A Phase 3 interventional study of Debamestrocel - MSC-NTF (NurOwn) and Placebo in Amyotrophic Lateral Sclerosis (ALS) and Amyotrophic Lateral Sclerosis, sponsored by Brainstorm-Cell Therapeutics. Not yet recruiting at 15 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2025-05-22.
Sponsored by Brainstorm-Cell Therapeutics · Phase 3, Interventional, and Treatment
The goal of this two-part clinical trial is:
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.
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.
978 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 277 are open to participants now.
This study's planned enrollment of 200 is above the median of 38 across 662 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →Brainstorm-Cell Therapeutics is the lead sponsor of 7 studies on the registry; 1 is open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 is comprised of Dulbecco Modified Eagle Medium (DMEM)
Biological: Placebo
MSC-NTF cells suspended in excipient Dulbecco Modified Eagle Medium (DMEM).
Also known as: NurOwn
Dulbecco Modified Eagle Medium (DMEM).
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.
Time frame: 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).
Time frame: From baseline to Week 48
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)
Time frame: 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
Time frame: From baseline to Week 24
Change in NfL from baseline to Week 24
To evaluate the effect of NurOwn compared to placebo on neurodegeneration based on neurofilament light (NfL) biomarker measurement
Time frame: From baseline to Week 24
Change in ALSAQ-40 scores from baseline to Week 24
To evaluate NurOwn compared to placebo in the treatment of participants with ALS based on the ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire) total score. The ALSAQ-40 is a patient-reported, 40-item questionnaire that measures five areas of health status or domains. Each item is scored on a 5-point Likert scale that ranges from 0 to 4. For the total score, the sum of the item scores for each domain is divided by the maximum possible score for that domain and then multiplied by 100 with the total score ranging between 0-100. A lower score indicates a higher health-related quality of life.
Time frame: From baseline to Week 24
Change in Zarit Caregiver Burden Interview scores from baseline to Week 24
To evaluate NurOwn compared to placebo in the treatment of participants with ALS based on the Zarit Caregiver Burden Interview (ZBI). The ZBI consists of 22 items rated on a 5-point Likert scale that ranges from 0 (never) to 4 (nearly always) with the sum of scores ranging between 0-88. Higher scores indicate greater burden.
Time frame: From baseline to Week 24
Plan to share: Undecided
No publications or documents are linked to this record.
Start is estimated for Jun 2025; primary completion for Nov 2028, 40 months later.
Of 24 phase 3 interventional studies under Amyotrophic Lateral Sclerosis, half reached primary completion within 28 months of starting (the median), counting those still running; a quarter within 25 and three quarters within 60. This study's planned 40 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is not yet recruiting, as verified in May 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Debamestrocel
Amyotrophic Lateral Sclerosis →
Brainstorm-Cell Therapeutics