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

NCT06889857 | Active Not Recruiting Early Phase 1

• 1 other identifier: SHED-CM-ALS-202401
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS - Amyotrophic Lateral Sclerosis; Stem Cells; Conditioned Medium; Therapy; Safety; Regenerative Medicine

Outcome Measures

Primary Outcomes (4)

• 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 Outcomes (3)

• Efficacy assessment: ALSFRS-R

  Change from baseline ALSFRS-R at follow-up session

• Efficacy assessment: %FVC

  Change from baseline %FVC at follow-up session

• Efficacy assessment: grip strength

  Change from baseline grip strength at follow-up session

Study Arms (1)

Receiving the study drug

EXPERIMENTAL

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

Interventions

The study drug is SHED-CM manufactured by U-Factor BIOLOGICAL

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.

Receiving the study drug

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Hitonowa Medical: lead
• U-Factor Co.,Ltd.: collaborator

Study Sites (1)

Hitonowa Medical

Chiyoda City, Tokyo, 102-0085, Japan

Location

Related Publications (1)

• 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.

  PMID: 35532908 BACKGROUND

Related Links

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

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: A group of ALS patients receiving the study drug

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: March 21, 2025
• Study Start: April 5, 2024
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: September 15, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

JP (1)