The classic website will no longer be available as of June 25, 2024. Please use the modernized ClinicalTrials.gov.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03280056
Recruitment Status : Completed
First Posted : September 12, 2017
Results First Posted : February 29, 2024
Last Update Posted : February 29, 2024
Sponsor:
Collaborator:
California Institute for Regenerative Medicine (CIRM)
Information provided by (Responsible Party):
Brainstorm-Cell Therapeutics

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Amyotrophic Lateral Sclerosis (ALS)
Interventions Biological: NurOwn® (MSC-NTF cells)
Other: Placebo
Other: Bone Marrow aspiration
Enrollment 196
Recruitment Details The first participant in Study BCT-002-US signed informed consent on 28 August 2017 The last participant visit took place on 29 September 2020. The study was conducted at 6 clinical sites in the USA. A total of 263 participants were screened for the study, and 196 participants (74.5% of participants screened) were randomly assigned (1:1) to treatment with either NurOwn or placebo.
Pre-assignment Details The most common reasons that participants did not meet inclusion criteria were failure to meet the SVC inclusion criterion at the Screening Visit or the required pre-treatment decline in ALSFRSR total score at the randomization visit. From the ITT population, 3 participants in the NurOwn group and 4 participants in the placebo group discontinued prior to treatment.
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo
Hide Arm/Group Description

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10'6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration
Period Title: Overall Study
Started 95 94
Completed 71 73
Not Completed 24 21
Reason Not Completed
Withdrawal by Subject             12             14
Death             9             2
Adverse Event             1             3
Lost to Follow-up             1             1
Physician Decision             0             1
Early Discontinuation             1             0
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo Total
Hide Arm/Group Description

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10'6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration

 Total of all reporting groups
Overall Number of Baseline Participants 95 94 189
Hide Baseline Analysis Population Description
Demographic characteristics were balanced between NurOwn and placebo treatment groups with no statistically significant differences at baseline.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 94 participants 189 participants
<=18 years 0 0 0
Between 18 and 65 years 95 94 189
>=65 years 0 0 0
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Mean Number Analyzed 95 participants 94 participants 189 participants
48.1  (9.71) 49.1  (8.38) 48.6  (9.07)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 94 participants 189 participants
Female
27
  28.4%
35
  37.2%
62
  32.8%
Male
68
  71.6%
59
  62.8%
127
  67.2%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 94 participants 189 participants
Hispanic or Latino
5
   5.3%
3
   3.2%
8
   4.2%
Not Hispanic or Latino
90
  94.7%
91
  96.8%
181
  95.8%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 95 participants 94 participants 189 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
5
   5.3%
7
   7.4%
12
   6.3%
Native Hawaiian or Other Pacific Islander
0
   0.0%
1
   1.1%
1
   0.5%
Black or African American
3
   3.2%
3
   3.2%
6
   3.2%
White
87
  91.6%
81
  86.2%
168
  88.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
2
   2.1%
2
   1.1%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 95 participants 94 participants 189 participants
95 94 189
Baseline Amyotrophic Lateral Sclerosis Functional Rating ScaleScore   [1] 
Mean (Standard Deviation)
Unit of measure:  Units on a scale
Number Analyzed 95 participants 94 participants 189 participants
30.3  (6.5) 31.4  (6.1) 30.9  (6.3)
[1]
Measure Description: 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. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, as measured by quantitative neuromuscular strength testing, and with quality of life measures, and predicted survival. The total score of ALSFRS-R ranges from 0-48, with higher score being better.
1.Primary Outcome
Title The Proportion of NurOwn® Treated Participants With a ≥1.25 Points/Month Improvement in Post-treatment Slope vs. Pre-treatment Slope in ALSFRS-R Score at 28 Weeks Following the First Treatment as Compared to Placebo
Hide Description 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. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, as measured by quantitative neuromuscular strength testing, and with quality of life measures, and predicted survival. The total score of ALSFRS-R ranges from 0-48, with higher score being better.
Time Frame 28 weeks following the first intrathecal injection
Hide Outcome Measure Data
Hide Analysis Population Description
The modified intent-to-treat (mITT) population was defined as all participants who were randomized, treated and had at least three ALSFRS-R assessments: one pre-treatment assessments of ALSFRS-R prior to the baseline assessment, a baseline assessment and one post-treatment assessment.
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo
Hide Arm/Group Description:

