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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-002191-41 | EudraCT Number |
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To evaluate the safety and efficacy of eculizumab in the treatment of refractory generalized myasthenia gravis (gMG) as an extension study for the participants who previously completed Study ECU-MG-301(NCT01997229).
ECU-MG-302 was an extension study designed to provide the participants who completed Study ECU-MG-301 an opportunity to receive eculizumab and collect clinical data to provide long-term safety and efficacy information on eculizumab in participants with refractory gMG.
After receiving blinded study treatment (eculizumab or placebo) in Study ECU-MG-301 for 26 weeks, participants were eligible to enroll in the ECU-MG-302 extension study. Participants were to enter Study ECU-MG-302 within 2 weeks after completing their Week 26 visit in Study ECU-MG-301.
Study ECU-MG-302 consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, an Open-Label Maintenance Phase (up to 4 years), and a Safety Follow-up visit 8 weeks after the last dose for participants who withdrew from the study or discontinued eculizumab treatment at any time and for any reason after receiving any amount of eculizumab.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eculizumab/Eculizumab | Experimental | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 milligrams [mg]) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. |
|
| Placebo/Eculizumab | Experimental | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eculizumab | Biological | Intravenous administration of eculizumab. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Count Of Participants With Treatment-Emergent Adverse Events | Treatment-emergent adverse events (TEAEs) are adverse events with onset on or after the first study drug dose in Study ECU-MG-302. Likewise, treatment-emergent serious adverse events (TESAEs) are serious adverse events that onset on or after the first study drug dose in Study ECU-MG-302. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. | Day 1 (after dosing) through End of Study (Week 208) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130 | The MG-ADL scale is a validated 8-item patient-reported outcome measure. Participants assessed their functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb impairment (2 items). These 8 items were not weighted and were individually graded from 0 (normal) to 3 (most severe), providing a total MG-ADL score ranging from 0 to 24 points. A reduction in score indicates improvement in condition. Baseline was defined as the last available assessment prior to treatment (first study drug infusion) with eculizumab in Study ECU-MG-302. Change from Baseline in MG-ADL total score at Week 4 (blind induction phase) and at Week 130 (open-label eculizumab phase) are presented. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Marcus Yountz, MD | Alexion Pharmaceuticals, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Birmingham | Alabama | 35233 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30767274 | Result | Muppidi S, Utsugisawa K, Benatar M, Murai H, Barohn RJ, Illa I, Jacob S, Vissing J, Burns TM, Kissel JT, Nowak RJ, Andersen H, Casasnovas C, de Bleecker JL, Vu TH, Mantegazza R, O'Brien FL, Wang JJ, Fujita KP, Howard JF Jr; Regain Study Group. Long-term safety and efficacy of eculizumab in generalized myasthenia gravis. Muscle Nerve. 2019 Jul;60(1):14-24. doi: 10.1002/mus.26447. Epub 2019 Mar 8. | |
| 31115842 |
Not provided
Not provided
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Participants who completed Study ECU-MG-301 (NCT01997229) were eligible to participate in Study ECU-MG-302.
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| ID | Title | Description |
|---|---|---|
| FG000 | Eculizumab/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 milligrams [mg]) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Blind Induction Phase |
|
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Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 19, 2016 | Dec 9, 2019 |
Not provided
Following the Blind Induction Phase, all participants received open-label eculizumab.
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The study consisted of a 4-week Blind Induction Phase to preserve the blinded nature of Study ECU-MG-301, followed by an Open-Label Maintenance Phase.
