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| ID | Type | Description | Link |
|---|---|---|---|
| 2020-005855-19 | EudraCT Number |
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Sponsor Decision
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The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| C3G cohort | Experimental | Approximately 14 eligible participants with C3G will be enrolled. |
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| IgAN cohort | Experimental | Approximately 14 eligible participants with IgAN will be enrolled. |
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| PMN cohort | Experimental | Approximately 14 eligible participants with PMN will be enrolled. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BCX9930 | Drug | Administered orally at a dose of 200 mg twice daily for the first 2 weeks, then 400 mg twice daily |
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| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in 24-hour uPCR at Week 12 | Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease. | Baseline, Week 12 |
| Percent Change From Baseline in 24-hour uPCR at Week 24 | Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease. | Baseline, Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Percent Change From Baseline in 24-hour Urinary Protein Excretion | Baseline, Weeks 12, 24, | |
| Change From Baseline in Estimated Glomerular Filtration Rate | eGFR was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for participants ≥ 18 years old and by the bedside Schwartz formula for participants ≤ 18 years old. eGFR was reported in milliliter per minute per 1.73 per square meter (mL/min/1.73m^2). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Carla M Nester, MD, MSA, FASN | University of Iowa Stead Family Children's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site | Poitiers | France | ||||
| Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39899371 | Derived | Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available. |
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At the time the study was discontinued, only participants with historical C3G were enrolled.
Participants with either complement 3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN), or primary membranous nephropathy (PMN) were planned to be enrolled in the study.
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| ID | Title | Description |
|---|---|---|
| FG000 | BCX9930 - C3G Cohort | Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26). |
| FG001 | BCX9930 - IgAN Cohort | Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 4, 2022 | Mar 7, 2024 |
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Three parallel treatment cohorts based on diagnosis of C3G, IgAN, or PMN. All eligible participants will receive open-label BCX9930.
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| Baseline, Weeks 12, 24 |
| Number of Participants With a Treatment-emergent Adverse Event (TEAE) | An Adverse event (AE) was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug. | From first dose up to safety follow-up period (Week 28) |
| Number of Participants Who Discontinued Due to a TEAE | An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug. | From first dose up to safety follow-up period (Week 28) |
| Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE) | An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug. A serious adverse event (SAE) was an adverse event/reaction that results in any of the following outcomes:
| From first dose up to safety follow-up period (Week 28) |
| Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE | An AE was any untoward medical occurrence has no causal relationship with the study drug or with the clinical study itself. It was an unfavorable & unintended sign, symptom, syndrome, or illness that developed or worsened during clinical study. TEAEs: AEs that started on or after first dose of treatment through 30 days after the last dose of study drug. All AEs were graded using the CTCAE Grades 1 through 5. Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL) Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL Grade 4: Life-threatening consequences; urgent intervention indicated Grade 5: Death Participants with Grades 3 or 4 AEs were reported | From first dose up to safety follow-up period (Week 28) |
| Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality | Treatment-emergent Laboratory Abnormality were defined as an event that started on or after the first dose of study treatment through 30 days after the last dose of study drug. CTCAE Grades for laboratory abnormalities include: Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL. Grade 4: Life-threatening consequences; urgent intervention indicated. Grade 5: Death Participants with Grades 3 or 4 laboratory abnormality were reported. | From first dose up to safety follow-up period (Week 28) |
| Toulouse |
| France |
| Investigative Site | Bari | Italy |
| Investigative Site | Bergamo | Italy |
| Investigative Site | Brescia | Italy |
| Investigative Site | Turin | Italy |
| Investigative Site #1 | Barcelona | Spain |
| Investigative Site #2 | Barcelona | Spain |
| Investigative Site #1 | Madrid | Spain |
| Investigative Site #2 | Madrid | Spain |
| Investigative Site | Oxford | United Kingdom |
| FG002 | BCX9930 - PMN Cohort | Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26). |
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| NOT COMPLETED |
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Safety population included all participants who received at least one dose of the study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | BCX9930 - C3G Cohort | Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26). |
| BG001 | BCX9930 - IgAN Cohort | Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26). |
| BG002 | BCX9930 - PMN Cohort | Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26). |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| 24-hour Urine Protein-to-creatinine Ratio (uPCR) | Urinary protein/creatinine ratio (milligram per millimole [mg/mmol]) =urine protein concentration (mg/deciliter [dL])/ urine creatinine concentration (mmol/dL). | Mean | Standard Deviation | milligram per millimole |
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| 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 | Percent Change From Baseline in 24-hour uPCR at Week 12 | Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease. | Safety population with available data was analyzed | Posted | Mean | Standard Deviation | percent change | Baseline, Week 12 |
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| Primary | Percent Change From Baseline in 24-hour uPCR at Week 24 | Urinary protein/creatinine ratio (mg/mmol) =urine protein concentration (mg/dL)/ urine creatinine concentration (mmol/dL). Decrease of urinary protein/creatinine ratio means improvement of renal disease. | Safety population with available data was analyzed | Posted | Mean | Standard Deviation | percent change | Baseline, Week 24 |
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| Secondary | Percent Change From Baseline in 24-hour Urinary Protein Excretion | Safety population with available data was analyzed | