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This will be a 6-month, widely inclusive, virtual, single-center, open-label pilot trial utilizing a historical control group.
This will be a 6-month, widely inclusive, virtual, single-center, open-label pilot trial utilizing a historical control group. Following informed consent and screening, participants with ALS will take Theracurmin 1 capsule (90mg) twice daily for 6-months. Treatment with the Theracurmin and all study outcome measures and labs are being performed exclusively for research purposes. Collected data includes saliva and stool microbiome sampling, adverse events, concomitant medications, weight and height, Theracurmin treatment evaluations, and Thrive Questionnaires. Participants will be asked to register on the website Patientslikeme.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Open Label Arm | Experimental | The intervention is twice daily dosage of Theracurmin 90 mg capsules. This same dose was used in a successful trial of patients with mild cognitive impairment. Theracurmin HP capsules containing 90 mg curcumin each that will be taken as one capsule twice daily for 6 months. Each capsule contains 300 mg Theracurmin enhanced bioavailable water-dispersible turmeric rhizome complex providing 30% curcumin (90 mg). The content of Theracurmin HP has been independently certified by NSF International under NSF/ANSI 173. |
|
| Healthy Control Arm | No Intervention | We will seek to enroll 50 healthy control participants. We will attempt to enroll one control subject from each enrolled primary participant's home, preferably a spouse or partner of similar age if possible. We plan to use this data to compare the microbiome of control participants to that of the ALS participants at baseline, week 4 and month 6. We will not conduct further follow-up or collect additional samples with the control subjects. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Theracurmin HP | Drug | The intervention is based on the twice daily dosage of Theracurmin 90 mg capsules used in a trial of patients with mild cognitive impairment |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in ALSFRS-R Slope | The ALSFRS-R (ALS Functional Rating Scale-Revised) will be determined at all video/telephone visits. ALSFRS-R is a quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Change in ALSFRS-R scores correlate with change in strength over time, and it is closely associated with quality of life measures and predicted survival. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best. | Starting at week 4 and then once every 30 days for 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With ALS Reversal | Number of participants who have an ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) score that improves by 4 points or more over 6 months. The ALSFRS-R is used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Change in ALSFRS-R scores correlate with change in strength over time, and it is closely associated with quality of life measures and predicted survival. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Richard Bedlack, MD, PhD | Duke University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Duke University Medical Center | Durham | North Carolina | 27705 | United States |
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Patients with ALS who met inclusion criteria were enrolled into the Open Label Arm. The healthy control arm was for people without ALS who came from the same household as the patients.
Patients were recruited through the Duke ALS Clinic over a 16 month period.
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| ID | Title | Description |
|---|---|---|
| FG000 | Open Label Arm | The intervention is twice daily dosage of Theracurmin 90 mg capsules. This same dose was used in a successful trial of patients with mild cognitive impairment. Theracurmin HP capsules containing 90 mg curcumin each that will be taken as one capsule twice daily for 6 months. Each capsule contains 300 mg Theracurmin enhanced bioavailable water-dispersible turmeric rhizome complex providing 30% curcumin (90 mg). The content of Theracurmin HP has been independently certified by NSF International under NSF/ANSI 173. Theracurmin HP: The intervention is based on the twice daily dosage of Theracurmin 90 mg capsules used in a trial of patients with mild cognitive impairment |
| FG001 | Healthy Control Arm | We will seek to enroll 50 healthy control participants. We will attempt to enroll one control subject from each enrolled primary participant's home, preferably a spouse or partner of similar age if possible. We plan to use this data to compare the microbiome of control participants to that of the ALS participants at baseline, week 4 and month 6. We will not conduct further follow-up or collect additional samples with the control subjects. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Baseline Characteristics not collected on the healthy control arm.