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10'6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration
Overall Number of Participants Analyzed 95 94
Measure Type: Count of Participants
Unit of Measure: Participants
YES (%)
31
  32.6%
26
  27.7%
NO (%)
64
  67.4%
68
  72.3%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NurOwn® (MSC-NTF Cells), Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.453
Comments [Not Specified]
Method Chi-squared
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 1.330
Confidence Interval (2-Sided) 95%
0.632 to 2.798
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Number of Participants Whose Disease Progression is Halted or Improved as Measured by a 100% or Greater Improvement in Post-treatment Slope vs. Pre-treatment Slope in ALSFRS-R Score of NurOwn® Treatment vs. Placebo
Hide Description 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. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, as measured by quantitative neuromuscular strength testing, and with quality of life measures, and predicted survival. The total score of ALSFRS-R ranges from 0-48, with higher score being better.
Time Frame 28 weeks following the first intrathecal injection
Hide Outcome Measure Data
Hide Analysis Population Description
The modified intent-to-treat (mITT) population was defined as all participants who were randomized, treated and had at least three ALSFRS-R assessments: one pre-treatment assessments of ALSFRS-R prior to the baseline assessment, a baseline assessment and one post-treatment assessment.
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo
Hide Arm/Group Description:

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10'6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration
Overall Number of Participants Analyzed 95 94
Measure Type: Count of Participants
Unit of Measure: Participants
YES (%)
13
  13.7%
13
  13.8%
NO (%)
82
  86.3%
81
  86.2%
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NurOwn® (MSC-NTF Cells), Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.997
Comments [Not Specified]
Method Chi-squared
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 0.998
Confidence Interval (2-Sided) 95%
0.416 to 2.395
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Score of NurOwn® (MSC-NTF Cells) Treated Patients vs. Placebo Treated Patients as Measured by Change From Baseline in ALSFRS-R Score at Week 28
Hide Description 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. All 12 activities are relevant in ALS. Initial validity was established by documenting that in ALS patients, change in ALSFRS-R scores correlated with change in strength over time, as measured by quantitative neuromuscular strength testing, and with quality of life measures, and predicted survival. The total score of ALSFRS-R ranges from 0-48, with higher score being better.
Time Frame 28 weeks following the first intrathecal injection
Hide Outcome Measure Data
Hide Analysis Population Description
The modified intent-to-treat (mITT) population was defined as all participants who were randomized, treated and had at least three ALSFRS-R assessments: one pre-treatment assessments of ALSFRS-R prior to the baseline assessment, a baseline assessment and one post-treatment assessment.
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo
Hide Arm/Group Description:

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10'6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration
Overall Number of Participants Analyzed 95 94
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
-5.52  (0.670) -5.88  (0.665)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NurOwn® (MSC-NTF Cells), Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.693
Comments [Not Specified]
Method Mixed Models Analysis
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 0.37
Confidence Interval (2-Sided) 95%
-1.47 to 2.20
Parameter Dispersion
Type: Standard Error of the Mean
Value: 0.926
Estimation Comments [Not Specified]
4.Secondary Outcome
Title NurOwn® (MSC-NTF Cells) Treated Patients vs. Placebo Treated Patients as Measured by the Combined Assessment of Function and Survival at 28 Weeks
Hide Description

The combined Assessment of Function and Survival (CAFS) is a composite endpoint based on (1) the change from baseline in ALS Functional Rating Scale-Revised (ALSFRS-R) score and (2) time to death.

On the ALSFRS-R, 12 functions are rated on 5-point ordinal rating scales (from 0 to 4) with a total score range (minimum and maximum score) of 0-48 (sum of all 12 items). The higher the score the better functioning. For the survival endpoint, the longer time the better outcome.

A patient's CAFS score represents a patient's rank in the study based on comparing the patient's outcome for both the change in ALSFRS-R and the time to death to all other patients in the study in a pairwise fashion. The ranked scores range from 001 to 189 (the number of subjects in the mITT population) with larger rank score numbers associated with a better outcome. The reported values are the mean rank scores in each group for the composite endpoint.
Time Frame 28 weeks following the first intrathecal injection
Hide Outcome Measure Data
Hide Analysis Population Description
The modified intent-to-treat (mITT) population was defined as all participants who were randomized, treated and had at least three ALSFRS-R assessments: one pre-treatment assessments of ALSFRS-R prior to the baseline assessment, a baseline assessment and one post-treatment assessment.
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo
Hide Arm/Group Description:

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10'6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration
Overall Number of Participants Analyzed 95 94
Least Squares Mean (Standard Error)
Unit of Measure: score on a scale
73.74  (5.210) 72.21  (4.890)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection NurOwn® (MSC-NTF Cells), Placebo
Comments [Not Specified]
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.804
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter Mean Difference (Final Values)
Estimated Value 1.53
Confidence Interval (2-Sided) 95%
-10.65 to 13.72
Parameter Dispersion
Type: Standard Error of the Mean
Value: 6.176
Estimation Comments [Not Specified]
Time Frame from the screening visit through the end of the study period (28 weeks after first treatment)
Adverse Event Reporting Description AE means any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality.
 
