Follow-up Study for Patients Who Completed Study ALX0681-... | NCT02878603

ID	Type	Description	Link
2016-001503-23	EudraCT Number
ALX0681-C302	Other Identifier	Ablynx

Label	Type	Description	Intervention Names
caplacizumab	Experimental	Participants who completed study ALX0681-C301 (NCT02553317) with standard of care (plasma exchange [PE], corticosteroid and other immunosuppressive agents) or caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 milligrams (mg) intravenous dose followed by a daily 10 mg subcutaneous injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.	Biological: Caplacizumab Other: Standard of Care

Name	Type	Description	Arm Group Labels	Other Names
Caplacizumab	Biological		caplacizumab
Standard of Care

Measure	Description	Time Frame
Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events	aTTP-related events were defined as: aTTP-related death, recurrence of aTTP (defined as recurrent thrombocytopenia requiring initiation of daily PE) or at least one major thromboembolic event (e.g., myocardial infarction, cerebrovascular accident, pulmonary embolism, or deep venous thrombosis). Percentage of participants with at least one aTTP-related events during the study were reported in this outcome measure.	From Baseline up to 36 months
Number of Acquired Thrombotic Thrombocytopenic Purpura-related Events	aTTP-related events were defined as: aTTP-related death, recurrence of aTTP (defined as recurrent thrombocytopenia requiring initiation of daily PE) or at least one major thromboembolic event (e.g., myocardial infarction, cerebrovascular accident, pulmonary embolism or deep venous thrombosis).	From Baseline up to 36 months
Time to First Acquired Thrombotic Thrombocytopenic Purpura-related Events	Time to first aTTP-related events was defined as the duration of time (in days) from Baseline up to first aTTP-related event in LTS16371. Participants without an event during LTS16371 were censored at the end of the study. Kaplan-Meier method was used for the analysis.	From Baseline up to 36 months
Number of Participants With aTTP Related Deaths Reported During the Study		From Baseline up to 36 months
Percentage of Participants With Recurrence of Disease (aTTP)	Recurrence of aTTP was defined as recurrent thrombocytopenia requiring initiation of daily PE.	From Baseline up to 36 months
Number of Disease (aTTP) Recurrence Reported During the Study

Name	Affiliation	Role
Medical Director Ablynx, MD	Ablynx NV	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Investigator site		St Louis	Missouri	63110	United States
Investigator site

PubMed Identifier	Type	Citation	Retractions
36138517	Derived	Scully M, de la Rubia J, Pavenski K, Metjian A, Knobl P, Peyvandi F, Cataland S, Coppo P, Kremer Hovinga JA, Minkue Mi Edou J, De Passos Sousa R, Callewaert F, Gunawardena S, Lin J. Long-term follow-up of patients treated with caplacizumab and safety and efficacy of repeat caplacizumab use: Post-HERCULES study. J Thromb Haemost. 2022 Dec;20(12):2810-2822. doi: 10.1111/jth.15892. Epub 2022 Oct 21.

ID	Title	Description
FG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	May 24, 2016	Oct 21, 2021

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

Percentage of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) Related Events

aTTP-related events were defined as: aTTP-related death, recurrence of aTTP (defined as recurrent thrombocytopenia requiring initiation of daily PE) or at least one major thromboembolic event (e.g., myocardial infarction, cerebrovascular accident, pulmonary embolism, or deep venous thrombosis). Percentage of participants with at least one aTTP-related events during the study were reported in this outcome measure.

Analysis was performed on efficacy intention-to-observe (efficacy ITO) population which included participants who were enrolled in LTS16371 and did not experience an aTTP recurrence in previous study ALX0681-C301 or prior to the beginning of LTS16371.

Posted

Number

percentage of participants

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)	Participants who completed study ALX0681-C301 and received caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to IMP, withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg IV dose followed by a daily 10 mg SC injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Units	Counts
Participants	OG00029 OG00149

Title

Denominators

Categories

Title	Measurements
	OG00037.9 OG0018.2

Primary

Number of Acquired Thrombotic Thrombocytopenic Purpura-related Events

Analysis was performed on efficacy ITO population which included participants who were enrolled in LTS16371 and did not experience an aTTP recurrence in previous study ALX0681-C301 or prior to the beginning of LTS16371.

Posted

Number

aTTP-related events

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)

Primary

Time to First Acquired Thrombotic Thrombocytopenic Purpura-related Events

Time to first aTTP-related events was defined as the duration of time (in days) from Baseline up to first aTTP-related event in LTS16371. Participants without an event during LTS16371 were censored at the end of the study. Kaplan-Meier method was used for the analysis.

