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

NCT06608719 | Completed

• 1 other identifier: SHED-CM-ALS-H-2023
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

In this study, we will retrospectively evaluate the safety and efficacy of administering SHED-CM for the treatment of ALS.

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 .

  after the 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 after the 4th treatment session will be evaluated based on predefined clinical thresholds.

  Baseline (pre-treatment) and after 4th treatment

• 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 the 4th treatment session, comparing values to baseline.

  Baseline (pre-treatment), post-treatment, and after 4th treatment

• Safety assessment during the study period: Adverse events - Self- and other findings

  Medical examination, subjective findings, Other findings

  after the treatment

Secondary Outcomes (3)

• Efficacy assessment: Changes in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised version

  Change from baseline at 16 weeks

• Efficacy assessment: Changes in %FVC

  Change from baseline at 16 weeks

• Efficacy assessment: Changes in grip strength

  Change from baseline at 16 weeks

Study Arms (1)

Receiving the study drug

Patients diagnosed with ALS who visited our hospital and were administered SHED-CM between January 1, 2022, and November 30, 2023.

Biological: The study drug is SHED-CM manufactured by U-Factor

Interventions

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

Patients diagnosed with ALS were administered SHED-CM

Receiving the study drug

Eligibility Criteria

• Age: 38 Years - 81 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Amyotrophic Lateral Sclerosis

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Hitonowa Medical: lead

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: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: September 23, 2024
• Study Start: December 25, 2023
• Primary Completion: July 1, 2024
• Study Completion: July 1, 2024
• Last Updated: September 23, 2024

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, CSR
• Time Frame: The original data presented in the study are openly available without restrictions.
• Access Criteria: The original data presented in the study are openly available in FigShare at 10.6084/m9.figshare.26201615.

Locations

JP (1)