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| ID | Type | Description | Link |
|---|---|---|---|
| NIP-ALS-2013 | Other Identifier | Other | |
| 2013P000981 | Other Identifier | Other |
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| Name | Class |
|---|---|
| ALS Association | OTHER |
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This is a multicenter, 15-month study evaluating the effect of immunosuppression treatment on the rate of change on the ALS Functional Rating Scale (Revised) (ALSFRS-R) score in up to 33 subjects with Amyotrophic Lateral Sclerosis (ALS).
In an ongoing safety trial of neural stem cell injections into the spinal cord of patients with ALS at Emory University, Atlanta, Georgia, one patient has demonstrated clear improvement by objective clinical and electrophysiological measures, a finding that is unheard of in patients with ALS.
This patient had an improvement in ALSFRS-R by 1.4 points per month. In 826 historical controls from the Northeast ALS Consortium (NEALS) and the Western ALS Consortium (WALS) database where ALSFRS-R was documented at 2 or more visits, there have been no patients that have shown improvement in ALSFRS as seen in this case. Additionally, 5 patients in the stem cell trial who were not on mechanical ventilators at the time of surgery seem to have very slow disease progression as compared to the expectation from current understanding of typical disease course. This observation raises consideration for a disease-modifying effect of the novel immunosuppression regimen used in this trial. Also, given that ALS is clinically an extraordinarily heterogeneous disease, the diagnosis of ALS may represent a group of phenotypically similar but pathogenically variable disorders. It is possible that there exists a subset of patients with an immune-responsive ALS subtype that has not been previously recognized.
Recent studies have furthered the understanding of the immune mechanisms that contribute to ALS progression. Microglia and lymphocytes have both neurotoxic and neuroprotective functions depending on activation states and physiologic conditions within the nervous system. Therefore, targeted immunotherapies that proportionally suppress neurotoxic immune elements, while sparing or promoting protective elements, seemingly have more potential to modify disease course in ALS than previously tested regimens. It is postulated that the immunosuppression treatment given to the stem cell patients may have exhibited neuroprotective effects by favorably promoting the ratio of regulatory T cells and other protective immune mediators in relation to neurotoxic immune modulators. It is hoped that this trial will optimize the chance of replicating these findings and advance knowledge about the complex changes that occur within the immune system in patients with ALS before and after treatment with an immunosuppression regimen.
The primary outcome measure will be rate of change of ALSFRS-R. A clinical response will be defined as a rate of change of ALSFRS-R of +6 points over a 6 month period (mean of change of +1 point per month).
Secondary outcome measures will include slow vital capacity (SVC), grip strength, and hand held dynamometry (HHD). The change in rate of progression in clinical measures will be monitored to look for a potential disease-modifying effect of the immunosuppression regimen. Blood and cerebrospinal fluid immune system markers will be also be studied.
If a clinical response is seen among study participants following treatment, further analyses will be conducted to explore any differential effects of immunosuppression in participants with early-stage disease and later-stage disease. To ensure adequate numbers of participants for conditional analyses stratifying by symptom onset date, participants will be enrolled based on symptom onset within 24 months of the screening visit or more than 24 months before screening. All participants will have the same treatment and will be treated as a single group for the analyses of the main study outcomes.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Immunosuppression Regimen | Experimental | Basiliximab Methylprednisolone Prednisone Tacrolimus Mycophenolate mofetil |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Basiliximab | Drug | 20 mg, IV (in the vein) on day 1 and 4. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With an Average Increase in ALSFRS-R Score of One Point Per Month | The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score (best of 48) from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). A clinical response is defined as a rate of change of ALSFRS-R of +6 points over 6 months (mean of +1 point per month), where typically patients with ALS have a decline in ALSFRS-R by an average of -1/month. | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Rate of Change of ALSFRS-R Scores During Treatment Compared to Pre-Treatment | The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability). ALSFRS-R was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in ability over time. |
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Inclusion Criteria for participants with symptom onset within the past 24 months:
Inclusion Criteria for participants with symptom onset greater than 24 months before screening:
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Jonathan D Glass, MD | Emory University | Principal Investigator |
| Christina N Fournier, MD | Emory University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Emory University | Atlanta | Georgia | 30322 | United States | ||
| Massachusetts General Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29308669 | Derived | Fournier CN, Schoenfeld D, Berry JD, Cudkowicz ME, Chan J, Quinn C, Brown RH, Salameh JS, Tansey MG, Beers DR, Appel SH, Glass JD. An open label study of a novel immunosuppression intervention for the treatment of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener. 2018 May;19(3-4):242-249. doi: 10.1080/21678421.2017.1421666. Epub 2018 Jan 8. |
| Label | URL |
|---|---|
| Northeast ALS Consortium Website | View source |
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Participants were recruited for this study from three Northeast ALS Consortium (NEALS) centers in the USA (Emory University in Atlanta, Georgia, Massachusetts General Hospital in Boston, Massachusetts, and University of Massachusetts Medical School in Worcester, Massachusetts. Recruitment occurred between October 2013 and October 2014.
