Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)
NCT ID: NCT06973629
Last Updated: 2025-05-22
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
NOT_YET_RECRUITING
PHASE3
200 participants
INTERVENTIONAL
2025-06-30
2029-05-01
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study of Autologous MSC-NTF Cells in Patients With ALS
NCT02017912
Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients
NCT03280056
Expanded Access Protocol: Repeated Administration of Nurown® (Autologous MSC-NTF Cells) for the Treatment of ALS
NCT04681118
Repeated Mesenchymal Stem Cell Injections in ALS
NCT04821479
Biomarkers in Different Types of Amyotrophic Lateral Sclerosis (ALS) Patients Being Treated With Edaravone
NCT04097158
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Up to approximately 200 participants are planned to be enrolled and randomized 1:1 to the NurOwn and placebo groups in Part A. All eligible participants who complete Part A will have the option of entering Part B.
The trial includes a 9-week screening period. After the first screening visit (Screening Visit 1), there will be a Screening Visit 2, during which randomization 1:1 to the NurOwn and placebo groups will occur after confirming that all entry criteria are met. Following randomization, bone marrow aspiration will be scheduled. Stem cells from the bone marrow of all participants will be isolated, and then cryopreserved. Prior to each intrathecal (IT) dose administration, cells will be thawed, propagated, and induced into MSC-NTF cells (NurOwn).
In Part A, participants will receive NurOwn or placebo via IT injection every eight weeks at Weeks 0, 8, and 16.
In Part B, participants will receive NurOwn via IT injection every eight weeks at Weeks 24, 32, and 40.
All participants will be offered the option to participate in the collection and storage of blood/serum, CSF, and buccal samples for future analysis of biomarkers and genetic testing.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Debamestrocel - MSC-NTF (NurOwn)
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
Debamestrocel - MSC-NTF (NurOwn)
MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).
Placebo
Placebo is comprised of Dulbecco Modified Eagle Medium (DMEM)
Placebo
Dulbecco Modified Eagle Medium (DMEM).
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Debamestrocel - MSC-NTF (NurOwn)
MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).
Placebo
Dulbecco Modified Eagle Medium (DMEM).
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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.
18 Years
75 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Brainstorm-Cell Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Barrow Neurological Institute
Phoenix, Arizona, United States
University of California San Diego Medical Center
La Jolla, California, United States
University of Southern California
Los Angeles, California, United States
California Pacific Medical Center
San Francisco, California, United States
University of California, San Francisco
San Francisco, California, United States
University of Colorado Anschutz Medical Campus School of Medicine
Aurora, Colorado, United States
Nova Southeastern University
Davie, Florida, United States
Mayo Clinic
Jacksonville, Florida, United States
University of South Florida
Tampa, Florida, United States
Northwestern Medicine
Chicago, Illinois, United States
Sean M. Healey & AMG Center For ALS At Massachusetts General Hospital
Boston, Massachusetts, United States
University of Massachusetts Medical School
Worcester, Massachusetts, United States
Mayo Clinic
Rochester, Minnesota, United States
Providence ALS Center
Portland, Oregon, United States
Temple University Of The Commonwealth System of Higher Education
Philadelphia, Pennsylvania, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Chief Medical Officer, Brainstorm Cell Therapeutics, MD
Role: CONTACT
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
BCT-006
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.