Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2020-004403-14 | EudraCT Number |
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Sponsor decision
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The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.
This was a randomized, placebo-controlled, double-blind, parallel-group, 2-part study. Parts 1 and 2 was to be conducted in the same participants.
Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of treatment with oral BCX9930 monotherapy for 12 weeks versus placebo in participants with PNH who were not currently receiving treatment with complement inhibitor therapy. Participants were randomized to receive BCX9930 or placebo under double blind conditions for the 12-week randomized treatment period. The primary efficacy and safety analyses were based on Part 1.
Part 2 of the study was designed to evaluate the long-term safety, tolerability, and effectiveness of open-label BCX9930 monotherapy when administered through Week 52. All participants in Part 2 received BCX9930. Participants who were randomized to BCX9930 monotherapy in Part 1 continued to receive BCX9930 in Part 2. Participants who were randomized to placebo in Part 1 discontinued that therapy at the Week 12 visit and received BCX9930 in Part 2.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BCX9930/BCX9930 | Experimental | Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg twice daily (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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days. |
|
| Placebo/BCX9930 | Placebo Comparator | Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Placebo | Drug | Administered orally twice daily |
| |
| BCX9930 monotherapy |
| Measure | Description | Time Frame |
|---|---|---|
| Part 1: Change From Baseline in Hemoglobin at Week 12 | Baseline, Week 12 |
| 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 12, 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| David J Kuter, MD, DPhil | Massachusetts General Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site | Ampang | Malaysia | ||||
| Investigative Site |
Not provided
Not provided
Not provided
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A total 12 participants were randomized and treated.
The study was conducted in Malaysia, South Africa, and Korea.
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| ID | Title | Description |
|---|---|---|
| FG000 | BCX9930/BCX9930 | Participants received BCX9930 monotherapy in double blind (DB) manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg twice daily (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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Part 1:DB Phase (up to 12 Weeks) |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 1, 2022 | Sep 13, 2024 |
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| Drug |
Administered orally twice daily |
|
| From Week 4 to Week 12 |
| Part 1: Number of Units of pRBCs Transfused | From Week 4 to Week 12 |
| Part 1: Percent Change From Baseline in Lactate Dehydrogenase | Baseline, Week 12 |
| 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. | Baseline, Week 12 |
| Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | The rate of pRBC units transfused from Week 4 to Week 12 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. | Prestudy (12 months prior to screening) and from Week 4 to Week 12 |
| Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL | At Week 12 |
| Part 1: Number of Participants Achieving 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 12. | From Week 4 to Week 12 |
| Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (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. | Baseline, Week 12 |
| 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 (i.e, 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. | Baseline, Week 12 |
| Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Baseline, Week 12 |
| Part 1: Number of Participants With ARC in the Normal Range | Number of participants with ARC in the normal range (50 - 100 x 10^9 cells/L) were reported. | Week 12 |
| Part 1: Change From Baseline in Haptoglobin | Baseline, Week 12 |
| 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. | Week 12 |
| Part 1: Change From Baseline in Total Bilirubin | Baseline, Week 12 |
| Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Baseline, Week 12 |
| Bloemfontein |
| South Africa |
| Investigative Site | Cape Town | South Africa |
| Investigative Site | Pretoria | South Africa |
| InvestigativeSite | Daejeon | South Korea |
| FG001 | Placebo/BCX9930 | Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
| Part 2:Open-label Phase (up to 52 Weeks) |
|
|
The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930. Therefore, BCX9930 group included participants who received BCX9930 throughout the study and placebo group included data for participants who received placebo initially and then switched to BCX9930.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BCX9930/BCX9930 | Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. 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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days. |
| BG001 | Placebo/BCX9930 | Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum treatment duration in Part 1 was 12 weeks. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Part 1: Change From Baseline in Hemoglobin at Week 12 | Participants in the All Subjects as Treated (ASaT) population (all participants who received at least 1 dose of study drug and had a post baseline laboratory assessment) in Part 1 were analyzed. | Posted | Mean | Standard Deviation | grams per deciliter (g/dL) | Baseline, Week 12 |
|
|
| ||||||||||||||||||||||||||||||||||||
| 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 12, 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 12 |
| ||||||||||||||||||||||||||||||||||||||
| 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 12 |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Percent Change From Baseline in Lactate Dehydrogenase | Participants in the ASaT population in Part 1 were analyzed. | Posted | Mean | Standard Deviation | percent change | Baseline, Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| 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 12 |
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | The rate of pRBC units transfused from Week 4 to Week 12 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 12 |
