BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglo... | NCT05116774

ID	Type	Description	Link
2020-004438-39	EudraCT Number

Label	Type	Description	Intervention Names
BCX9930/BCX9930	Experimental	Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.	Drug: BCX9930
C5-Inhibitor (C5-INH)/BCX9930	Active Comparator	Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.	Drug: Eculizumab Drug: Ravulizumab Drug: BCX9930

Name	Type	Description	Arm Group Labels	Other Names
Eculizumab	Drug	Administered by intravenous infusion per current dose regimen	C5-Inhibitor (C5-INH)/BCX9930

Measure	Description	Time Frame
Part 1: Change From Baseline in Hemoglobin at Week 24		Baseline, Week 24

Measure	Description	Time Frame
Part 1: Number of Participants Who Were Transfusion-free	The number of participants who did not receive any transfusions (packed red blood cells [pRBCs] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.

Name	Affiliation	Role
Austin G Kulasekararaj, MBBS, MD	King's College Hospital NHS Trust	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Investigative Site		Paris			France
Investigative Site

ID	Title	Description
FG000	BCX9930/BCX9930	Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg twice daily (BID) orally and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Aug 1, 2022	Sep 13, 2024

ID	Title	Description
BG000	BCX9930/BCX9930	Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.
BG001	C5-INH/BCX9930	Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG0008 BG0014 BG00212

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

Part 1: Change From Baseline in Hemoglobin at Week 24

Participants in the all subjects as treated (ASaT) population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

grams per deciliter (g/dL)

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG0008 OG0014

Title

Denominators

Categories

Baseline

- ParticipantsOG0008
- ParticipantsOG0014

Title	Measurements
	OG0009.12± 1.096

Secondary

Part 1: Number of Participants Who Were Transfusion-free

The number of participants who did not receive any transfusions (packed red blood cells [pRBCs] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.

Participants in the ASaT population in Part 1 were analyzed.

Posted

Count of Participants

Participants

From Week 4 to Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH

Secondary

Part 1: Number of Units of pRBCs Transfused

Participants in the ASaT population in Part 1 were analyzed.

Posted

Number

units of pRBCs

From Week 4 to Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Secondary

Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score

The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 ("not at all") to 4 ("very much"), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

Scores on a scale

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Secondary

Part 1: Percent Change From Baseline in Lactate Dehydrogenase

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

percent change

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5 INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Secondary

Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused

The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate * 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions.

Participants in the ASaT population in Part 1 were analyzed.

Posted

Number

percent reduction

Prestudy (12 months prior to screening) and from Week 4 to Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Secondary

Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Count of Participants

Participants

Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Secondary

Part 1: Number of Participants Who Achieved Hemoglobin Stabilization

Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24.

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Count of Participants

Participants

From Week 4 to Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants

Secondary

Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs)

Red blood cells opsonized by C3 were to be assessed by flow cytometry .

No data was collected for this endpoint.

Posted

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Secondary

Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size

The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population.

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

% of PNH-RBC within total RBC population

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 377 days.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants

Secondary

Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size

The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs.

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

ratio

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Secondary

Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

10^6 cells/microliter (μL)

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Secondary

Part 1: Number of Participants With ARC in the Normal Range

Number of participants with ARC in the normal range (0.03 - 0.12 10^6 cells/uL) were reported.

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Count of Participants

Participants

Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants

Secondary

Part 1: Change From Baseline in Haptoglobin

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

grams per liter (g/L)

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Secondary

Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range

Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Count of Participants

Participants

Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants

Secondary

Part 1: Change From Baseline in Total Bilirubin

Participants in the ASaT population in Part 1 with available data were analyzed.

Posted

Mean

Standard Deviation

milligram per deciliter (mg/dL)

Baseline, Week 24

ID	Title	Description
OG000	BCX9930	Participants were randomized to receive BCX9930 during Part 1. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks.
OG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH in Part 1 was 24 weeks.