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| Placebo | Drug | Intravenous administration of matching placebo. Participants received placebo only during the Blind Induction Phase. |
|
| Baseline, Week 4 and Week 130 |
| Los Angeles |
| California |
| 90033 |
| United States |
| Orange | California | 92868 | United States |
| Palo Alto | California | 94304 | United States |
| San Francisco | California | 94115 | United States |
| New Haven | Connecticut | 06519 | United States |
| Jacksonville | Florida | 32209 | United States |
| Miami | Florida | 33136 | United States |
| Tampa | Florida | 33612 | United States |
| Springfield | Illinois | 62702 | United States |
| Indianapolis | Indiana | 46202 | United States |
| Iowa City | Iowa | 52242 | United States |
| Kansas City | Kansas | 66160 | United States |
| Baltimore | Maryland | 21201-1595 | United States |
| Baltimore | Maryland | 21287-0876 | United States |
| Boston | Massachusetts | 02115 | United States |
| Burlington | Massachusetts | 01805 | United States |
| Las Vegas | Nevada | 89145 | United States |
| Chapel Hill | North Carolina | 27599 | United States |
| Charlotte | North Carolina | 28207 | United States |
| Durham | North Carolina | 27710 | United States |
| Columbus | Ohio | 43210 | United States |
| Portland | Oregon | 97239 | United States |
| San Antonio | Texas | 78229 | United States |
| Burlington | Vermont | 05401 | United States |
| Seattle | Washington | 98195 | United States |
| Buenos Aires | Argentina |
| Edegem | Belgium |
| Ghent | Belgium |
| Leuven | Belgium |
| São Paulo | Brazil |
| Edmonton | Canada |
| Ostrava - Poruba | Czechia |
| Prague | Czechia |
| Arhus C | Denmark |
| Copenhagen | Denmark |
| Turku | Finland |
| Szeged | Hungary |
| Milan | Italy |
| Naples | Italy |
| Roma | Italy |
| Chiba | Japan |
| Fukuoka | Japan |
| Hanamaki | Japan |
| Hokkaido | Japan |
| Miyagi | Japan |
| Nagasaki | Japan |
| Osaka | Japan |
| Osaka-Fu | Japan |
| Amsterdam | Netherlands |
| Seoul | South Korea |
| Barcelona | Spain |
| Madrid | Spain |
| Stockholm | Sweden |
| Ankara | Turkey (Türkiye) |
| Izmir | Turkey (Türkiye) |
| Kocaeli | Turkey (Türkiye) |
| Birmingham | United Kingdom |
| Liverpool | United Kingdom |
| London | United Kingdom |
| Result |
| Andersen H, Mantegazza R, Wang JJ, O'Brien F, Patra K, Howard JF Jr; REGAIN Study Group. Correction to: Eculizumab improves fatigue in refractory generalized myasthenia gravis. Qual Life Res. 2019 Aug;28(8):2255. doi: 10.1007/s11136-019-02204-x. |
| 30905021 | Result | Andersen H, Mantegazza R, Wang JJ, O'Brien F, Patra K, Howard JF Jr; REGAIN Study Group. Eculizumab improves fatigue in refractory generalized myasthenia gravis. Qual Life Res. 2019 Aug;28(8):2247-2254. doi: 10.1007/s11136-019-02148-2. Epub 2019 Mar 23. |
| 31698177 | Result | Murai H, Uzawa A, Suzuki Y, Imai T, Shiraishi H, Suzuki H, Okumura M, O'Brien F, Wang JJ, Fujita KP, Utsugisawa K; REGAIN Study Group. Long-term efficacy and safety of eculizumab in Japanese patients with generalized myasthenia gravis: A subgroup analysis of the REGAIN open-label extension study. J Neurol Sci. 2019 Dec 15;407:116419. doi: 10.1016/j.jns.2019.08.004. Epub 2019 Aug 3. |
| 34590717 | Derived | Siddiqi ZA, Nowak RJ, Mozaffar T, O'Brien F, Yountz M, Patti F; REGAIN Study Group. Eculizumab in refractory generalized myasthenia gravis previously treated with rituximab: subgroup analysis of REGAIN and its extension study. Muscle Nerve. 2021 Dec;64(6):662-669. doi: 10.1002/mus.27422. Epub 2021 Oct 14. |
| 33796147 | Derived | Murai H, Suzuki S, Hasebe M, Fukamizu Y, Rodrigues E, Utsugisawa K. Safety and effectiveness of eculizumab in Japanese patients with generalized myasthenia gravis: interim analysis of post-marketing surveillance. Ther Adv Neurol Disord. 2021 Mar 16;14:17562864211001995. doi: 10.1177/17562864211001995. eCollection 2021. |