Arm/Group Title NurOwn® (MSC-NTF Cells) Placebo
Hide Arm/Group Description

NurOwn® (MSC-NTF): One course of treatment that includes three separate intrathecal injections of 100-125 x 10^6 cells every 8 weeks

NurOwn® (MSC-NTF): Autologous, bone marrow-derived, mesenchymal stem cells secreting neurotrophic factors

Bone marrow aspiration

One course of treatment that includes three separate intrathecal injections of Placebo every 8 weeks

Placebo: liquid solution in syringe for injection

Bone marrow aspiration
All-Cause Mortality
NurOwn® (MSC-NTF Cells) Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   10/95 (10.53%)      4/94 (4.26%)    
Hide Serious Adverse Events
NurOwn® (MSC-NTF Cells) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   23/95 (24.21%)      17/94 (18.09%)    
Cardiac disorders     
Palpitations *  1/95 (1.05%)  1 0/94 (0.00%)  0
Cardiac arrest *  0/95 (0.00%)  0 1/94 (1.06%)  1
Gastrointestinal disorders     
Dysphagia *  3/95 (3.16%)  3 2/94 (2.13%)  2
Gastrointestinal haemorrhage *  1/95 (1.05%)  1 0/94 (0.00%)  0
General disorders     
Asthenia *  1/95 (1.05%)  1 0/94 (0.00%)  0
Disease progression *  1/95 (1.05%)  1 2/94 (2.13%)  2
Euthanasia *  1/95 (1.05%)  1 0/94 (0.00%)  0
Pain *  1/95 (1.05%)  1 0/94 (0.00%)  0
Immune system disorders     
Anaphylactic reaction *  0/95 (0.00%)  0 1/94 (1.06%)  1
Infections and infestations     
Pneumonia *  2/95 (2.11%)  2 2/94 (2.13%)  3
Intervertebral discitis *  1/95 (1.05%)  1 0/94 (0.00%)  0
Clostridium difficile infection *  0/95 (0.00%)  0 1/94 (1.06%)  1
Sepsis *  0/95 (0.00%)  0 1/94 (1.06%)  1
Injury, poisoning and procedural complications     
Fall *  1/95 (1.05%)  1 0/94 (0.00%)  0
Procedural complication *  1/95 (1.05%)  1 0/94 (0.00%)  0
Procedural pain *  1/95 (1.05%)  1 0/94 (0.00%)  0
Skull fracture *  1/95 (1.05%)  1 0/94 (0.00%)  0
Subdural haematoma *  1/95 (1.05%)  1 0/94 (0.00%)  0
Facial bones fracture *  0/95 (0.00%)  0 1/94 (1.06%)  1
Procedural headache *  0/95 (0.00%)  0 1/94 (1.06%)  1
Procedural nausea *  0/95 (0.00%)  0 1/94 (1.06%)  1
Metabolism and nutrition disorders     
Malnutrition *  1/95 (1.05%)  1 0/94 (0.00%)  0
Dehydration *  0/95 (0.00%)  0 1/94 (1.06%)  1
Nervous system disorders     
Dizziness *  1/95 (1.05%)  1 0/94 (0.00%)  0
Loss of consciousness *  1/95 (1.05%)  1 1/94 (1.06%)  1
Syncope *  1/95 (1.05%)  1 0/94 (0.00%)  0
Cerebral haemorrhage *  0/95 (0.00%)  0 1/94 (1.06%)  1
Cerebrospinal fluid leakage *  0/95 (0.00%)  0 1/94 (1.06%)  1
Renal and urinary disorders     
Urinary retention *  1/95 (1.05%)  1 0/94 (0.00%)  0
Renal colic *  0/95 (0.00%)  0 1/94 (1.06%)  1
Respiratory, thoracic and mediastinal disorders     
Respiratory failure *  5/95 (5.26%)  6 3/94 (3.19%)  3
Respiratory distress *  2/95 (2.11%)  2 0/94 (0.00%)  0
Dyspnoea *  1/95 (1.05%)  1 0/94 (0.00%)  0
Pulmonary embolism *  1/95 (1.05%)  1 1/94 (1.06%)  1
Respiratory arrest *  1/95 (1.05%)  1 0/94 (0.00%)  0
Respiratory muscle weakness *  1/95 (1.05%)  1 0/94 (0.00%)  0
Acute respiratory failure *  0/95 (0.00%)  0 1/94 (1.06%)  1
Pneumonia aspiration *  0/95 (0.00%)  0 1/94 (1.06%)  1
Vascular disorders     
Haemorrhage *  1/95 (1.05%)  1 0/94 (0.00%)  0
Deep vein thrombosis *  0/95 (0.00%)  0 1/94 (1.06%)  1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
NurOwn® (MSC-NTF Cells) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   93/95 (97.89%)      92/94 (97.87%)    
