A Study of STRO4 in Patients Amyotrophic Lateral Sclerosis (ALS)

NCT06910384 | Terminated Phase 2 DMC FDA Drug

• 1 other identifier: NECT 24-03
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

This study intends to evaluate the safety and efficacy of STR04 administered intravenously in participants with Amyotrophic Lateral Sclerosis.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS

Outcome Measures

Primary Outcomes (1)

• Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Score

  25 weeks

Secondary Outcomes (5)

• Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Score

  49 weeks

• Change in Percent-predicted forced vital capacity (%FVC)

  From Baseline to 25 weeks and 49 weeks

• Change in Manual Muscle Test (MMT)

  From Baseline to 25 weeks and 49 weeks

• Change in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40)

  From Baseline to 25 weeks and 49 weeks

• Change in plasma neurofilament light (NfL)

  From Baseline to 25 weeks and 49 weeks

Study Arms (1)

Single Arm, Open Label

OTHER

Biological: STR04

Interventions

STR04 BIOLOGICAL

autologous bone-marrow-derived mesenchymal stem cells

Single Arm, Open Label

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• "Definite" or "Probable" ALS according to the Revised El Escorial criteria (Airlie House)
• ALS severity grade 1 or 2 according to the Japan ALS severity classification.
• ALSFRS-R scores of 2 or more for all items, with scores of 4 for all breathing- related items (i.e., dyspnea, orthopnea, respiratory insufficiency).
• Within 2 years of ALS onset.
• %FVC of 80% or more.
• Aged 18 to ≤75 years at the time of informed consent.

You may not qualify if:

• Current Viral Infection (e.g. HBV, HCV, HIV, HTLV-1, HPVB19, syphilis, COVID- 19).
• Current low blood cell counts
• History of cancer, congenital malformations, or chromosomal abnormalities
• History of allergy to penicillin or streptomycin, or other serious allergies.
• Current poor medical condition, due to endocrine disease, metabolic disease, immune disease, blood disease, psychiatric disorders, neurological diseases, cardiovascular diseases, respiratory diseases, gastrointestinal diseases, musculoskeletal or connective tissue diseases, renal or urogenital diseases.
• History of intracranial lesions, or stenosis, dissection or severe atherosclerotic disease of a blood vessel in the head or neck.
• Current uncontrolled hypertension.
• Prior treatment with a cell or gene therapy.
• Currently participating in any other clinical trial.
• Women who are pregnant, breastfeeding, or plan to become pregnant during study participation
• Men with plan for their partner to become pregnant during study participation.

Sponsors & Collaborators

• New England Cell Therapeutics, Inc.: lead

Study Sites (1)

National Neuromuscular Research Institute

Austin, Texas, 78759, United States

Location

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 Officials

• Alan Jacobs, MD, PhD

  New England Cell Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open Label

Study Record Dates

• First Submitted: March 7, 2025
• First Posted: April 4, 2025
• Study Start: August 12, 2025
• Primary Completion: December 16, 2025
• Study Completion: December 16, 2025
• Last Updated: December 18, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)