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| ID | Title | Description |
|---|---|---|
| FG000 | Immunosuppression Regimen | All participants received the same treatment intervention consisting of basiliximab, methylprednisolone, prednisone, tacrolimus and mycophenolate mofetil. Doses of each medication varied by the day of treatment (described below) and the treatment period lasted for six months. Basiliximab: 20 mg, IV (in the vein) on day 1 and 4. Methylprednisolone: 125 mg, IV (in the vein) on day 1. Prednisone: 60 mg PO (by mouth) on days 2-7, 40 mg PO days 8-14, 20 mg PO days 15-21, and 10mg PO days 22-28. Tacrolimus: 1-5 mg PO, twice a day (BID) days 2-180. Mycophenolate mofetil: 500 mg PO, BID days 2-7, 500 mg PO each morning and 1000 mg each night, days 8-14, 1000 mg PO BID days 15-180. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Immunosuppression Regimen | All participants received the same treatment intervention consisting of basiliximab, methylprednisolone, prednisone, tacrolimus and mycophenolate mofetil. Doses of each medication varied by the day of treatment and the treatment period lasted for six months. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With an Average Increase in ALSFRS-R Score of One Point Per Month | The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score (best of 48) from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). A clinical response is defined as a rate of change of ALSFRS-R of +6 points over 6 months (mean of +1 point per month), where typically patients with ALS have a decline in ALSFRS-R by an average of -1/month. | All study participants who received treatment are included in the analysis for this outcome measure, including participants who discontinued treatment early (available ALSFRS-R scores were used). | Posted | Count of Participants | Participants | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
Participants were monitored for adverse events (AEs) from the time they signed the consent form until completion of their participation in the study (defined as death, withdrawal of consent, loss to follow up, early termination due to other reasons, or completion of the entire study).
For the purposes of this study, an adverse event is considered any unfavorable and unintended sign (including clinically significant abnormal laboratory findings), symptom, or disease temporally associated with a study, whether or not the adverse event is considered to be related to the drug product under examination.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Immunosuppression Regimen | All participants received the same treatment intervention consisting of basiliximab, methylprednisolone, prednisone, tacrolimus and mycophenolate mofetil. Doses of each medication varied by the day of treatment and the treatment period lasted for six months. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hospital admission for dehyration due to dysphagia from ALS | Nervous system disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fall | General disorders | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jonathan Glass, MD | Emory University | 404-727-3507 | jglas03@emory.edu |
Not provided
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D009468 | Neuromuscular Diseases |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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Not provided
| ID | Term |
|---|---|
| D000077552 | Basiliximab |
| D008775 | Methylprednisolone |
| D008776 | Methylprednisolone Hemisuccinate |
| D011241 | Prednisone |
| D016559 | Tacrolimus |
| D009173 | Mycophenolic Acid |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
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| Methylprednisolone | Drug | 125 mg, IV (in the vein) on day 1. |
|
|
| Prednisone | Drug | 60 mg PO (by mouth) on days 2-7, 40 mg PO days 8-14, 20 mg PO days 15-21, and 10mg PO days 22-28. |
|
|
| Tacrolimus | Drug | 1-5 mg PO, twice a day (BID) days 2-180. |
|
|
| Mycophenolate mofetil | Drug | 500 mg PO, BID days 2-7, 500 mg PO each morning and 1000 mg each night, days 8-14, 1000 mg PO BID days 15-180. |
|
|
| Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
| Mean Rate of Change of Slow Vital Capacity (SVC) During Treatment Compared to Pre-Treatment | Vital capacity (VC), percent of predicted normal, was determined using the slow VC method. SVC measures the amount of air exhaled following a deep breath. For this test, participants hold a mouthpiece in their mouth, breathe in deeply, and breathe out as much air as they can. The test was done seated in a chair and then repeated while lying on an exam table at the Screening Visit. For all other visits, this test was done while seated in a chair. This test takes 15-20 minutes. SVC was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. SVC is a way to measure respiratory insufficiency in persons with ALS and SVC decreases as ALS progresses. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline over time. | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
| Mean Rate of Change of Hand-Held Dynamometry (HHD) During Treatment Compared to Pre-Treatment | Hand held dynamometry (HHD) is a measure of muscle strength and scores decrease as ALS progresses. Six proximal muscle groups were examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group are established from the initial values for each participant. Strength determinations were converted to Z scores and averaged to provide an HHD megascore. The Z-score indicates the number of standard deviations away from the mean of 0. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. HHD was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in strength over time. | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