Posted

Median

95% Confidence Interval

days

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)

Primary

Number of Participants With aTTP Related Deaths Reported During the Study

Posted

Count of Participants

Participants

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)	Participants who completed study ALX0681-C301 and received caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to IMP, withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg IV dose followed by a daily 10 mg SC injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Percentage of Participants With Recurrence of Disease (aTTP)

Recurrence of aTTP was defined as recurrent thrombocytopenia requiring initiation of daily PE.

Posted

Number

percentage of participants

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)	Participants who completed study ALX0681-C301 and received caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to IMP, withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg IV dose followed by a daily 10 mg SC injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Number of Disease (aTTP) Recurrence Reported During the Study

Recurrence of aTTP was defined as recurrent thrombocytopenia requiring initiation of daily PE.

Posted

Number

aTTP recurrences

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)	Participants who completed study ALX0681-C301 and received caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to IMP, withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg IV dose followed by a daily 10 mg SC injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Time to Recurrence of Disease (aTTP)

Time to first recurrence of disease (aTTP) was defined as the duration of time (in days) from Baseline up to first recurrence of aTTP event in LTS16371. Recurrence of aTTP: defined as recurrent thrombocytopenia requiring initiation of daily PE. Participants without an event during LTS16371 were censored at the end of the study. Kaplan-Meier method was used for the analysis.

Posted

Median

95% Confidence Interval

days

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001

Primary

Percentage of Participants With Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura (TTP)

Major thromboembolic events (e.g., myocardial infarction, cerebrovascular accident, pulmonary embolism, or deep venous thrombosis) were assessed based on Standardized Medical Dictionary for Regulatory Activities (MedDRA) Query (SMQ). Reported major thromboembolic events included TTP recurrences.

Posted

Number

percentage of participants

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001

Primary

Number of Major Thromboembolic Events Including Thrombotic Thrombocytopenic Purpura

Major thromboembolic events (e.g., myocardial infarction, cerebrovascular accident, pulmonary embolism, or deep venous thrombosis) were assessed based on SMQ. Reported major thromboembolic events included TTP recurrences.

Posted

Number

major thromboembolic events

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)

Primary

Time to First Major Thromboembolic Event

Time to first major thromboembolic event was defined as the duration of time (in days) from Baseline up to first major thromboembolic event in LTS16371. Participants without an event during LTS16371 were censored at the end of the study. Kaplan-Meier method was used for the analysis.

Posted

Median

95% Confidence Interval

days

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
OG001	Caplacizumab (Treated in Study C301)

Primary

Cognitive Function: Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) Total Absolute Scores at Baseline, 36 Months Follow-up Visit, and Change From Baseline in RBANS Total Score at 36 Months Follow-up Visit

The RBANS is a 30-minute comprehensive screening test with five individual domains (immediate memory, delayed memory, attention, language, and visuospatial ability) to examine the cognitive mental status of a participant. Scores from all individual domain were aggregated into a total score and thus RBANS total score ranged from 40 to 160, where higher scores reflected better performance.

Analyzed on efficacy ITO population: participants who were enrolled in LTS16371 and did not experience aTTP recurrence in previous study ALX0681-C301 or prior to beginning of LTS16371. Here, 'number analyzed'=participants with available data for each specified category.

Posted

Mean

Standard Deviation

units on a scale

Baseline, 36 Months follow-up visit

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life (HRQoL): Change From Baseline in Headache Impact Test (HIT-6) Total Scores at Month 12, 24, and 36 Follow-up Visits

HIT-6 is an easy to administer assessment that was used as a clinical evaluation of the impact of headache on a participant's QoL in both clinical practice and clinical research. The questionnaire included 6 questions covering the 6 areas of functioning most impacted in headache sufferers including pain, role functioning (the ability to carry out usual activities), social functioning, vitality (energy/ fatigue), cognitive functioning, and psychological/emotional distress. Total HIT-6 scores (sum of all individual questions) ranged from 36 (best outcome) to 78 (worst outcome), where higher scores indicated worse condition.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire (SF-36) Health Survey - Physical Functioning Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Physical functioning domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in physical functioning domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Role Functioning/Physical Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Role Functioning/Physical domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in role functioning/physical domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Role Functioning/Emotional Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Role functioning/emotional domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in role functioning/emotional domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Energy/Fatigue Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Energy/fatigue domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in energy/fatigue domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Emotional Well-being Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Emotional well-being domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in emotional well-being domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Social Functioning Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Social functioning domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in social functioning domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Pain Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Pain domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in pain domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - General Health Domain Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. General Health domain scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in general health domain score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Health-Related Quality of Life: Change From Baseline in 36-Item Short Form Questionnaire Health Survey - Change in Health Status Scores at Month 12, 24, and 36 Follow-up Visits

