Not provided
Not provided
Not provided
Not provided
Not provided
As recommended by the study's independent Data and Safety Monitoring Board (DSMB)
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
About 213 people with ALS will participate in this study. There will be locations in North and South America.
During the first part, participants will be randomly assigned to a group (like by flipping a coin). Out of every 3:
During the second part, everyone will get the study drug.
Participation will help doctors find out if Acthar can help or slow down the symptoms of ALS better than placebo.
This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult participants with ALS. Approximately 213 participants will be enrolled.
Following a screening period of up to 28 days, participants with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units [U]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3-week taper.
Participants who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase in which all participants will receive Acthar 0.2 mL (16 U) daily.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm A: Treatment Period Acthar | Experimental | Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39. |
|
| Arm B: Treatment Period Placebo | Placebo Comparator | Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (Matching Placebo), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39. |
|
| Arm C: Extension Period Acthar-Acthar | Experimental | Participants who receive Acthar during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. |
|
| Arm D: Extension Period Placebo-Acthar | Experimental | Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Acthar | Drug | Repository corticotropin for subcutaneous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) | The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival. ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory) A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function. | Baseline, Week 36 |
| Number of Participants Experiencing an Adverse Event During the Treatment Period | Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events. | by the end of the treatment period (within 36 Weeks) |
| Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period | Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events. | by the time of database lock (within 84 weeks) |
| Measure | Description | Time Frame |
|---|---|---|
| Treatment Period: Spirometry (%) | Spirometry (meaning the measuring of breath) is the most common of the lung function tests. It measures how much air can be inhaled [Forced Vital Capacity (FVC)] and exhaled [(Forced Expiratory Volume in one second (FEV1)]. | Baseline, Week 36 |
| Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Clinical Team Leader | Mallinckrodt | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Neuromuscular Research Center | Phoenix | Arizona | 85028 | United States | ||
| Mayo Clinic - Arizona |
Not provided
Not provided
Not provided
Not provided
Not provided
Of 207 potential participants screened, 143 were randomized to begin treatment
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Arm A: Randomized Treatment Period (RTP) Acthar Gel | Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks. Those who do not continue into the extension period had 3 weeks of tapering off the drug, ending their participation by Week 39. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Randomized Treatment Period (RTP) |
|
Not provided
Not provided
| 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 | Aug 11, 2017 |
Not provided
Not provided
Participants will be assigned to treatment group randomly (like flipping a coin). They will have a 2 out of 3 chance of receiving the study drug, and a 1 out of 3 chance of receiving placebo during the treatment period. All participants who continue into the extension period receive Acthar.
Not provided
Not provided
The Treatment Period is defined as Randomized (2:1, Arm A: Arm B), Double-blind (with care provider and outcomes assessor also blinded).
The Extension Period has no masking, because all who participate receive open label study drug.
|
| Placebo | Drug | Matching placebo for subcutaneous injection |
|
|
The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory). The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function. |
| Baseline, Week 36 |
| Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R | The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory). The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function. | Baseline, Week 84 |
| Scottsdale |
| Arizona |
| 85259 |
| United States |
| University of California San Diego | La Jolla | California | 92037 | United States |
| Loma Linda University Health System, Department of Neurology | Loma Linda | California | 92354 | United States |
| Keck School of Medicine, University of Southern California | Los Angeles | California | 90033 | United States |
| University of California Los Angeles | Los Angeles | California | 90095 | United States |
| University of California Irvine Medical Center | Orange | California | 92868 | United States |
| California Pacific Medical Center | San Francisco | California | 94115 | United States |
| University of California San Francisco | San Francisco | California | 94143 | United States |
| Colorado Springs Neurological Associates | Colorado Springs | Colorado | 80907 | United States |
| Georgetown University | Washington D.C. | District of Columbia | 20007 | United States |
| George Washington University | Washington D.C. | District of Columbia | 20037 | United States |
| University of Florida - McKnight Brain Institute | Gainesville | Florida | 32611 | United States |
| University of South Florida | Tampa | Florida | 33612 | United States |
| Emory University | Atlanta | Georgia | 30322 | United States |
| Augusta University | Augusta | Georgia | 30912 | United States |
| Indiana University-Neuroscience Center of Excellence/Goodman Hall | Indianapolis | Indiana | 46202 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| University of Kentucky Chandler Medical Center | Lexington | Kentucky | 40536 | United States |
| John Hopkins Outpatient Center | Baltimore | Maryland | 21287 | United States |
| University of Massachusetts Medical School | Worcester | Massachusetts | 01655 | United States |
