Amyotrophic Lateral Sclerosis (ALS)Phase 3NCT06973629
Efficacy and Safety of MSC-NTF (NurOwn) in Participants With Early Symptomatic ALS and Moderate Disease Presentation in ALS (ENDURANCE STUDY)
A Phase 3 study testing Debamestrocel - MSC-NTF (NurOwn) in people with Amyotrophic Lateral Sclerosis (ALS) and Amyotrophic Lateral Sclerosis, run by Brainstorm-Cell Therapeutics at 15 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis
- What is being tested?
- Debamestrocel - MSC-NTF (NurOwn)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 200 participants
- Where
- 15 sitesUnited States
- Study dates
- 30 Jun 2025to 1 May 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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).
- Primary brain cancer or cancer with CNS involvement is exclusionary.
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- 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.
Trial locations and contacts
15 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Chief Medical Officer, Brainstorm Cell Therapeutics, MD contact
15 sites in 1 country
United States15 sites
University of Colorado Anschutz Medical Campus School of Medicine
Aurora, Colorado, 80045
Principal Investigator, 720-848-0000, contact@cuanschutz.edu
Sean M. Healey & AMG Center For ALS At Massachusetts General Hospital
Boston, Massachusetts, 02114
Principal Investigator, 617-410-4534, contact@lahey.org
Northwestern Medicine
Chicago, Illinois, 60611
Principal Investigator, 312-926-2000, contact@northwestern.edu
Nova Southeastern University
Davie, Florida, 33328
Principal Investigator, 800-541-6682, contact@nova.edu
Mayo Clinic
Jacksonville, Florida, 32224
Principal Investigator, 904-953-0853, contact@mayo.edu
University of California San Diego Medical Center
La Jolla, California, 92093
Principal Investigator, 858-657-7000, contact@ucsd.edu
University of Southern California
Los Angeles, California, 90033
Principal Investigator, 213-740-2311, contact@usc.edu
Temple University Of The Commonwealth System of Higher Education
Philadelphia, Pennsylvania, 19140
Principal Investigator, 215-204-7000, contact@tuhs.temple.edu
Barrow Neurological Institute
Phoenix, Arizona, 85013
Principal Investigator, 844-324-6684, contact@commonspirit.org
Providence ALS Center
Portland, Oregon, 97213
Principal Investigator, 503-215-8580, contact@providence.org
Mayo Clinic
Rochester, Minnesota, 55905
Principal Investigator, contact@mayo.edu
California Pacific Medical Center
San Francisco, California, 94115
Principal Investigator, 415-600-3604, contact@cpmccri.org
University of California, San Francisco
San Francisco, California, 94143
Principal Investigator, 415-476-1000, contact@ucsf.edu
University of South Florida
Tampa, Florida, 33612
Principal Investigator, 813-974-2011, contact@usf.edu
University of Massachusetts Medical School
Worcester, Massachusetts, 01655
Principal Investigator, 508-856-8989, contact@@umassmemorial.org
About this study
Official title: A Two-Part Phase 3b Randomized, Double-Blind, Placebo-Controlled, Followed by Open-Label Extension, Multicenter Study of the Efficacy and Safety of MSC-NTF (NurOwn) in Participants With With Early Symptomatic and Moderate Disease Presentation in Amyotrophic Lateral Sclerosis (ALS)
The goal of this two-part clinical trial is:
- 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.
Read more
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.
Read the detailed description
This is a multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with early symptomatic ALS and moderate disease presentation in ALS. The study comprises two parts: a 24-week randomized, double blind placebo controlled period (Part A) followed by a 24-week open label expansion period (Part B).
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.
- Being tested
- Debamestrocel - MSC-NTF (NurOwn), Placebo
- Keywords
- NurOwn, ENDURANCE, Debamestrocel
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Debamestrocel - MSC-NTF (NurOwn)Biological
Also called NurOwn
MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).
PlaceboBiological
Dulbecco Modified Eagle Medium (DMEM).
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Biological: Debamestrocel - MSC-NTF (NurOwn) |
| Placebo | Placebo comparator | Placebo is comprised of Dulbecco Modified Eagle Medium (DMEM) Receives: Biological: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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).
Measured: From baseline to Week 48
Secondary outcomes (2)
Change in SVC (%predicted) from baseline to Week 24
To evaluate the efficacy of NurOwn compared to placebo on respiratory function based on slow vital capacity (SVC)
Measured: From baseline to Week 24
Change in HHD from baseline to Week 24
To evaluate the efficacy of NurOwn compared to placebo on upper limb muscle strength based on the hand-held dynamometry (HHD) measurement
Measured: From baseline to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2025
- Study start30 Jun 2025 (estimated)
- Primary completion1 Nov 2028 (estimated)
- Study completion1 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis (ALS)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Brainstorm-Cell Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06973629
- Sponsor's protocol ID
- BCT-006
- Status last verified
- May 2025
- First submitted
- 5 May 2025
- Last updated
- 22 May 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Two-Part Phase 3b Randomized, Double-Blind, Placebo-Controlled, Followed by Open-Label Extension, Multicenter Study of the Efficacy and Safety of MSC-NTF (NurOwn) in Participants With With Early Symptomatic and Moderate Disease Presentation in Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT06973629
- Acronym / sponsor ID
- BCT-006
- Keywords
- NurOwn, ENDURANCE, Debamestrocel
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Brainstorm-Cell TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2025 (estimated)
- Primary completion
- 1 Nov 2028 (estimated)
- Study completion
- 1 May 2029 (estimated)
- First posted
- 15 May 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this two-part clinical trial is:
- 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.
Detailed description
This is a multicenter, Phase 3b study to assess the efficacy and safety of NurOwn in participants with early symptomatic ALS and moderate disease presentation in ALS. The study comprises two parts: a 24-week randomized, double blind placebo controlled period (Part A) followed by a 24-week open label expansion period (Part B).
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Debamestrocel - MSC-NTF (NurOwn) | Experimental | Biological: 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 |
| Placebo | Placebo comparator | Biological: PlaceboPlacebo is comprised of Dulbecco Modified Eagle Medium (DMEM) |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (9 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Debamestrocel - MSC-NTF (NurOwn)Also: NurOwnMSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM). | Biological | Debamestrocel - MSC-NTF (NurOwn) |
| PlaceboDulbecco Modified Eagle Medium (DMEM). | Biological | Placebo |
Primary outcome
Change in the ALSFRS-R total score from baseline to Week 24
Time frame: 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
Time frame: 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).
2 secondary outcomes
Change in SVC (%predicted) from baseline to Week 24
Time frame: From baseline to Week 24
Change in HHD from baseline to Week 24
Time frame: From baseline to Week 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 May 2025.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Chief Medical Officer, Brainstorm Cell Therapeutics, MD contact
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Debamestrocel - MSC-NTF (NurOwn) in people with Amyotrophic Lateral Sclerosis (ALS) and Amyotrophic Lateral Sclerosis, run by Brainstorm-Cell Therapeutics at 15 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chief Medical Officer, Brainstorm Cell Therapeutics, MD (201-488-0460, ClinicalTrial@Brainstorm-cell.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the ALSFRS-R total score from baseline to Week 24 (time frame: From baseline to Week 24). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 May 2025.