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL | Participants in the ASaT population in Part 1 were analyzed. | Posted | Count of Participants | Participants | At Week 12 |
|
| ||||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Number of Participants Achieving 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 12. | Participants in the ASaT population in Part 1 with available data were analyzed. | Posted | Count of Participants | Participants | From Week 4 to Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (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 Part 1 were analyzed. | Posted | Mean | Standard Deviation | % of PNH-RBC within total RBC population | Baseline, Week 12 |
|
| ||||||||||||||||||||||||||||||||||||
| 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 (i.e, 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 12 |
| |||||||||||||||||||||||||||||||||||||
| 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 | 10^9 cells/microliter (μL) | Baseline, Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Number of Participants With ARC in the Normal Range | Number of participants with ARC in the normal range (50 - 100 x 10^9 cells/L) were reported. | Participants in the ASaT population in Part 1 with available data were analyzed. | Posted | Count of Participants | Participants | Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Change From Baseline in Haptoglobin | Participants in the ASaT population in Part 1 were analyzed. | Posted | Mean | Standard Deviation | grams per liter (g/L) | Baseline, Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| 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 were analyzed. | Posted | Count of Participants | Participants | Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Change From Baseline in Total Bilirubin | Participants in the ASaT population in Part 1 were analyzed. | Posted | Mean | Standard Deviation | milligram per deciliter (mg/dL) | Baseline, Week 12 |
|
| |||||||||||||||||||||||||||||||||||||
| Secondary | Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Participants in the ASaT population in Part 1 were analyzed. | Posted | Mean | Standard Deviation | Units per liter (U/L) | Baseline, Week 12 |
|
|
From Day 1 up to 408 days
The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | BCX9930/BCX9930 | Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. 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 was 378 days. | 0 | 10 | 5 | 10 | 10 | 10 |
| EG001 | Placebo | Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). | 0 | 2 | 1 | 2 | 2 | 2 |
| EG002 | BCX9930 After Placebo | Participants who were initially randomized to placebo group received BCX9930 monotherapy in open label manner, if they had completed Week 12 on placebo, or earlier after the 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 was 281 days. | 0 | 1 | 1 | 1 | 1 | 1 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Enterovirus infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia fungal | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Tuberculosis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Breakthrough haemolysis | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Budd-Chiari syndrome | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Renal failure | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dysphagia | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Varices oesophageal | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Abdominal tenderness | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Gastric varices | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Alopecia | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Rash papular | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Pulmonary tuberculosis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Tonsillitis | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Malaise | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Swelling face | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Vaccination site pruritus | General disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Arthritis reactive | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Flank pain | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Joint stiffness | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Periarthritis | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Breakthrough haemolysis | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haemolysis | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Neutropenia | Blood and lymphatic system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Blood uric acid increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Serum ferritin decreased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Weight increased | Investigations | MedDRA v24.0 | Systematic Assessment |
| |
| Acute kidney injury | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Haemoglobinuria | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Nephropathy | Renal and urinary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Breast pain | Reproductive system and breast disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Heavy menstrual bleeding | Reproductive system and breast disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Menometrorrhagia | Reproductive system and breast disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Jaundice | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA v24.0 | Systematic Assessment |
| |
| Subcutaneous haematoma | Injury, poisoning and procedural complications | MedDRA v24.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v24.0 | Systematic Assessment |
|
The sponsor decided to terminate the development program (including this study).
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | BioCryst Pharmaceuticals Inc | +1 919-859-1302 | clinicaltrials@biocryst.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 4, 2023 | Sep 13, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D009190 | Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
|
| More than one race |
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| Unknown or Not Reported |
|
Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). |
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|---|---|
| Participants |
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| Units |
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| Counts |
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| Participants |
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