| 33329303 | Derived | Nowak RJ, Muppidi S, Beydoun SR, O'Brien FL, Yountz M, Howard JF Jr. Concomitant Immunosuppressive Therapy Use in Eculizumab-Treated Adults With Generalized Myasthenia Gravis During the REGAIN Open-Label Extension Study. Front Neurol. 2020 Nov 24;11:556104. doi: 10.3389/fneur.2020.556104. eCollection 2020. |
| 33229455 | Derived | Mantegazza R, Wolfe GI, Muppidi S, Wiendl H, Fujita KP, O'Brien FL, Booth HDE, Howard JF Jr; REGAIN Study Group. Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension. Neurology. 2021 Jan 26;96(4):e610-e618. doi: 10.1212/WNL.0000000000011207. Epub 2020 Nov 23. |
| 32700461 | Derived | Mantegazza R, O'Brien FL, Yountz M, Howard JF Jr; REGAIN study group. Consistent improvement with eculizumab across muscle groups in myasthenia gravis. Ann Clin Transl Neurol. 2020 Aug;7(8):1327-1339. doi: 10.1002/acn3.51121. Epub 2020 Jul 22. |
| 32426038 | Derived | Jacob S, Murai H, Utsugisawa K, Nowak RJ, Wiendl H, Fujita KP, O'Brien F, Howard JF Jr. Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. Ther Adv Neurol Disord. 2020 May 6;13:1756286420911784. doi: 10.1177/1756286420911784. eCollection 2020. |
| 32189108 | Derived | Vissing J, Jacob S, Fujita KP, O'Brien F, Howard JF; REGAIN study group. 'Minimal symptom expression' in patients with acetylcholine receptor antibody-positive refractory generalized myasthenia gravis treated with eculizumab. J Neurol. 2020 Jul;267(7):1991-2001. doi: 10.1007/s00415-020-09770-y. Epub 2020 Mar 18. |
| FG001 | Placebo/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Open-label Maintenance Phase |
|
|
All eligible enrolled participants who received at least 1 dose of study drug in this extension study.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Eculizumab/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 mg) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. |
| BG001 | Placebo/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. |
| 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/Ethnicity, Customized | 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 | Count Of Participants With Treatment-Emergent Adverse Events | Treatment-emergent adverse events (TEAEs) are adverse events with onset on or after the first study drug dose in Study ECU-MG-302. Likewise, treatment-emergent serious adverse events (TESAEs) are serious adverse events that onset on or after the first study drug dose in Study ECU-MG-302. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module. | All participants who received at least 1 dose of eculizumab in this extension study. | Posted | Count of Participants | Participants | Day 1 (after dosing) through End of Study (Week 208) |
|
|
| ||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline In Myasthenia Gravis Activities Of Daily Living Profile (MG-ADL) Total Score At Week 4 And Week 130 | The MG-ADL scale is a validated 8-item patient-reported outcome measure. Participants assessed their functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb impairment (2 items). These 8 items were not weighted and were individually graded from 0 (normal) to 3 (most severe), providing a total MG-ADL score ranging from 0 to 24 points. A reduction in score indicates improvement in condition. Baseline was defined as the last available assessment prior to treatment (first study drug infusion) with eculizumab in Study ECU-MG-302. Change from Baseline in MG-ADL total score at Week 4 (blind induction phase) and at Week 130 (open-label eculizumab phase) are presented. | All participants who received at least 1 dose of eculizumab in this extension study and had an MG-ADL efficacy assessment after study drug infusion at the specified time points. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Week 4 and Week 130 |
|
Day 1 after dosing of study drug through Week 208.
All eligible enrolled participants who received at least 1 dose of study drug.
