Retrospective Study on the Safety and Therapeutic/Improvement Effects of Intravenous Administration of SHED-CM for ALS (SHED-CAH2023)

Completed

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

• Registration Number: NCT06608719
• Lead Sponsor: Hitonowa Medical

Brief Summary

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 Timeline

• Study Start: 2023-12-25
• Primary Completion: 2024-07-01
• Study Completion: 2024-07-01
• Study First Submitted: 2024-07-18
• Status Verified: 2024-09
• Study First Submitted QC: 2024-09-19
• Last Update Submitted: 2024-09-19
• Study First Posted: 2024-09-23
• Last Update Posted: 2024-09-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 24

Inclusion Criteria

• 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

Exclusion Criteria

• Patients who have a tracheostomy at the time of visit.
• Patients who have expressed their refusal to participate in this study.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Receiving the study drug	The study drug is SHED-CM manufactured by U-Factor	Patients diagnosed with ALS who visited our hospital and were administered SHED-CM between January 1, 2022, and November 30, 2023.

Primary Outcome Measures

Name	Time	Method
All Adverse Events	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	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	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	after the treatment	Medical examination, subjective findings, Other findings

Secondary Outcome Measures

Name	Time	Method
Efficacy assessment: Changes in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised version	Change from baseline at 16 weeks	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: Changes in %FVC	Change from baseline at 16 weeks	% forced vital capacity in percent
Efficacy assessment: Changes in grip strength	Change from baseline at 16 weeks	grip strength in kilograms

Trial Locations

Locations (1)

Hitonowa Medical; 🇯🇵 Chiyoda, Tokyo, Japan