The SF-36 consisted of 36 items that was summarized into 8 domains: physical functioning, social functioning, role functioning/physical, role functioning/emotional, emotional well-being, energy/fatigue, pain, general health and an additional single item covering change in health status. Change in health status scores ranged from 0 (worst value) to 100 (best value), with higher scores reflecting better health status. Change from baseline in change in health status score at months 12, 24, and 36 were reported in this outcome measure.

Posted

Mean

Standard Deviation

units on a scale

Baseline, Month 12, 24, and 36 Follow-up visits

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Percentage of Participants With Drug-induced Treatment-emergent (TE) Anti-drug Antibodies (ADA) Positive Response

Drug-induced TE ADA positive was based on the outcome of a tiered assay approach that included a modified ADA (mADA) method to eliminate the effects of pre-existing antibodies (pre-Ab). TE ADA responses reported here included both pre-Ab positive and negative responses. A participant was considered as drug-induced TE ADA positive if post-dose samples were positive, regardless of the status of pre-dose samples in the ADA and modified ADA assay.

Analysis was performed on overall ITO population which included participants who were enrolled in LTS16371, grouped by whether they received caplacizumab during previous study ALX0681-C301 versus those who never received caplacizumab before enrollment in LTS16371.

Posted

Number

percentage of participants

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Primary

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE was any untoward medical occurrence in a clinical study participant administered a medicinal product and which did not necessarily had to have a causal relationship with the treatment. An SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event.

Posted

Count of Participants

Participants

From Baseline up to 36 months

ID	Title	Description
OG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Allergic Transfusion Reaction	Injury, poisoning and procedural complications	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Acute Myocardial Infarction	Cardiac disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Angina Unstable	Cardiac disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Pericarditis	Cardiac disorders	MedDRA22.0	Systematic Assessment	EG0003 events1 affected29 at risk EG0010 events0 affected75 at risk
Lymphadenitis	Blood and lymphatic system disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Thrombotic Thrombocytopenic Purpura	Blood and lymphatic system disorders	MedDRA22.0	Systematic Assessment	EG0009 events8 affected29 at risk EG00121 events11 affected75 at risk
Haemorrhage Intracranial	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Ischaemic Stroke	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Migraine	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Seizure	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Gastrointestinal Haemorrhage	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Ileus	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Glomerulonephritis Membranous	Renal and urinary disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Haematuria	Renal and urinary disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Haemorrhage Urinary Tract	Renal and urinary disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Renal Infarct	Renal and urinary disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Back Pain	Musculoskeletal and connective tissue disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected75 at risk
Osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Appendicitis	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Cellulitis	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Hepatitis Viral	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Localised Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Lower Respiratory Tract Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Meningitis Aseptic	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Ophthalmic Herpes Zoster	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Pneumonia	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0013 events2 affected75 at risk
Pneumonia Pneumococcal	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Upper Respiratory Tract Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Urinary Tract Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Abortion Induced	Surgical and medical procedures	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected75 at risk
Cholecystectomy	Surgical and medical procedures	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Breast Cancer	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Invasive Ductal Breast Carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Plasma Cell Myeloma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Renal Cell Carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Transitional Cell Carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk
Abortion Spontaneous	Pregnancy, puerperium and perinatal conditions	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0012 events2 affected75 at risk
Pre-Eclampsia	Pregnancy, puerperium and perinatal conditions	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Psychotic Disorder	Psychiatric disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Colpocele	Reproductive system and breast disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Hysterocele	Reproductive system and breast disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0011 events1 affected75 at risk
Acute Respiratory Failure	Respiratory, thoracic and mediastinal disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0010 events0 affected75 at risk

