Clinical trial NCT06889857: Safety and Efficacy of Intravenous Administration of SHED-CM for ALS
Official title: Safety and Efficacy of Intravenous Administration of SHED-CM for ALS
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: EARLY_PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS - Amyotrophic Lateral Sclerosis
Stem Cells
Conditioned Medium
Therapy
Safety
Regenerative Medicine
Lead sponsor: Hitonowa Medical
Sponsor class: OTHER
Collaborators: name: U-Factor Co.,Ltd.
First posted: 2025-03-21
Enrollment: 10
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: A group of ALS patients receiving the study drug
Primary purpose: TREATMENT

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.

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 ?

Eligibility criteria: 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.

Primary outcomes:
measure: All Adverse Events
timeFrame: Immediately after each administration and up to 4 weeks post-treatment
measure: Number of Clinically Significant Changes in Laboratory Test Results
timeFrame: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
measure: Number of Clinically Significant Changes in Vital Signs
timeFrame: Baseline, Week 4, Week 8, Week 12, and at follow-up (Week 16).
measure: Safety assessment during the study period: Adverse events - Self- and other findings
timeFrame: Immediately after each administration and up to 4 weeks post-treatment
Secondary outcomes:
measure: Efficacy assessment: ALSFRS-R
timeFrame: Change from baseline ALSFRS-R at follow-up session
measure: Efficacy assessment: %FVC
timeFrame: Change from baseline %FVC at follow-up session
measure: Efficacy assessment: grip strength
timeFrame: Change from baseline grip strength at follow-up session
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.