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

NCT06973629 | Not Yet Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: BCT-006
• Study Type: interventional
• Target: 200
• Locations: 1 country
• Sites: 15

Brief 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.

Conditions

Amyotrophic Lateral Sclerosis (ALS); Amyotrophic Lateral Sclerosis

Keywords

NurOwn; ENDURANCE; Debamestrocel

Outcome Measures

Primary Outcomes (2)

• Change in the ALSFRS-R total score 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.

  From baseline to Week 24

• 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

  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).

  From baseline to Week 48

Secondary Outcomes (2)

• Change in SVC (%predicted) from baseline to Week 24

  From baseline to Week 24

• Change in HHD from baseline to Week 24

  From baseline to Week 24

Other Outcomes (3)

• Change in NfL from baseline to Week 24

  From baseline to Week 24

• Change in ALSAQ-40 scores from baseline to Week 24

  From baseline to Week 24

• Change in Zarit Caregiver Burden Interview scores from baseline to Week 24

  From baseline to Week 24

Study Arms (2)

Debamestrocel - MSC-NTF (NurOwn)

EXPERIMENTAL

NTF-secreting mesenchymal stem cells (MSC-NTF cells) are a novel cell-therapeutic approach aimed at effectively delivering NTFs directly to the site of damage in ALS patients

Biological: Debamestrocel - MSC-NTF (NurOwn)

Placebo

PLACEBO COMPARATOR

Placebo is comprised of Dulbecco Modified Eagle Medium (DMEM)

Biological: Placebo

Interventions

Debamestrocel - MSC-NTF (NurOwn) BIOLOGICAL

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

Also known as: NurOwn

Debamestrocel - MSC-NTF (NurOwn)

Placebo BIOLOGICAL

Dulbecco Modified Eagle Medium (DMEM).

Placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female participants 18 to 75 years old (inclusive) at Screening Visit 1.
• ALS diagnosed as laboratory-supported probable, clinically probable, or definite as defined by the revised El Escorial criteria.
• Having onset of ALS symptoms, including muscle weakness, within 24 months from Screening Visit 1.
• ≥2 points on each item of the ALSFRS-R at the Screening Visit 1.
• ≤45 points on ALSFRS-R total score at Screening Visit 1.
• Upright slow vital capacity (SVC) measure ≥65% of predicted for gender, height, and age at Screening Visit 1.
• Participants must adhere to highly effective methods of contraception as specified in the study protocol.

You may not qualify if:

• Prior stem cell therapy of any kind.
• Active participation in any other ALS interventional study.
• 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.
• Any unstable clinically significant medical condition other than ALS
• 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).
• Other types of motor neuron disease such as primary lateral sclerosis, progressive muscular atrophy, and progressive bulbar palsy.
• Usage of a feeding tube at Screening Visit 1 or Screening Visit 2.
• Pregnant women or women currently breastfeeding.

Sponsors & Collaborators

• Brainstorm-Cell Therapeutics: lead

Study Sites (15)

Barrow Neurological Institute

Phoenix, Arizona, 85013, United States

Location

University of California San Diego Medical Center

La Jolla, California, 92093, United States

Location

University of Southern California

Los Angeles, California, 90033, United States

Location

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

University of California, San Francisco

San Francisco, California, 94143, United States

Location

University of Colorado Anschutz Medical Campus School of Medicine

Aurora, Colorado, 80045, United States

Location

Nova Southeastern University

Davie, Florida, 33328, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Northwestern Medicine

Chicago, Illinois, 60611, United States

Location

Sean M. Healey & AMG Center For ALS At Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

University of Massachusetts Medical School

Worcester, Massachusetts, 01655, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Providence ALS Center

Portland, Oregon, 97213, United States

Location

Temple University Of The Commonwealth System of Higher Education

Philadelphia, Pennsylvania, 19140, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Chief Medical Officer, Brainstorm Cell Therapeutics, MD

CONTACT

201-488-0460; ClinicalTrial@Brainstorm-cell.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Sponsor, CRO, vendors
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 5, 2025
• First Posted: May 15, 2025
• Study Start: June 30, 2025
• Primary Completion (Estimated): November 1, 2028
• Study Completion (Estimated): May 1, 2029
• Last Updated: May 22, 2025

Record last verified: 2025-05

Locations

US (15)