Safety and Efficacy of Intravenous Administration of SHED-CM for ALS (SCA202401)

Hitonowa Medical

Status and phase: Enrolling; Early Phase 1

Conditions: Amyotrophic Lateral Sclerosis

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

Study type: Interventional

Funder types: Other

Identifiers

NCT06889857

SHED-CM-ALS-202401

Details and patient eligibility

About

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.

Full 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 ?

Enrollment: 10 estimated patients

Sex: All

Ages: 20+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Receiving the study drug

Experimental group

Description:

The study drug will be administered intravenously at 120 ml once a week for 12 week.

Treatment:

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