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

An Open Label Study of STR04 in Participants With Amyotrophic Lateral Sclerosis

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

Study Overview

• Status: Terminated
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Biological: STR04

Detailed Description

This study is a 69 week open label study of autologous bone-marrow-derived mesenchymal stem cells (STR04) in participants with ALS.

Participants will undergo a screening visit, followed by collection of peripheral blood and bone marrow for the manufacture of STR04. Once manufacturing is successfully completed, participants will have a Baseline visit followed by 4 doses of STR04, one dose every 12 weeks. Participants will have a final assessments 12 weeks after the 4th dose.

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Texas
    • Austin, Texas, United States, 78759
      • National Neuromuscular Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion:

• "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.

Exclusion:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Single Arm, Open Label	Biological: STR04; autologous bone-marrow-derived mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Score	25 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: New England Cell Therapeutics, Inc.
• Investigators: Study Director: Alan Jacobs, MD, PhD, New England Cell Therapeutics, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): August 12, 2025
• Primary Completion (Actual): December 16, 2025
• Study Completion (Actual): December 16, 2025

Study Registration Dates

• First Submitted: March 7, 2025
• First Submitted That Met QC Criteria: March 27, 2025
• First Posted (Actual): April 4, 2025

Study Record Updates

• Last Update Posted (Actual): December 18, 2025
• Last Update Submitted That Met QC Criteria: December 16, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: ALS
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: NECT 24-03

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No