| Mean Rate of Change in Grip Strength Treatment Compared to Pre-Treatment | Hand grip was measured using a study approved dynamometer to test the maximum isometric strength of the hand and forearm muscles. The grip strength of the left and right hands were analyzed together. Grip strength was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. Grip strength is a measurement of muscle strength and declines as ALS progresses. A positive value means that scores during treatment were higher than pre-treatment scores, indicating an increase in grip strength over time. | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
| Mean Rate of Change of T-cell Subsets in Blood Treatment Compared to Pre-Treatment | Blood was collected for ribonucleic acid (RNA) and the mean rate of decline of T-cells during the 6 month treatment period compared to the pre-treatment period was assessed (blood was collected twice during the 3-month long lead in period). The precise role that T-cells have in ALS is unknown and this study aims to further the understanding of how T-cells operate in persons with ALS. T-cell measurement is a ratio where the relative expression levels of FOXP3 messenger ribonucleic acid (mRNA) was calculated using the Comparative CT Method (ΔΔCT Method), normalizing to β-actin. Samples were obtained during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in T-cells over time. | Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6) |
| Collection of Cerebrospinal Fluid for Future Analysis of Cytokine Levels | Lumbar punctures (LPs) were performed to collect cerebrospinal fluid (CSF). CSF is banked for future use to characterize immune system markers and to further the understanding of the immune factors that contribute to disease progression in ALS. Cytokines are markers of neuroinflammation and can be categorized as neurotoxic or neuroprotective. The role that cytokines play in in ALS progression is still not yet fully understood. | Pre-Treatment Period (two months prior to the start of treatment), Treatment Period (Months 2 and 6), Post-Treatment Period (Month 12) |
| Collection of Blood for Future Analysis of Peripheral Blood Mononuclear Cells (PBMCs) | Blood was drawn and banked for future use in order to characterize immune system markers and further the understanding of the immune factors that contribute to disease progression in ALS. | Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6), Post-Treatment Period (Months 8 and 12) |
| Boston |
| Massachusetts |
| 02114 |
| United States |
| University of Massachusetts Medical School | Worcester | Massachusetts | 01655 | United States |
| Disease progression |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| Timing of Symptom Onset | Count of Participants | Participants |
|
| ID | Title | Description |
|---|---|---|
| OG000 | Immunosuppression Regimen | Participants were monitored for a three month lead-in period prior to beginning treatment. The Baseline Visit 1 occurred within 21 days after the Screening visit and 3 months prior to receiving the first does of the immunosuppressant regimen. Baseline Visits 2 and 3 occurred 2 and 1 month prior to the start of treatment. All participants received the same treatment intervention consisting of basiliximab, methylprednisolone, prednisone, tacrolimus and mycophenolate mofetil. Doses of each medication varied by the day of treatment and the treatment period lasted for six months. |
|
|
| Secondary | Mean Rate of Change of ALSFRS-R Scores During Treatment Compared to Pre-Treatment | The ALS Functional Rating Scale - Revised (ALSFRS-R) is an ordinal rating scale (0 through 4) used to determine the ALS patient's self assessment of their ability and need for assistance in 12 activities or functions. This is a validated scale, both in person and by phone, which provides a total score from four sub-scores which assess speech and swallowing, (bulbar function), use of upper extremities (cervical function), gait and turning in bed (lumbar function), and breathing (respiratory function). Total scores range from 0 (most impaired) to 48 (normal ability). ALSFRS-R was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in ability over time. | All study participants who received treatment are included in the analysis for this outcome measure, including participants who discontinued treatment early (available ALSFRS-R scores were used) | Posted | Mean | Standard Error | units on a scale change per month | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
|
|
|
| Secondary | Mean Rate of Change of Slow Vital Capacity (SVC) During Treatment Compared to Pre-Treatment | Vital capacity (VC), percent of predicted normal, was determined using the slow VC method. SVC measures the amount of air exhaled following a deep breath. For this test, participants hold a mouthpiece in their mouth, breathe in deeply, and breathe out as much air as they can. The test was done seated in a chair and then repeated while lying on an exam table at the Screening Visit. For all other visits, this test was done while seated in a chair. This test takes 15-20 minutes. SVC was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. SVC is a way to measure respiratory insufficiency in persons with ALS and SVC decreases as ALS progresses. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline over time. | All study participants who received treatment are included in the analysis for this outcome measure. | Posted | Mean | Standard Error | percent predicted change per month | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
|
|
|
