Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)

NCT06973629 · tracked via the Priya Life Science USA tracker

Sponsor

Brainstorm-Cell Therapeutics

Phase

Phase 3

Started

2025-06-30

Last updated

2025-05-22

Condition(s) studied

Amyotrophic Lateral Sclerosis (ALS)Amyotrophic Lateral Sclerosis

Investigational drug(s) / intervention(s)

Debamestrocel - MSC-NTF (NurOwn)Placebo

Debamestrocel - MSC-NTF (NurOwn): MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).

Placebo: Dulbecco Modified Eagle Medium (DMEM).

Study summary

The goal of this two-part clinical trial is:

1\) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period.

Researchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS.

Participants will:

Receive NurOwn or a placebo every 8 weeks for 24 weeks. After that, every participant will receive NurOwn every 8 weeks for an additional 24 weeks.

They will visit the clinic approximately every 8 weeks for checkups and tests.

Eligibility

Sex

ALL

Min age

18 Years

Max age

75 Years

Healthy volunteers

Inclusion Criteria: 1. Male and female participants 18 to 75 years old (inclusive) at Screening Visit 1. 2. ALS diagnosed as laboratory-supported probable, clinically probable, or definite as defined by the revised El Escorial criteria. 3. Having onset of ALS symptoms, including muscle weakness, within 24 months from Screening Visit 1. 4. ≥2 points on each item of the ALSFRS-R at the Screening Visit 1. 5. ≤45 points on ALSFRS-R total score at Screening Visit 1. 6. Upright slow vital capacity (SVC) measure ≥65% of predicted for gender, height, and age at Screening Visit 1. 7. Participants must adhere to highly effective methods of contraception as specified in the study protocol. Exclusion Criteria: 1. Prior stem cell therapy of any kind. 2. Active participation in any other ALS interventional study. 3. Inability to lie flat for the duration of IT cell treatment and/or bone marrow biopsy, or inability to tolerate study procedures for any other reason. 4. Any unstable clinically significant medical condition other than ALS 5. Any history of malignancy, within the previous 5 years, with the exception of localized skin cancers, cervical cancer in-situ or prostate cancer in-situ (with no evidence of metastasis, significant invasion, or reoccurrence within 3 years of baseline). 6. Primary brain cancer or cancer with CNS involvement is exclusionary. 7. Other types of motor neuron disease such as primary lateral sclerosis, progressive muscular atrophy, and progressive bulbar palsy. 8. Usage of a feeding tube at Screening Visit 1 or Screening Visit 2. 9. Pregnant women or women currently breastfeeding.

Primary outcome measure(s)

Change in the ALSFRS-R total score from baseline to Week 24 — From baseline to Week 24
To evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS based on the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) which is a 12-item clinician-administered questionnaire. Each item is scored between 0 and 4, with the total ALSFRS-R score between 0 and 48 where a higher score reflects a better outcome.
Frequency and severity of adverse events including treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), death, abnormalities in laboratory evaluations, physical examinations, vital signs and electrocardiogram (ECG) assessments — From baseline to Week 48
To evaluate the safety and tolerability of NurOwn in the treatment of participants with ALS throughout the study including the DBPC period (Part A) to Week 24 and the Open Label Extension (Part B).

Trial sites (15)

Facility	City	Region
Barrow Neurological Institute	Phoenix	Arizona
University of California San Diego Medical Center	La Jolla	California
University of Southern California	Los Angeles	California
California Pacific Medical Center	San Francisco	California
University of California, San Francisco	San Francisco	California
University of Colorado Anschutz Medical Campus School of Medicine	Aurora	Colorado
Nova Southeastern University	Davie	Florida
Mayo Clinic	Jacksonville	Florida
University of South Florida	Tampa	Florida
Northwestern Medicine	Chicago	Illinois
Sean M. Healey & AMG Center For ALS At Massachusetts General Hospital	Boston	Massachusetts
University of Massachusetts Medical School	Worcester	Massachusetts
Mayo Clinic	Rochester	Minnesota
Providence ALS Center	Portland	Oregon
Temple University Of The Commonwealth System of Higher Education	Philadelphia	Pennsylvania

Other trials for the same condition

Precise Robotically IMplanted Brain-Computer InterfacE Recruiting

Rho Kinase Inhibitor in Amyotrophic Lateral Sclerosis (REAL) Active, not recruiting

PHENOGENE-1A (Cromolyn) Treatment in Patients With Mild to Moderate ALS Recruiting

Control of Assistive Devices Via Brain-Computer Interface Technology Enrolling by invitation

RAPA-501 Therapy for ALS Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06973629 on ClinicalTrials.gov ↗ ← All trials in the USA