Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 18 October 2021
• Main ID: NCT03280056
• Date of registration: 29/08/2017
• Prospective Registration: No
• Primary sponsor: Brainstorm-Cell Therapeutics
• Public title: Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients
• Scientific title: A Phase 3, Randomized Double-Blind, Placebo-Controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Participants With ALS
• Date of first enrolment: August 28, 2017
• Target sample size: 263
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03280056
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Anthony J. Windebank, MD
• Affiliation: Mayo Clinic

• Name: Namita A. Goyal, MD
• Affiliation: UC Irvine

• Name: Robert H. Brown, MD, PhD
• Affiliation: UMass Medical School

• Name: Robert G. Miller, MD
• Affiliation: California Pacific Medical Center (CPM) Research Institute

• Name: Robert Baloh, MD, Ph.D.
• Affiliation: Cedars-Sinai Medical Center

• Name: Merit E. Cudkowicz, MD
• Affiliation: Massachusetts General Hospital

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 60 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis (ALS)

Intervention(s)

Other: Placebo

Other: Bone Marrow aspiration

Biological: NurOwn® (MSC-NTF cells)

Primary Outcome(s)

To evaluate the efficacy and safety of NurOwn® (autologous MSC-NTF cells) as compared to placebo as measured by the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) [Time Frame: 28 weeks following the first treatment]

Secondary Outcome(s)

Biomarkers [Time Frame: Through selected post-treatment time points up to 20 weeks post transplant]

Secondary ID(s)

BCT-002-US

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

California Institute for Regenerative Medicine (CIRM)