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

New England Cell Therapeutics, Inc.

Status and phase

Terminated

Phase 2

Conditions

Amyotrophic Lateral Sclerosis

Treatments

Biological: STR04

Study type

Interventional

Funder types

Industry

Identifiers

NCT06910384

NECT 24-03

Details and patient eligibility

About

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

Full 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.

Enrollment

1 patient

Sex

All

Ages

18 to 75 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

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.