Amyotrophic Lateral Sclerosis
Conditions
Keywords
ALS
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.
Interventions
BIOLOGICALSTR04
autologous bone-marrow-derived mesenchymal stem cells
Sponsors
New England Cell Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open Label
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in Amyotrophic Lateral Sclerosis Functional Rating Scale Score | 25 weeks |
Secondary
| 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 |
Countries
United States
Outcome results
None listed