| Mercy Health- Saint Mary's | Grand Rapids | Michigan | 49503 | United States |
| Neurology Associates | Lincoln | Nebraska | 68510 | United States |
| University of Nebraska Medical Center - Physicians Clinical Neurosciences Center | Omaha | Nebraska | 68198 | United States |
| Las Vegas Clinic | Las Vegas | Nevada | 89145 | United States |
| Jersey Shore University Medical Center | Neptune City | New Jersey | 07753 | United States |
| Columbia Presbyterian Hospital | New York | New York | 10032 | United States |
| Providence ALS Center | Portland | Oregon | 97213 | United States |
| Penn State Health Milton S. Hershey Medical Center | Hershey | Pennsylvania | 17033 | United States |
| Temple University School of Medicine | Philadelphia | Pennsylvania | 19140 | United States |
| Allegheny General Hospital | Pittsburgh | Pennsylvania | 15212 | United States |
| Wesley Neurology Clinic | Cordova | Tennessee | 38018 | United States |
| Austin Neuromuscular Center | Austin | Texas | 78756 | United States |
| Texas Neurology, P.A. | Dallas | Texas | 75214 | United States |
| The Methodist Hospital | Houston | Texas | 77030 | United States |
| University of Vermont Medical Center | Colchester | Vermont | 05401 | United States |
| VCU Medical Center | Richmond | Virginia | 23298 | United States |
| Swedish Neuroscience Institute | Seattle | Washington | 98122 | United States |
| Medical College of Wisconsin/Froedtert Hospital | Milwaukee | Wisconsin | 53226 | United States |
| IADIN | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1015ABR | Argentina |
| STAT Research | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1023AAB | Argentina |
| DIABAID | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1061ABD | Argentina |
| INEBA | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1192AAW | Argentina |
| Hospital Italiano de Buenos Aires | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1199ABB | Argentina |
| Hospital Español | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1209AAB | Argentina |
| Hospital Británico de Buenos Aires | Ciudad Autonoma de Buenos Aires | Buenos Aires | C1280AEB | Argentina |
| ILAIM | Córdoba | Córdoba Province | X5000BNB | Argentina |
| Fundación Scherbovsky | Mendoza | Mendoza Province | CP 5500 | Argentina |
| Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL ) | Santa Fe | Santa Fe Province | S3000ASL | Argentina |
| Edmonton Kaye Clinic | Edmonton | Alberta | T6G 1Z1 | Canada |
| Recherche Sepmus inc | Greenfield Park | Quebec | J4V 2J2 | Canada |
| Centre de recherché du Centre Hospitalier de l'Universite de Montreal (CRCHUM) | Montreal | Quebec | H2XOA9 | Canada |
| Montreal Neurological Institute & Hospital | Montreal | Quebec | H3A 2B4 | Canada |
| Centro de Trastornos del Movimiento (CETRAM) | Santiago | Santiago Metropolitan | 8380815 | Chile |
| Clinica Dávila | Santiago | Santiago Metropolitan | 8431657 | Chile |
| Biomedica Research Group AV Salvador 149, oficina 1101 | Santiago | 7500710 | Chile |
| Centro de Investigaciones Clínicas SAS | Cali | 760036 | Colombia |
| Clinical Research Institute Saltillo S.A. de C.V. | Saltillo | Coahuila | 25020 | Mexico |
| Hospital Universitario "Dr. José Eleuterio González" | Monterrey | Nuevo León | 64460 | Mexico |
| SMIQ BRCR Global México | Querétaro City | Querétaro | 76090 | Mexico |
| Clinical Research Institute S.C. | San Lucas Tepetlacalco | Tlalnepantla De Baz | 54055 | Mexico |
| Centro Especializado en Investigación Clínica S.C. | Boca del Río | Veracruz | 94290 | Mexico |
| Phylasis Clinicas Research | Mexico City | 54769 | Mexico |
| FAICIC Clinical Researc | Veracruz | 91900 | Mexico |
| Hospital Nivel IV Carlos Alberto Seguin Escobedo | Arequipa | 04001 | Peru |
| Hospital Nacional IV Alberto Sabogal Sologuren | Callao | 07016 | Peru |
| Hospital Almenara | Lima | 15033 | Peru |
| Instituto Neuro Cardiovascular de las Américas | Lima | 15074 | Peru |
| Hospital Nacional Cayetano Heredia | Lima | 15102 | Peru |
| Arm B: RTP Placebo |
Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks. Those who do not continue into the extension period had 3 weeks of simulated tapering, ending their participation by Week 39. |
| FG002 | Arm C: Open Label Extension Period (OLE) Acthar Gel-Acthar Gel | Participants who received Acthar Gel during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. |
| FG003 | Arm D: OLE Placebo-Acthar Gel | Participants who received Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. |
| Intention to Treat (ITT) Population |
|
| Safety Population |
|
| Modified ITT (mITT) |
|
| Completed Taper | Defined as completing the End of RTP Taper Visit at about Week 39 or an early termination visit |
|
| Completed 36 Weeks of Treatment |
|
| Completed RTP | Defined as completing 36 weeks of treatment, followed by Week 39 visit or entering the OLE period |
|
| Enrolled in OLE Period |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open Label Extension Period (OLE) |
|
|
Safety population
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Arm A: RTP Acthar Gel | Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39. |
| BG001 | Arm B: RTP Placebo | Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| 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 | Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) | The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival. ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory) A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function. | Safety Population | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 36 |
|
|
| ||||||||||||||||||||||||||||
| Primary | Number of Participants Experiencing an Adverse Event During the Treatment Period | Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events. | Safety Population | Posted | Count of Participants | Participants | by the end of the treatment period (within 36 Weeks) |
|
| ||||||||||||||||||||||||||||||
| Primary | Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period | Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events. | Safety Population | Posted | Count of Participants | Participants | by the time of database lock (within 84 weeks) |
| |||||||||||||||||||||||||||||||