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| ID | Title | Description |
|---|---|---|
| BG000 | Open Label Arm | The intervention is twice daily dosage of Theracurmin 90 mg capsules. This same dose was used in a successful trial of patients with mild cognitive impairment. Theracurmin HP capsules containing 90 mg curcumin each that will be taken as one capsule twice daily for 6 months. Each capsule contains 300 mg Theracurmin enhanced bioavailable water-dispersible turmeric rhizome complex providing 30% curcumin (90 mg). The content of Theracurmin HP has been independently certified by NSF International under NSF/ANSI 173. Theracurmin HP: The intervention is based on the twice daily dosage of Theracurmin 90 mg capsules used in a trial of patients with mild cognitive impairment |
| 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 | Change in ALSFRS-R Slope | The ALSFRS-R (ALS Functional Rating Scale-Revised) will be determined at all video/telephone visits. ALSFRS-R is a quickly administered (five minute) ordinal rating scale (ratings 0-4) used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Change in ALSFRS-R scores correlate with change in strength over time, and it is closely associated with quality of life measures and predicted survival. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best. | Participants with ALSFRS-R scores available. | Posted | Mean | Standard Deviation | ALSFRS-R points per week | Starting at week 4 and then once every 30 days for 6 months |
|
Up to 6 months
Adverse Events data not collected on the healthy control arm.
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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 | Open Label Arm | The intervention is twice daily dosage of Theracurmin 90 mg capsules. This same dose was used in a successful trial of patients with mild cognitive impairment. Theracurmin HP capsules containing 90 mg curcumin each that will be taken as one capsule twice daily for 6 months. Each capsule contains 300 mg Theracurmin enhanced bioavailable water-dispersible turmeric rhizome complex providing 30% curcumin (90 mg). The content of Theracurmin HP has been independently certified by NSF International under NSF/ANSI 173. Theracurmin HP: The intervention is based on the twice daily dosage of Theracurmin 90 mg capsules used in a trial of patients with mild cognitive impairment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Pulmonary embolus | Respiratory, thoracic and mediastinal disorders | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhea, loose stools | Gastrointestinal disorders | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Richard Bedlack, MD, PhD | Duke University | 919-668-2839 | richard.bedlack@duke.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Jul 16, 2020 | Aug 25, 2023 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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This will be a 6-month, widely inclusive, largely remote/virtual, single-center, open-label pilot trial utilizing a historical control group.
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| Month 6 |
| Total Number of Adverse Events as Measured by Patient Reporting | Adverse and serious adverse events will be recorded throughout the study. | up to 6 months |
| Enrollment Rate | The number of participants enrolled divided by the number of months it took to enroll them. | up to 6 months |
| Retention as Measured by the Number of Participants Who Completed the 6 Month Study Visit | The percentage of enrolled participants who completed the 6 month study visit. | month 6 |
| Shannon Diversity Index of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. The Shannon diversity index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness). The minimum value is 0, which indicates no diversity (only one species is found). There is no upper limit to the index; the maximum value occurs when all species have the same number of individuals. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Faith's Phylogenetic Diversity of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. Phylogenetic diversity (PD) is a measure of biodiversity, based on phylogeny (the tree of life). PD is defined as equal to the sum of the lengths of all the branches on the tree that span the members of the set. The branch lengths on the tree count the relative number of new features arising along that part of the tree. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, and month 6 |
| Observed Features (Amplicon Sequence Variants, ASV) of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. An amplicon sequence variant (ASV) is any one of the inferred single DNA sequences recovered from a high-throughput analysis of marker genes. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Pielou's Evenness Index of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. Pielou's evenness is an index that measures diversity along with species richness. While species richness is the number of different species in a given area, evenness is the count of individuals of each species in an area. A calculated value of Pielou's evenness ranges from 0 (no evenness) to 1 (complete evenness). Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Shannon Diversity Index of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. The Shannon diversity index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness). The minimum value is 0, which indicates no diversity (only one species is found). There is no upper limit to the index; the maximum value occurs when all species have the same number of individuals. