Safety and Efficacy of Intravenous Administration of SHED-CM for ALS (SCA202401)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Biological: The study drug is SHED-CM manufactured by U-Factor

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 Type: Interventional
• Enrollment (Estimated): 10
• Phase: Early Phase 1

Contacts and Locations

Study Locations

• Japan
  • Tokyo
    • Chiyoda City, Tokyo, Japan, 102-0085
      • Hitonowa Medical

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Receiving the study drug; The study drug will be administered intravenously at 120 ml once a week for 12 week.	Biological: The study drug is SHED-CM manufactured by U-Factor; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
All Adverse Events	This measure will check the total number of adverse events (AEs) reported during the study, categorized by CTCAE Ver 5.0 .	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.	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.	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	Immediately after each administration and up to 4 weeks post-treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Change from baseline ALSFRS-R at follow-up session
Efficacy assessment: %FVC	% forced vital capacity in ALS Patients	Change from baseline %FVC at follow-up session
Efficacy assessment: grip strength	grip strength in ALS Patients	Change from baseline grip strength at follow-up session

Collaborators and Investigators

• Sponsor: Hitonowa Medical
• Collaborators: U-Factor Co.,Ltd.

Publications and helpful links

General 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.

Helpful Links

• We referenced this paper because of the Phase III trial in ALS.

Study record dates

Study Major Dates

• Study Start (Actual): April 5, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: July 18, 2024
• First Submitted That Met QC Criteria: March 17, 2025
• First Posted (Actual): March 21, 2025

Study Record Updates

• Last Update Posted (Estimated): September 15, 2025
• Last Update Submitted That Met QC Criteria: September 8, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Safety; Stem Cells; Therapy; Conditioned Medium; Regenerative Medicine; ALS - Amyotrophic Lateral Sclerosis
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: SHED-CM-ALS-202401

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No