| Secondary | Treatment Period: Spirometry (%) | Spirometry (meaning the measuring of breath) is the most common of the lung function tests. It measures how much air can be inhaled [Forced Vital Capacity (FVC)] and exhaled [(Forced Expiratory Volume in one second (FEV1)]. | Safety population | Posted | Mean | Standard Deviation | liters | Baseline, Week 36 |
|
| |||||||||||||||||||||||||||||
| Secondary | Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R | The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory). The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function. | Safety Population, with an actual score at the given time point | Posted | Mean | Standard Deviation | score on a scale | Baseline, Week 36 |
| ||||||||||||||||||||||||||||||
| Secondary | Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R | The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory). The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function. | OLE population with scores at the given week; baseline is defined as the value at randomization (week 0) | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 84 |
|
From the start to the end of the period; RTP is 36 weeks (plus 3 weeks if ending there), OLE is up to 21 months (up to Week 84)
Treatment-emergent adverse events (TEAEs) were collected. A TEAE is defined as an adverse event that starts or worsens on or after first dose of study drug and within 28 days from the last dose date of the study drug (For subjects entered into OLE, any TEAE started prior to first OLE dose is counted in RTP). For each system organ class and preferred term, subjects are counted only once, even if they experienced multiple events in that system organ class or preferred term.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Arm A: RTP Acthar Gel | Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar Gel), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39. | 2 | 95 | 13 | 95 | 71 | 95 |
| EG001 | Arm B: RTP Placebo | Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (matching Placebo), daily for up to 36 weeks. Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39. | 3 | 47 | 6 | 47 | 34 | 47 |
| EG002 | Arm C: OLE Acthar Gel-Acthar Gel | Participants who receive Acthar Gel during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar Gel, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. | 1 | 25 | 4 | 25 | 18 | 25 |
| EG003 | Arm D: OLE Placebo-Acthar Gel | Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months. | 2 | 8 | 2 | 8 | 7 | 8 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory arrest | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Haemothorax | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Cellulitis staphylococcal | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Empyema | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Pyelonephritis acute | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urosepsis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Failure to thrive | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Amyotrophic lateral sclerosis | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cerebellar infarction | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypertensive emergency | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Mental status changes | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Suicidal ideation | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Constipation | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dry Mouth | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Salivary hypersecretion | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Blood pressure increased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Weight decreased | Investigations | MedDRA (19.1) | Systematic Assessment |
| |
| Increased appetite | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle atrophy | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Neck Pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Muscle contractions involuntary | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Excessive cerumen production | Ear and labyrinth disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Inflammatory bowel disease | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Pain | General disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (19.1) | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA (19.1) | Systematic Assessment |
| |
| Abnormal loss of weight | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Diabetes mellitus | Metabolism and nutrition disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Bursitis | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Mobility decreased | Musculoskeletal and connective tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Tremor | Nervous system disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Chronic respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Dyspnoea exertional | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Increased viscosity of upper respiratory secretion | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Skin discolouration | Skin and subcutaneous tissue disorders | MedDRA (19.1) | Systematic Assessment |
| |
| Hot flush | Vascular disorders | MedDRA (19.1) | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information Call Center | Mallinckrodt | 800-556-3314 | 5 | clinicaltrials@mnk.com |
| Aug 14, 2020 |
| Prot_SAP_000.pdf |
Not provided
| 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 |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000324 | Adrenocorticotropic Hormone |
| ID | Term |
|---|---|
| D053486 | Melanocortins |
| D011333 | Pro-Opiomelanocortin |
| D007028 | Hypothalamic Hormones |
| D036361 | Peptide Hormones |
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D010908 | Pituitary Hormones, Anterior |
| D010907 | Pituitary Hormones |
| D009479 | Neuropeptides |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D009419 | Nerve Tissue Proteins |
| D011506 | Proteins |
Not provided
Not provided
| Death |
|
| Disease progression |
|
| Withdrawal by Subject |
|
| Subject Discontinued for Non-efficacy |
|
| Trial Terminated by Sponsor |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Week 36 |
|
|
|
|
| Units | Counts |
|---|---|
| Participants |
|
|
|
| Units |
|---|
| Counts |
|---|
| Participants |
|
|
| Units | Counts |
|---|---|
| Participants |
|
|