Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03280056 | Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients | PHASE3 | COMPLETED | 196 | — | — | Aug 28, 2017 | Sep 29, 2020 | Feb 29, 2024 | 6 | United States |
The ALSFRS-R is a quickly administered (10 minutes) ordinal, validated rating scale (ratings 0-4) used to determine participants' assessment of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, as measured by quantitative neuromuscular strength testing, and with quality of life measures, and predicted survival. The total score of ALSFRS-R ranges from 0-48, with higher score being better.
| Arm | Type | Description |
|---|---|---|
| NurOwn® (MSC-NTF cells) | ACTIVE_COMPARATOR | Three Intrathecal administrations of NurOwn® (MSC-NTF cells) at bi-monthly intervals |
| Placebo | PLACEBO_COMPARATOR | Three Intrathecal administrations of Placebo at bi-monthly intervals |
| Name | Type | Description |
|---|---|---|
| NurOwn® (MSC-NTF cells) | BIOLOGICAL | NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10\^6 cells every 8 weeks NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors |
| Placebo | OTHER | One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks Placebo: liquid solution in syringe for injection |
| Bone Marrow aspiration | OTHER | Bone Marrow aspiration |
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. * Uprigh...