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 | Eculizumab/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with eculizumab in Study ECU-MG-301 were administered eculizumab (4 vials/1200 mg) on Day 1 and Week 2 and placebo (4 vials/0 mg) at Weeks 1 and 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. | 2 | 56 | 30 | 56 | 54 | 56 |
| EG001 | Placebo/Eculizumab | Blind Induction Phase: Participants who had received blinded treatment with placebo in Study ECU-MG-301 were administered eculizumab/placebo (3 vials/900 mg, plus 1 vial/0 mg, respectively) on Day 1 and Weeks 1 through 3. Open-Label Maintenance Phase: Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. | 1 | 61 | 30 | 61 | 59 | 61 |
| EG002 | Eculizumab (Combined Total) | All participants who received at least 1 dose of eculizumab in the extension study. Participants received open-label eculizumab (4 vials/1200 mg) every 2 weeks starting at Week 4 and continued throughout the study. Eculizumab 1200 mg was administered for up to 4 years in this extension study. | 3 | 117 | 60 | 117 | 113 | 117 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Disseminated intravascular coagulation | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Histiocytosis haematophagic | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Acute myocardial infarction | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Aortic valve incompetence | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Cardiogenic shock | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Myocardial infarction | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Vertigo positional | Ear and labyrinth disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Abdominal incarcerated hernia | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Ascites | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Gastritis | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Intestinal obstruction | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Large intestine polyp | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pancreatitis acute | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Small intestinal obstruction | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Chronic hepatic failure | Hepatobiliary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Hepatic failure | Hepatobiliary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Autoimmune disorder | Immune system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Appendicitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Aspergillus infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Asymptomatic bacteriuria | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Bursitis infective staphylococcal | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Colonic abscess | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Cytomegalovirus infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Device related sepsis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Localised infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Meningitis meningococcal | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Periorbital cellulitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Pseudomonal sepsis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Pseudomonas infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Rectal abscess | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Respiratory syncytial virus infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Wound infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Fibula fracture | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Gastrostomy tube site complication | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Spinal compression fracture | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Haemoglobin decreased | Investigations | MedDRA (21.0) | Systematic Assessment |
| |
| Central obesity | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Obesity | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Arthritis reactive | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Foot deformity | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Fracture nonunion | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Myositis | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Synovitis | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Colon cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment |
| |
| Lymphoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment |
| |
| Malignant melanoma in situ | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment |
| |
| Neuroendocrine carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment |
| |
| Prostate cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment | Adverse event affects male participants only |
|
| Skin papilloma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment |
| |
| Carotid artery stenosis | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Encephalopathy | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Loss of consciousness | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Metabolic encephalopathy | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Myasthenia gravis crisis | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Quadriparesis | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Cervical dysplasia | Reproductive system and breast disorders | MedDRA (21.0) | Systematic Assessment | Adverse event affects female participants only |
|
| Ovarian cyst | Reproductive system and breast disorders | MedDRA (21.0) | Systematic Assessment | Adverse event affects female participants only |
|
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA (21.0) | Systematic Assessment | Adverse event affects female participants only |
|
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pneumonia aspiration | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Arterial occlusive disease | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Deep vein thrombosis | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Extremity necrosis | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Lupus vasculitis | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Lymphopenia | Blood and lymphatic system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Ear pain | Ear and labyrinth disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Vertigo | Ear and labyrinth disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Eye pain | Eye disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Tooth disorder | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Gastroenteritis viral | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA (21.0) | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Infusion related reaction | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA (21.0) | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA (21.0) | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Ovarian cyst | Reproductive system and breast disorders | MedDRA (21.0) | Systematic Assessment | Adverse event affects female participants only |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Productive cough | Respiratory, thoracic and mediastinal disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA (21.0) | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA (21.0) | Systematic Assessment |
|
Sponsor can review results communications at least 60 days prior to public release. Within 30 days of receipt, Sponsor shall advise of any information which is Confidential Information (other than Study Data) or which may impair the availability of patent protection for Inventions. Sponsor can require removal of specifically identified Confidential Information (other than Study Data) and/or delay the communication an additional 60 days to enable Sponsor to seek patent protection for Inventions.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals Inc. | Alexion Pharmaceuticals Inc. | 855-752-2356 | clinicaltrials@alexion.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 6, 2019 | Dec 9, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C481642 | eculizumab |
Not provided
Not provided
Not provided
| Physician Decision |
|
| Death |
|
| Participant felt no change in condition |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Black or African American |
|
| White |
|
| Multiple |
|
| Unknown |
|
| Other |
|
| TESAEs |
|
| TESAEs leading to withdrawal |
|
| Deaths |
|
|
|