A Phase 2 interventional study of Nurown MSC-NTF cells and Placebo in Amyotrophic Lateral Sclerosis (ALS), sponsored by Brainstorm-Cell Therapeutics. Completed at 3 sites in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2024-06-06.
Sponsored by Brainstorm-Cell Therapeutics · Phase 2, Interventional, and Treatment
This is a multi-center, randomized, double blind, placebo controlled study to evaluate the safety and efficacy of autologous (self) transplantation of Neurotrophic factors-secreting Mesenchymal Stromal Cells (MSC-NTF, NurOwn™) in patients with ALS .
MSC-NTF cells are a novel cell-therapeutic approach which is expected to effectively deliver Neurotrophic factors, which are potent survival factors for neurons, directly to the site of damage.
The MSC-NTF cell therapy (NurOwn™) is based on transplantation of autologous bone marrow derived mesenchymal stromal cells (MSC), which are enriched from the patients' own bone marrow, propagated ex vivo and induced to secrete NTFs. The autologous MSC-NTF cells are back-transplanted into the ALS patient into the sites of damage, the spinal cord and the muscles.
NTFs are potent survival factors for embryonic, neonatal, and adult neurons and are considered potential therapeutic candidates for ALS. Delivery of appropriate NTFs to the immediate environment of afflicted neurons in ALS patients is expected to improve their survival and thus slow down disease progression and alleviate symptoms.
Previous open-label clinical trials have shown that MSC-NTF cells treatment was well tolerated and appears to be generally safe. Some initials indications of clinical benefit were also observed in some patients.
This multi-center, randomized, double blind, placebo controlled study will evaluate the safety and efficacy of a single combined intramuscular and intrathecal administration of MSC-NTF cells in early-stage ALS patients. Patients will be followed for approximately three months before transplantation with their autologous MSC-NTF cells or placebo. During this period of time, patient bone-marrow will be harvested and mesenchymal stromal cells will be isolated and expanded. Following treatment patients will be followed for a total of six months at monthly visits.
978 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 277 are open to participants now.
This study's enrollment of 48 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.
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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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
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.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
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.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Single autologous MSC-NTF cells treatment by combined intramuscular and intrathecal administration
Biological: Nurown MSC-NTF cells
Combined intramuscular and intrathecal placebo administration
Biological: Placebo
Single autologous MSC-NTF cells treatment by combined intramuscular and intrathecal administration
Also known as: NurOwn
Excipient administration by combined intramuscular and intrathecal administration
Number of Patients With at Least One Treatment Emergent Adverse Events
Safety assessed based on the incidence of treatment-emergent adverse events (TEAEs) (including serious adverse events \[SAEs\]) including clinically relevant changes in vital signs, clinical laboratory assessments, physical and neurological examinations, and electrocardiogram (ECG) tests, during transplantation of expanded autologous MSC-NTF cells administered on a single occasion via combined intrathecal (IT) administration and intramuscular (IM) injections
Time frame: Up to 24 weeks following the first intrathecal injection, or End of Study
Change in Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) Slopes From the Pre-transplantation Period to the Post-transplantation Period Between the Treatment and Placebo Groups Through 24 Weeks Post-transplantation.
The ALSFRS-R is a quickly administered (10 minutes) ordinal, validated rating scale (ratings 0-4) used to determine participants' assessment of their capability and independence in 12 functional activities. The total score of ALSFRS-R ranges from 0-48, with higher score being better. The slope of ALSFRS-R for the pre- and post-treatment is obtained from a linear regression model using all available data points in the corresponding period, respectively. The change in slope is obtained from a fixed-effect linear model which is defined as the post-treatment slope minus the pre-treatment slope. The unit of the slope is score on a scale per month. A positive change in slope, means that the patient's decline in the ALSFRS-R score is slower than pre-treatment. A negative change in slope, means that the patient decline in the ALSFRS-R score is faster than pre-treatment.
