Clinical trial NCT06608719: Retrospective Study on the Safety and Therapeutic/Improvement Effects of Intravenous Administration of SHED-CM for ALS (SHED-CAH2023)
Official title: Retrospective Study on the Safety and Therapeutic/Improvement Effects of Intravenous Administration of SHED-CM for ALS
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS - Amyotrophic Lateral Sclerosis
Stem Cells
Conditioned Medium
Therapy
Safety
Regenerative Medicine
Lead sponsor: Hitonowa Medical
Sponsor class: OTHER
First posted: 2024-09-23
Enrollment: 24
Sex: ALL
Minimum age (years): 38
Maximum age (years): 81
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Amyotrophic Lateral Sclerosis

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.

Eligibility criteria: 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.

Primary outcomes:
measure: All Adverse Events
timeFrame: after the treatment
measure: Number of Clinically Significant Changes in Laboratory Test Results
timeFrame: Baseline (pre-treatment) and after 4th treatment
measure: Number of Clinically Significant Changes in Vital Signs
timeFrame: Baseline (pre-treatment), post-treatment, and after 4th treatment
measure: Safety assessment during the study period: Adverse events - Self- and other findings
timeFrame: after the treatment
Secondary outcomes:
measure: Efficacy assessment: Changes in Amyotrophic Lateral Sclerosis Functional Rating Scale Revised version
timeFrame: Change from baseline at 16 weeks
measure: Efficacy assessment: Changes in %FVC
timeFrame: Change from baseline at 16 weeks
measure: Efficacy assessment: Changes in grip strength
timeFrame: Change from baseline at 16 weeks
Other outcomes:
Locations:
city: Chiyoda
country: Japan
facility: Hitonowa Medical
state: Tokyo
Study officials:
References:
citation: 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.