Phase 3
N=189
Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients
Amyotrophic Lateral Sclerosis (ALS)
Bottom Line
View on ClinicalTrials.gov: NCT03280056 ↗Enrolled (actual)
189
Serious AEs
21.2%
Results posted
Feb 2024
Primary outcome: Primary: The Proportion of NurOwn® Treated Participants With a ≥1.25 Points/Month Improvement in Post-treatment Slope vs. Pre-treatment Slope in ALSFRS-R Score at 28 Weeks Following the First Treatment as Compared to Placebo — 31; 26; 64; 68 Participants — p=0.453
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- NurOwn® (MSC-NTF cells) (Biological); Placebo (Other); Bone Marrow aspiration (Other)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Brainstorm-Cell Therapeutics
- Primary completion
- Sep 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Proportion of NurOwn® Treated Participants With a ≥1.25 Points/Month Improvement in Post-treatment Slope vs. Pre-treatment Slope in ALSFRS-R Score at 28 Weeks Following the First Treatment as Compared to Placebo |
31; 26; 64; 68 | 0.453 |
| SECONDARY Number of Participants Whose Disease Progression is Halted or Improved as Measured by a 100% or Greater Improvement in Post-treatment Slope vs. Pre-treatment Slope in ALSFRS-R Score of NurOwn® Treatment vs. Placebo |
13; 13; 82; 81 | 0.997 |
| SECONDARY Score of NurOwn® (MSC-NTF Cells) Treated Patients vs. Placebo Treated Patients as Measured by Change From Baseline in ALSFRS-R Score at Week 28 |
-5.52; -5.88 | 0.693 |
| SECONDARY NurOwn® (MSC-NTF Cells) Treated Patients vs. Placebo Treated Patients as Measured by the Combined Assessment of Function and Survival at 28 Weeks |
73.74; 72.21 | 0.804 |
Summary
This study will evaluate the safety and efficacy of repeated administration of NurOwn® (MSC-NTF cells) therapy, which is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patient's own bone marrow, propagated ex vivo and induced to secrete Neurotrophic factors (NTFs).
The autologous NurOwn® (MSC-NTF cells) are back-transplanted into the patient intrathecally by standard lumbar puncture where neurons and glial cells are expected to take up the neurotrophic factors secreted by the transplanted cells
Eligibility Criteria
Inclusion Criteria
- ALS diagnosed as possible, laboratory-supported probable, probable, or definite as defined by revised El Escorial criteria.
- Having onset of ALS disease symptoms, including limb weakness within 24 months at the Screening Visit.
- ALSFRS-R ≥ 25 at the screening Visit.
- Upright slow vital capacity (SVC) measure ≥ 65% of predicted for gender, height, and age at the screening Visit.
- Rapid progressors
- Participants taking a stable dose of Riluzole are permitted in the study
- Citizen or permanent resident of the United States or Canadian citizen able to travel to a US site for all follow-up study visits
Exclusion Criteria
- Prior stem cell therapy of any kind
- History of autoimmune or other serious disease (including malignancy and immune deficiency) that may confound study results
- Current use of immunosuppressant medication or anticoagulants (per Investigator discretion)
- Exposure to any other experimental agent or participation in an ALS clinical trial within 30 days prior to Screening Visit
- Use of RADICAVA (edaravone injection) within 30 days of screening or intent to use edaravone at any time during the course of the study including the follow up period
- Use of non-invasive ventilation (BIPAP), diaphragm pacing system or invasive ventilation (tracheostomy)
- Feeding tube
- Pregnant women or women currently breastfeeding
Data sourced from ClinicalTrials.gov (NCT03280056). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.