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Faith's Phylogenetic Diversity of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Metagenomic analysis of deidentified selected fecal sample will be done on patients that positively respond to Theracurmin to achieve strain level identification of microbes positively and negatively associated with improved outcomes. Phylogenetic diversity (PD) is a measure of biodiversity, based on phylogeny (the tree of life). PD is defined as equal to the sum of the lengths of all the branches on the tree that span the members of the set. The branch lengths on the tree count the relative number of new features arising along that part of the tree. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Observed Features (Amplicon Sequence Variants, ASVs) of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. An amplicon sequence variant (ASV) is any one of the inferred single DNA sequences recovered from a high-throughput analysis of marker genes. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Pielou's Evenness Index of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Pielou's evenness is an index that measures diversity along with species richness. While species richness is the number of different species in a given area, evenness is the count of individuals of each species in an area. A calculated value of Pielou's evenness ranges from 0 (no evenness) to 1 (complete evenness). Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Baseline, month 1, month 6 |
| Lack of Efficacy |
|
| Lost to Follow-up |
|
| BG001 | Healthy Control Arm | We will seek to enroll 50 healthy control participants. We will attempt to enroll one control subject from each enrolled primary participant's home, preferably a spouse or partner of similar age if possible. We plan to use this data to compare the microbiome of control participants to that of the ALS participants at baseline, week 4 and month 6. We will not conduct further follow-up or collect additional samples with the control subjects. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
| OG001 | Healthy Control Arm | We will seek to enroll 50 healthy control participants. We will attempt to enroll one control subject from each enrolled primary participant's home, preferably a spouse or partner of similar age if possible. We plan to use this data to compare the microbiome of control participants to that of the ALS participants at baseline, week 4 and month 6. We will not conduct further follow-up or collect additional samples with the control subjects. |
|
|
|
| Secondary | Number of Participants With ALS Reversal | Number of participants who have an ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) score that improves by 4 points or more over 6 months. The ALSFRS-R is used to determine patients' assessments of their capability and independence in 12 functional activities. All 12 activities are relevant in ALS. Change in ALSFRS-R scores correlate with change in strength over time, and it is closely associated with quality of life measures and predicted survival. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best. | Two open label arm participants withdrew consent after enrollment. Data not collected on the healthy control arm. | Posted | Count of Participants | Participants | Month 6 |
|
|
|
| Secondary | Total Number of Adverse Events as Measured by Patient Reporting | Adverse and serious adverse events will be recorded throughout the study. | Two open label arm participants withdrew consent after enrollment. Data not collected on the healthy control arm. | Posted | Number | adverse events | up to 6 months |
|
|
|
| Secondary | Enrollment Rate | The number of participants enrolled divided by the number of months it took to enroll them. | Data not collected on the healthy control arm. | Posted | Number | participants per month | up to 6 months |
|
|
|
| Secondary | Retention as Measured by the Number of Participants Who Completed the 6 Month Study Visit | The percentage of enrolled participants who completed the 6 month study visit. | Data not collected on the healthy control arm. | Posted | Count of Participants | Participants | month 6 |
|
|
|
| Secondary | Shannon Diversity Index of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. The Shannon diversity index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness). The minimum value is 0, which indicates no diversity (only one species is found). There is no upper limit to the index; the maximum value occurs when all species have the same number of individuals. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | Shannon diversity index | Baseline, month 1, month 6 | samples | samples |
|
|
|
|
| Secondary | Faith's Phylogenetic Diversity of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. Phylogenetic diversity (PD) is a measure of biodiversity, based on phylogeny (the tree of life). PD is defined as equal to the sum of the lengths of all the branches on the tree that span the members of the set. The branch lengths on the tree count the relative number of new features arising along that part of the tree. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | units of PD | Baseline, month 1, and month 6 | samples | samples |
|
|
|
|