Time frame: Up to 24 weeks following the first intrathecal injection
Change in SVC Slopes From the Pre-transplantation Period to the Post-transplantation Period Between the Treatment and Placebo Groups Through 24 Weeks Post-transplantation
SVC measures the maximum amount of air exhaled in a single breath, this lung function is influenced by gender, height, and age, in a complex, non-linear, way. The SVC reported is a normalized value obtained from a non-linear prediction model that accounts for the influence of participant's gender, height and age. SVC was done 3 times at each visit, in order to capture and report the maximal, effort-dependent, lung function that a participant can deliver. The slope of SVC is obtained from a linear regression model using all available data points in the corresponding period, respectively. The change in slope is from a fixed-effect linear model which is defined as the post-treatment slope minus the pre-treatment slope. The unit of the slope is score on a scale per month. Positive change in slope, means that the patient's decline in the SVC score is slower than pre-treatment. Negative change in slope, means that the patient decline in the SVC score is faster than pre-treatment.
Time frame: Up to 24 weeks following the first intrathecal injection
The subject population was planned to include up to 48 treated adults, aged 18 to 75 years, with ALS who were randomly assigned (3:1) to the treatment group (36 patients) or placebo group (12 patients). 48 patients were randomized and all 48 patients underwent treatment.
| Milestone | Nurown MSC-NTF Cells | Excipient |
|---|---|---|
| Started | 36 | 12 |
| Completed | 33 | 10 |
| Not completed | 3 | 2 |
| Withdrew: Withdrawal by subject | 2 | 1 |
| Withdrew: Miscellaneous | 1 | 1 |
Safety assessed based on the incidence of treatment-emergent adverse events (TEAEs) (including serious adverse events \[SAEs\]) including clinically relevant changes in vital signs, clinical laboratory assessments, physical and neurological examinations, and electrocardiogram (ECG) tests, during transplantation of expanded autologous MSC-NTF cells administered on a single occasion via combined intrathecal (IT) administration and intramuscular (IM) injections
| Participants | Nurown MSC-NTF Cells | Excipient |
|---|---|---|
| Number of Patients With at Least One Treatment Emergent Adverse Events | 36 | 12 |
The ALSFRS-R is a quickly administered (10 minutes) ordinal, validated rating scale (ratings 0-4) used to determine participants' assessment of their capability and independence in 12 functional activities. The total score of ALSFRS-R ranges from 0-48, with higher score being better. The slope of ALSFRS-R for the pre- and post-treatment is obtained from a linear regression model using all available data points in the corresponding period, respectively. The change in slope is obtained from a fixed-effect linear model which is defined as the post-treatment slope minus the pre-treatment slope. The unit of the slope is score on a scale per month. A positive change in slope, means that the patient's decline in the ALSFRS-R score is slower than pre-treatment. A negative change in slope, means that the patient decline in the ALSFRS-R score is faster than pre-treatment.
| score on a scale per month | Nurown MSC-NTF Cells | Excipient |
|---|---|---|
| Change in Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) Slopes From the Pre-transplantation Period to the Post-transplantation Period Between the Treatment and Placebo Groups Through 24 Weeks Post-transplantation. | -0.556 ± 0.2784 | -0.356 ± 0.4821 |
SVC measures the maximum amount of air exhaled in a single breath, this lung function is influenced by gender, height, and age, in a complex, non-linear, way. The SVC reported is a normalized value obtained from a non-linear prediction model that accounts for the influence of participant's gender, height and age. SVC was done 3 times at each visit, in order to capture and report the maximal, effort-dependent, lung function that a participant can deliver. The slope of SVC is obtained from a linear regression model using all available data points in the corresponding period, respectively. The change in slope is from a fixed-effect linear model which is defined as the post-treatment slope minus the pre-treatment slope. The unit of the slope is score on a scale per month. Positive change in slope, means that the patient's decline in the SVC score is slower than pre-treatment. Negative change in slope, means that the patient decline in the SVC score is faster than pre-treatment.
