Safety and Efficacy of Intravenous Administration of SHED-CM for ALS

Early Phase 1

Recruiting

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Biological: The study drug is SHED-CM manufactured by U-Factor

• Registration Number: NCT06889857
• Lead Sponsor: Hitonowa Medical

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 ?

Study Timeline

• Study Start: 2024-04-05
• Study First Submitted: 2024-07-18
• Status Verified: 2025-03
• Study First Submitted QC: 2025-03-17
• Last Update Submitted: 2025-03-17
• Study First Posted: 2025-03-25
• Last Update Posted: 2025-03-25
• Primary Completion: 2025-03-31
• Study Completion: 2025-03-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

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 exclusion criteria (1) to (15) at the time of Informed consent

1. Patients with a tracheostomy
2. Patients with a history of non-invasive respiratory support
3. Patients with a percent FVC of 60 or less
4. Patients with chronic obstructive pulmonary disease (COPD)
5. Patients newly treated with edaravone or riluzole (oral) within 4 weeks prior to Informed consent
6. Patients receiving HAL medical leg type treatment
7. Patients receiving intravenous edaravone
8. Patients with cognitive impairment
9. Pregnant women or patients who may be pregnant
10. Patients with serious respiratory, cardiovascular, hepatic, or renal disease
11. Patients with malignant tumors
12. Patients with uncontrolled infection
13. Patients who have participated in other clinical trials within 12 weeks prior to obtaining consent
14. Patients with a history of drug allergy or severe allergic disease (e.g., anaphylactic shock) or concomitant history
15. Patients who are deemed inappropriate to participate in the study by the principal investigator or research coordinator.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Receiving the study drug	The study drug is SHED-CM manufactured by U-Factor	The study drug will be administered intravenously at 120 ml once a week for 12 week.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.
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

Trial Locations

Locations (1)

Hitonowa Medical; 🇯🇵 Chiyoda, Tokyo, Japan