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Contusion	Injury, poisoning and procedural complications	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0012 events2 affected75 at risk
Adamts13 Activity Decreased	Investigations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG00113 events13 affected75 at risk
Blood Cholesterol Increased	Investigations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0014 events4 affected75 at risk
Dizziness	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG00110 events10 affected75 at risk
Headache	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0009 events9 affected29 at risk EG00117 events16 affected75 at risk
Paraesthesia	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0005 events5 affected29 at risk EG0014 events4 affected75 at risk
Seizure	Nervous system disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected75 at risk
Abdominal Pain	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0013 events3 affected75 at risk
Abdominal Pain Upper	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0014 events4 affected75 at risk
Constipation	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0015 events5 affected75 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0005 events5 affected29 at risk EG0015 events5 affected75 at risk
Nausea	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0017 events7 affected75 at risk
Toothache	Gastrointestinal disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected75 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected75 at risk
Pruritus	Skin and subcutaneous tissue disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0017 events6 affected75 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0015 events5 affected75 at risk
Urticaria	Skin and subcutaneous tissue disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0014 events4 affected75 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0018 events8 affected75 at risk
Back Pain	Musculoskeletal and connective tissue disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0014 events4 affected75 at risk
Hypercholesterolaemia	Metabolism and nutrition disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0012 events2 affected75 at risk
Hypokalaemia	Metabolism and nutrition disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0012 events2 affected75 at risk
Iron Deficiency	Metabolism and nutrition disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0015 events5 affected75 at risk
Bronchitis	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0000 events0 affected29 at risk EG0015 events5 affected75 at risk
Herpes Zoster	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0012 events2 affected75 at risk
Influenza	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0017 events7 affected75 at risk
Lower Respiratory Tract Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0014 events4 affected75 at risk
Nasopharyngitis	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0006 events6 affected29 at risk EG0016 events6 affected75 at risk
Rhinitis	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0012 events2 affected75 at risk
Tonsillitis	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected75 at risk
Tooth Abscess	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected75 at risk
Upper Respiratory Tract Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0017 events7 affected75 at risk
Urinary Tract Infection	Infections and infestations	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0015 events4 affected75 at risk
Hypertension	Vascular disorders	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0011 events1 affected75 at risk
Drug Hypersensitivity	Immune system disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0015 events4 affected75 at risk
Hypersensitivity	Immune system disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0011 events1 affected75 at risk
Asthenia	General disorders	MedDRA22.0	Systematic Assessment	EG0002 events2 affected29 at risk EG0010 events0 affected75 at risk
Fatigue	General disorders	MedDRA22.0	Systematic Assessment	EG0001 events1 affected29 at risk EG0017 events7 affected75 at risk
Pyrexia	General disorders	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0013 events3 affected75 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA22.0	Systematic Assessment	EG0003 events3 affected29 at risk EG0017 events7 affected75 at risk

ID	Term
D011696	Purpura, Thrombocytopenic
D011693	Purpura
D001778	Blood Coagulation Disorders
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D057049	Thrombotic Microangiopathies
D013921	Thrombocytopenia
D001791	Blood Platelet Disorders
D000095542	Cytopenia
D019851	Thrombophilia
D006470	Hemorrhage
D010335	Pathologic Processes
D013568	Pathological Conditions, Signs and Symptoms
D012877	Skin Manifestations
D012816	Signs and Symptoms

ID	Term
D019984	Quality Indicators, Health Care
D011787	Quality of Health Care
D006298	Health Services Administration
D017530	Health Care Quality, Access, and Evaluation

ID	Title	Description
BG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
BG001	Caplacizumab (Treated in Study C301)	Participants who completed study ALX0681-C301 and received caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to IMP, withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg IV dose followed by a daily 10 mg SC injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.
BG002	Total Title

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Standard of Care (SoC) (Treated in Study C301)	Participants who completed study ALX0681-C301 with SoC (plasma exchange [PE], corticosteroid and other immunosuppressive agents) treatment were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to investigational medicinal product [IMP], withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg intravenous (IV) dose followed by a daily 10 milligrams (mg) subcutaneous (SC) injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.	1	29	16	29	22	29
EG001	Caplacizumab (Treated in Study C301)	Participants who completed study ALX0681-C301 and received caplacizumab with PE and immunosuppressive agents were enrolled in study LTS16371. Participants upon each recurrence of aTTP in LTS16371 and not meeting any criteria (namely: pregnancy, history of severe and/or serious hypersensitivity reaction to IMP, withdrawal before receiving IMP, received more than 1 PE) were treated with caplacizumab initial 10 mg IV dose followed by a daily 10 mg SC injections during the period of PE and for 30 days after stop of PE (and eventually 28-day extension period, if needed). Participants with or without recurrence were followed up twice yearly up to maximum of 36 months in LTS16371.	0	75	28	75	58	75