| score on a scale per month. | Nurown MSC-NTF Cells | Excipient |
|---|---|---|
| Change in SVC Slopes From the Pre-transplantation Period to the Post-transplantation Period Between the Treatment and Placebo Groups Through 24 Weeks Post-transplantation | -0.73 ± 0.430 | -0.31 ± 0.769 |
Collected over From the screening visit through the end of the study period (24 weeks after first treatment). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Nurown MSC-NTF Cells | 0/36 (0%) | 8/36 (22.2%) | 36/36 (100%) |
| Placebo | 0/12 (0%) | 1/12 (8.3%) | 12/12 (100%) |
| Event | Nurown MSC-NTF Cells | Placebo |
|---|---|---|
| DysphagiaGastrointestinal disorders | 6/36 | 1/12 |
| Breast hyperplasiaReproductive system and breast disorders | 1/11 | 0/2 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 2/36 | 0/12 |
| Acute respiratory failureRespiratory, thoracic and mediastinal disorders | 1/36 | 0/12 |
| Respiratory fatigueRespiratory, thoracic and mediastinal disorders | 1/36 | 0/12 |
| Stoma site painInjury, poisoning and procedural complications | 1/36 | 0/12 |
| HypophagiaMetabolism and nutrition disorders | 1/36 | 0/12 |
| Event | Nurown MSC-NTF Cells | Placebo |
|---|---|---|
| HeadacheNervous system disorders | 27/36 | 6/12 |
| FallInjury, poisoning and procedural complications | 26/36 | 9/12 |
| Back painMusculoskeletal and connective tissue disorders | 26/36 | 1/12 |
| Injection site bruisingGeneral disorders | 19/36 | 5/12 |
| Muscular weaknessMusculoskeletal and connective tissue disorders | 17/36 | 6/12 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 12/36 | 0/12 |
| PyrexiaGeneral disorders | 12/36 | 0/12 |
| Muscle spasmsMusculoskeletal and connective tissue disorders | 10/36 | 2/12 |
| Musculoskeletal painMusculoskeletal and connective tissue disorders | 10/36 | 2/12 |
| Injection site painGeneral disorders | 10/36 | 1/12 |
The Full Analysis Set (FAS) includes all participants who were randomized. The FAS was analyzed based upon treatment group subjects were randomized to.
| Age, Continuous(years) | Nurown MSC-NTF Cells | Excipient | Total |
|---|---|---|---|
| Mean | 50.3 ± 11.90 | 53.5 ± 9.11 | 51.1 ± 11.27 |
| Sex: Female, Male(Participants) | Nurown MSC-NTF Cells | Excipient | Total |
|---|---|---|---|
| Female | 11 | 2 | 13 |
| Male | 25 | 10 | 35 |
| Region of Enrollment(participants) | Nurown MSC-NTF Cells | Excipient | Total |
|---|---|---|---|
| United States | 36 | 12 | 48 |
| El Escorial Criteria(Participants) | Nurown MSC-NTF Cells | Excipient | Total |
|---|---|---|---|
| Possible | 3 | 1 | 4 |
| Laboratory-supported Probable | 5 | 1 | 6 |
| Probable | 16 | 7 | 23 |
| Definite | 12 | 3 | 15 |
| ALS Medical History since diagnosis(months) | Nurown MSC-NTF Cells | Excipient | Total |
|---|---|---|---|
| Mean | 9.00 ± 5.578 | 9.01 ± 4.637 | 9.00 ± 5.311 |
| ALS Medical History since first symptom(months) | Nurown MSC-NTF Cells | Excipient | Total |
|---|---|---|---|
| Mean | 17.62 ± 3.796 | 16.75 ± 3.104 | 17.40 ± 3.624 |
PDFs hosted by the registry, ClinicalTrials.gov.
The record gives a start of May 2014, a primary completion of Mar 2016 and a completion of Jul 2016. It does not say whether these dates are actual or estimated.
Results were first posted in Jun 2024, 8 years 3 months after primary completion. A results delay was requested in Jul 2018.
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Studies of the drug tested here: Debamestrocel
Amyotrophic Lateral Sclerosis →
Brainstorm-Cell Therapeutics