Gastrointestinal disorders     
Nausea *  16/95 (16.84%)  20 18/94 (19.15%)  22
Dysphagia *  9/95 (9.47%)  9 5/94 (5.32%)  5
Constipation *  7/95 (7.37%)  7 6/94 (6.38%)  6
Diarrhoea *  5/95 (5.26%)  6 7/94 (7.45%)  9
Vomiting *  3/95 (3.16%)  4 6/94 (6.38%)  6
General disorders     
Pyrexia *  9/95 (9.47%)  10 9/94 (9.57%)  9
Injection site pain *  8/95 (8.42%)  13 9/94 (9.57%)  10
Chills *  2/95 (2.11%)  3 5/94 (5.32%)  5
Fatigue *  0/95 (0.00%)  0 8/94 (8.51%)  8
Infections and infestations     
Nasopharyngitis *  8/95 (8.42%)  8 5/94 (5.32%)  6
Upper respiratory tract infection *  6/95 (6.32%)  7 12/94 (12.77%)  12
Injury, poisoning and procedural complications     
Procedural pain *  50/95 (52.63%)  145 34/94 (36.17%)  52
Procedural headache *  31/95 (32.63%)  58 30/94 (31.91%)  51
Fall *  28/95 (29.47%)  61 34/94 (36.17%)  76
Post lumbar puncture syndrome *  22/95 (23.16%)  36 29/94 (30.85%)  48
Post procedural complication *  16/95 (16.84%)  22 7/94 (7.45%)  10
Contusion *  9/95 (9.47%)  9 9/94 (9.57%)  16
Laceration *  7/95 (7.37%)  7 11/94 (11.70%)  12
Postoperative fever *  7/95 (7.37%)  7 1/94 (1.06%)  2
Limb injury *  6/95 (6.32%)  7 1/94 (1.06%)  2
Procedural anxiety *  5/95 (5.26%)  7 1/94 (1.06%)  1
Skin abrasion *  3/95 (3.16%)  3 5/94 (5.32%)  8
Musculoskeletal and connective tissue disorders     
Back pain *  42/95 (44.21%)  78 24/94 (25.53%)  33
Pain in extremity *  16/95 (16.84%)  29 11/94 (11.70%)  15
Musculoskeletal pain *  15/95 (15.79%)  16 8/94 (8.51%)  9
Coccydynia *  11/95 (11.58%)  14 1/94 (1.06%)  1
Muscular weakness *  11/95 (11.58%)  15 12/94 (12.77%)  16
Arthralgia *  10/95 (10.53%)  11 7/94 (7.45%)  7
Muscle spasms *  9/95 (9.47%)  14 6/94 (6.38%)  6
Muscle tightness *  7/95 (7.37%)  7 1/94 (1.06%)  1
Myalgia *  7/95 (7.37%)  8 2/94 (2.13%)  2
Neck pain *  7/95 (7.37%)  9 8/94 (8.51%)  8
Musculoskeletal stiffness *  3/95 (3.16%)  4 5/94 (5.32%)  5
Nervous system disorders     
Headache *  45/95 (47.37%)  85 32/94 (34.04%)  57
Dizziness *  5/95 (5.26%)  6 5/94 (5.32%)  5
Dysarthria *  2/95 (2.11%)  3 5/94 (5.32%)  6
Balance disorder *  1/95 (1.05%)  1 5/94 (5.32%)  5
Psychiatric disorders     
Anxiety *  5/95 (5.26%)  5 5/94 (5.32%)  5
Insomnia *  5/95 (5.26%)  5 3/94 (3.19%)  3
Respiratory, thoracic and mediastinal disorders     
Cough *  4/95 (4.21%)  5 7/94 (7.45%)  7
Dyspnoea *  4/95 (4.21%)  4 6/94 (6.38%)  8
*
Indicates events were collected by non-systematic assessment
This trial had a unique and atypical trial population that included more participants than anticipated with advanced ALS, who had a low ALSFRS-R score. As compared with recent large ALS clinical trials, this study population is an outlier, with a mean overall ALSFRS-R score at baseline lower than other studies, and this impacted the power of this study.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Chief Medical Officer
Organization: Brainstorm Cell Therapeutics
Phone: +1-201-488-0460
EMail: ClinicalTrial@brainstorm-cell.com
Layout table for additonal information
Responsible Party: Brainstorm-Cell Therapeutics
ClinicalTrials.gov Identifier: NCT03280056    
Other Study ID Numbers: BCT-002-US
First Submitted: August 29, 2017
First Posted: September 12, 2017
Results First Submitted: December 1, 2023
Results First Posted: February 29, 2024
Last Update Posted: February 29, 2024