| Secondary | Mean Rate of Change of Hand-Held Dynamometry (HHD) During Treatment Compared to Pre-Treatment | Hand held dynamometry (HHD) is a measure of muscle strength and scores decrease as ALS progresses. Six proximal muscle groups were examined bilaterally in both upper and lower extremities. Mean and standard deviation for each muscle group are established from the initial values for each participant. Strength determinations were converted to Z scores and averaged to provide an HHD megascore. The Z-score indicates the number of standard deviations away from the mean of 0. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean. HHD was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in strength over time. | All study participants who received treatment are included in the analysis for this outcome measure, including participants who discontinued treatment early. | Posted | Mean | Standard Error | z-score change per month | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
|
|
|
| Secondary | Mean Rate of Change in Grip Strength Treatment Compared to Pre-Treatment | Hand grip was measured using a study approved dynamometer to test the maximum isometric strength of the hand and forearm muscles. The grip strength of the left and right hands were analyzed together. Grip strength was measured during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. Grip strength is a measurement of muscle strength and declines as ALS progresses. A positive value means that scores during treatment were higher than pre-treatment scores, indicating an increase in grip strength over time. | Posted | Mean | Standard Error | pounds change per month | Pre-Treatment Period (3 months prior to the start of treatment, 2 months prior to the start of treatment, and 1 month prior to the start of treatment), Treatment Period (Day 1 and then monthly until Month 6) |
|
|
|
| Secondary | Mean Rate of Change of T-cell Subsets in Blood Treatment Compared to Pre-Treatment | Blood was collected for ribonucleic acid (RNA) and the mean rate of decline of T-cells during the 6 month treatment period compared to the pre-treatment period was assessed (blood was collected twice during the 3-month long lead in period). The precise role that T-cells have in ALS is unknown and this study aims to further the understanding of how T-cells operate in persons with ALS. T-cell measurement is a ratio where the relative expression levels of FOXP3 messenger ribonucleic acid (mRNA) was calculated using the Comparative CT Method (ΔΔCT Method), normalizing to β-actin. Samples were obtained during the 3 month lead in period and the 6 month treatment period. A random slopes model was fit to the lead-in and treatment periods, with a change point when treatment started. The analysis was based on the difference in slope after the change point. A negative value means that scores during treatment were lower than pre-treatment scores, indicating a decline in T-cells over time. | Posted | Mean | Standard Error | fold change per month | Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6) |
|
|
|
| Secondary | Collection of Cerebrospinal Fluid for Future Analysis of Cytokine Levels | Lumbar punctures (LPs) were performed to collect cerebrospinal fluid (CSF). CSF is banked for future use to characterize immune system markers and to further the understanding of the immune factors that contribute to disease progression in ALS. Cytokines are markers of neuroinflammation and can be categorized as neurotoxic or neuroprotective. The role that cytokines play in in ALS progression is still not yet fully understood. | Cerebrospinal fluid was collected from 29 participants and banked for future analysis. | Posted | Count of Participants | Participants | Pre-Treatment Period (two months prior to the start of treatment), Treatment Period (Months 2 and 6), Post-Treatment Period (Month 12) |
|
|
|
| Secondary | Collection of Blood for Future Analysis of Peripheral Blood Mononuclear Cells (PBMCs) | Blood was drawn and banked for future use in order to characterize immune system markers and further the understanding of the immune factors that contribute to disease progression in ALS. | Blood was collected for all 31 participants and banked for future use. | Posted | Count of Participants | Participants | Pre-Treatment Period (2 months prior to the start of treatment), Treatment Period (Day 1 and Months 1, 2, 4, 6), Post-Treatment Period (Months 8 and 12) |
|
|
|
| 2 |
| 31 |
| 5 |
| 31 |
| 31 |
| 31 |
| Hospitalization due to knee injury from a fall | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Hospitalization due to a kidney stone | Renal and urinary disorders | Non-systematic Assessment |
|
| Hospitalization due to pulmonary embolism | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Hospitalization due to bilateral pneumonia | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Asymptomatic T-wave inversions with troponin elevation | Cardiac disorders | Non-systematic Assessment | Asymptomatic T-wave inversions with slight troponin elevation; no indication of an acute cardiac event |
|
| Diarrhea | Gastrointestinal disorders | Non-systematic Assessment |
|
| Muscular Weakness | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Headache | General disorders | Non-systematic Assessment |
|
| Muscular Spasms | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Back Pain | Musculoskeletal and connective tissue disorders | Non-systematic Assessment |
|
| Nausea | General disorders | Non-systematic Assessment |
|
| Cold Sore | Infections and infestations | Non-systematic Assessment |
|
| Tracheal bronchitis | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
|
| Renal insufficiency (elevated creatinine) | Renal and urinary disorders | Non-systematic Assessment |
|
Not provided
Not provided
Not provided
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D007162 |
| Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D011239 | Prednisolone |
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D011244 | Pregnadienediols |
| D018942 | Macrolides |
| D007783 | Lactones |
| D009930 | Organic Chemicals |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |