A Double Blind, Randomized, Placebo Controlled Phase 2b Study to Evaluate the Safety and Clinical Efficacy of Treatment With the Autologous Cell Therapy Product, NG01, in Patients With Secondary Progressive Multiple Sclerosis
NeuroGenesis Ltd.
Summary
The goal of this clinical trial is to assess the safety and efficacy of repeated intrathecal (IT) injection of NG01, autologous bone marrow derived human stromal cells, in treating Secondary Progressive Multiple Sclerosis (SPMS), compared to placebo. The study will assess the proportion of participants demonstrating improvement in walking ability, defined as a reduction in the average time to complete the Timed 25-Foot Walk (T25FW) at 6, 9, and 12 months compared to baseline. This will be analyzed by the mean change in walking speed across these time points. The study will also evaluate the incidence and nature of treatment-emergent adverse events (AEs). Participants will receive intrathecal administrations of NG01, by lumbar puncture, and will be followed up for 6 months after their fourth administration.
Description
This is a multi-center, international Phase 2b, dose finding, randomized, double-blinded, placebo-controlled, three arm study, designed to assess the safety and efficacy of 4 IT administrations of NG01, compared to placebo, with a 4-month run-in period followed by a period of 9 months treatment with 6 months of follow-up, in patients with SPMS. Participants will continue to receive their customary MS treatment regimen at a stable dose. The study will enroll 45 participants with secondary progressive multiple sclerosis (SPMS), randomized in a 1:1:1 ratio, to receive four intrathecal administra…
Eligibility
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Participants aged 18 to 65 years old. 2. Diagnosis of SPMS. 3. Documented EDSS worsening over the 2 years prior to study entry of ≥1 point for participants with EDSS \<6.0 at screening, and ≥0.5 point for participants with EDSS ≥6.0 at screening, or a documented worsening of at least 20% in the T25FW. If documented T25fW or EDSS is not available, a written summary of the clinical evidence of disability worsening over the previous 2 years and retrospective assessment of EDSS score from data up to 2 years prior to screening, must be submitted for central review by adjudic…
Interventions
- BiologicalNG01 - Autologous bone marrow derived human stromal cells
NG01 is a cellular therapy product of autologous stromal cells derived from the bone marrow of multiple sclerosis (MS) patients. NG01 is provided as a cell suspension for intrathecal injection.
- OtherSodium Chloride 0.9%
The placebo for use in the NG01 clinical trial is 0.9% Sodium Chloride Solution for Injection
Locations (2)
- University of Miami Hospital & Clinics - Sylvester Comprehensive Cancer Center - NeurologyMiami, Florida
- Hadassah University HospitalJerusalem