| Secondary | Observed Features (Amplicon Sequence Variants, ASV) of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. An amplicon sequence variant (ASV) is any one of the inferred single DNA sequences recovered from a high-throughput analysis of marker genes. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | ASVs | Baseline, month 1, month 6 | samples | samples |
|
|
|
|
| Secondary | Pielou's Evenness Index of the Oral Microbiome | Comparing the changes in the saliva microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Data will be compared within subjects with ALS to assess possible changes over the length of the study and identify microbial correlates of disease progression. Pielou's evenness is an index that measures diversity along with species richness. While species richness is the number of different species in a given area, evenness is the count of individuals of each species in an area. A calculated value of Pielou's evenness ranges from 0 (no evenness) to 1 (complete evenness). Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | Pielou's evenness index | Baseline, month 1, month 6 | samples | samples |
|
|
|
|
| Secondary | Shannon Diversity Index of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. The Shannon diversity index takes into account the number of species living in a habitat (richness) and their relative abundance (evenness). The minimum value is 0, which indicates no diversity (only one species is found). There is no upper limit to the index; the maximum value occurs when all species have the same number of individuals. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | Shannon diversity index | Baseline, month 1, month 6 | samples | samples |
|
|
|
|
| Secondary | Faith's Phylogenetic Diversity of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Metagenomic analysis of deidentified selected fecal sample will be done on patients that positively respond to Theracurmin to achieve strain level identification of microbes positively and negatively associated with improved outcomes. Phylogenetic diversity (PD) is a measure of biodiversity, based on phylogeny (the tree of life). PD is defined as equal to the sum of the lengths of all the branches on the tree that span the members of the set. The branch lengths on the tree count the relative number of new features arising along that part of the tree. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | units of PD | Baseline, month 1, month 6 | samples | samples |
|
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|
|
| Secondary | Observed Features (Amplicon Sequence Variants, ASVs) of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. An amplicon sequence variant (ASV) is any one of the inferred single DNA sequences recovered from a high-throughput analysis of marker genes. Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | ASVs | Baseline, month 1, month 6 | samples | samples |
|
|
|
|
| Secondary | Pielou's Evenness Index of the Stool Microbiome | The microbiome of study participants will be analyzed in stool samples at enrollment, week 4 and month 6 visits. Changes will be compared in the stool microbiome over time in patients with ALS on Theracurmin to the changes observed in untreated healthy controls. Pielou's evenness is an index that measures diversity along with species richness. While species richness is the number of different species in a given area, evenness is the count of individuals of each species in an area. A calculated value of Pielou's evenness ranges from 0 (no evenness) to 1 (complete evenness). Baseline was compared to post-treatment samples from months 1 and 6 analyzed together. | Participants with samples collected. Post-treatment samples from months 1 and 6 were analyzed together. | Posted | Mean | Full Range | Pielou's evenness index | Baseline, month 1, month 6 | samples | samples |
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|
| 1 |
| 50 |
| 1 |
| 50 |
| 23 |
| 50 |
| EG001 | Healthy Control Arm | We will seek to enroll 50 healthy control participants. We will attempt to enroll one control subject from each enrolled primary participant's home, preferably a spouse or partner of similar age if possible. We plan to use this data to compare the microbiome of control participants to that of the ALS participants at baseline, week 4 and month 6. We will not conduct further follow-up or collect additional samples with the control subjects. | 0 | 0 | 0 | 0 | 0 | 0 |
| Hot flashes | General disorders | Non-systematic Assessment |
|
| COVID Infection | Infections and infestations | Non-systematic Assessment |
|
| Itching | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Increased weakness | General disorders | Non-systematic Assessment |
|
| Malaise | General disorders | Non-systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | Non-systematic Assessment |
|
| Anxiety | Psychiatric disorders | Non-systematic Assessment |
|
| Flu-like symptoms | General disorders | Non-systematic Assessment |
|
| Constipation | Gastrointestinal disorders | Non-systematic Assessment |
|
| Abdominal pain | General disorders | Non-systematic Assessment |
|
| Headache | General disorders | Non-systematic Assessment |
|
| Cramps | General disorders | Non-systematic Assessment |
|
Not provided
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| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
|
| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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| Post-treatment (months 1 and 6 combined) |
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