Clinical trial NCT06910384: A Study of STRO4 in Patients Amyotrophic Lateral Sclerosis (ALS)
Official title: An Open Label Study of STR04 in Participants With Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Lead sponsor: New England Cell Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2025-04-04
Enrollment: 9
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open Label
Primary purpose: TREATMENT

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

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.

Eligibility 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.

Primary outcomes:
measure: Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Score
timeFrame: 25 weeks
Secondary outcomes:
measure: Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Score
timeFrame: 49 weeks
measure: Change in Percent-predicted forced vital capacity (%FVC)
timeFrame: From Baseline to 25 weeks and 49 weeks
measure: Change in Manual Muscle Test (MMT)
timeFrame: From Baseline to 25 weeks and 49 weeks
measure: Change in Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40)
timeFrame: From Baseline to 25 weeks and 49 weeks
measure: Change in plasma neurofilament light (NfL)
timeFrame: From Baseline to 25 weeks and 49 weeks
Other outcomes:
Locations:
Study officials:
affiliation: New England Cell Therapeutics, Inc.
name: Alan Jacobs, MD, PhD
role: STUDY_DIRECTOR
References: