provided written informed consent to participate in the study. 2. Patients who are at least 20 years of age at the time of obtaining informed consent. 3. Patients diagnosed with isolated or familial ALS and diagnosed as definite, the probable, or the probable laboratory-supported by updated Awaji criteria. 4. Patients with severity 1 or 2 on ALS severity criteria. 5. Outpatients. 6. Patients residing in Japan who can communicate in Japanese.
Exclusion criteria
Patients who do not meet any of the
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| All Adverse Events | 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 | 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 | 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 | Immediately after each administration and up to 4 weeks post-treatment | Medical examination, subjective findings, Other findings |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy assessment: %FVC | Change from baseline %FVC at follow-up session | % forced vital capacity in ALS Patients |
| Efficacy assessment: grip strength | Change from baseline grip strength at follow-up session | grip strength in ALS Patients |
| Efficacy assessment: ALSFRS-R | Change from baseline ALSFRS-R at follow-up session | The revised ALS Functional Rating Scale in ALS Patients. This scale is scored from 0 to 48, the higher the score the better. |
Countries
Japan
Outcome results
None listed