Units	Counts
Participants	OG000

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Dysphagia	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0003 events3 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Nausea	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0003 events3 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0004 events2 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Abdominal pain	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Inguinal hernia	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Vomiting	Gastrointestinal disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Asthenia	General disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0014 events3 affected4 at risk EG0020 events0 affected3 at risk
Fatigue	General disorders	MedDRA v24.0	Systematic Assessment	EG0005 events3 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Chest pain	General disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Face oedema	General disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Pain	General disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Pyrexia	General disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Nasopharyngitis	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0003 events2 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Urinary tract infection	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Bronchitis	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
COVID-19	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0002 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Influenza	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Localised infection	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Respiratory tract infection	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Subcutaneous abscess	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Viral infection	Infections and infestations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA v24.0	Systematic Assessment	EG0006 events3 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Rash macular	Skin and subcutaneous tissue disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Rash maculo-papular	Skin and subcutaneous tissue disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA v24.0	Systematic Assessment	EG0003 events2 affected8 at risk EG0012 events2 affected4 at risk EG0020 events0 affected3 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA v24.0	Systematic Assessment	EG0002 events2 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Axillary mass	Musculoskeletal and connective tissue disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA v24.0	Systematic Assessment	EG0002 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Headache	Nervous system disorders	MedDRA v24.0	Systematic Assessment	EG0004 events4 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Lethargy	Nervous system disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Loss of consciousness	Nervous system disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Nervous system disorder	Nervous system disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Sciatica	Nervous system disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Acute kidney injury	Renal and urinary disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Chromaturia	Renal and urinary disorders	MedDRA v24.0	Systematic Assessment	EG0003 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Haemoglobinuria	Renal and urinary disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Urinary incontinence	Renal and urinary disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Dyspnoea exertional	Respiratory, thoracic and mediastinal disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0012 events2 affected4 at risk EG0020 events0 affected3 at risk
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Haemolysis	Blood and lymphatic system disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Palpitations	Cardiac disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Tachycardia	Cardiac disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Hepatic cytolysis	Hepatobiliary disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk
Jaundice	Hepatobiliary disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Blood lactate dehydrogenase increased	Investigations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Prothrombin time prolonged	Investigations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Erectile dysfunction	Reproductive system and breast disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Scrotal oedema	Reproductive system and breast disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0011 events1 affected4 at risk EG0020 events0 affected3 at risk
Vertigo	Ear and labyrinth disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Vision blurred	Eye disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Hypokalaemia	Metabolism and nutrition disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk

ID	Term
D000743	Anemia, Hemolytic
D000740	Anemia
D006402	Hematologic Diseases
D006425	Hemic and Lymphatic Diseases
D009190	Myelodysplastic Syndromes
D001855	Bone Marrow Diseases

ID	Term
C481642	eculizumab
C000629409	ravulizumab

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	BCX9930/BCX9930	Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days.	8	2	8	7	8
EG001	C5-INH	Participants randomized to this group continued the existing C5-INH therapy during Part 1. The maximum treatment duration on C5-INH was 24 weeks.	4	0	4	3	4
EG002	BCX9930 After C5-INH	Participants who were initially randomized to continue C5-INH therapy received BCX9930 monotherapy after they had completed 24 weeks on C5-INH, or earlier after the date when sponsor decided to halt enrolment in the study permanently and terminate the study. Initially participants were to receive BCX9930 500 mg BID orally. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 was 197 days.	3	1	3	3	3

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Blood creatinine increased	Investigations	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Acute kidney injury	Renal and urinary disorders	MedDRA v24.0	Systematic Assessment	EG0001 events1 affected8 at risk EG0010 events0 affected4 at risk EG0020 events0 affected3 at risk
Rash erythematous	Skin and subcutaneous tissue disorders	MedDRA v24.0	Systematic Assessment	EG0000 events0 affected8 at risk EG0010 events0 affected4 at risk EG0021 events1 affected3 at risk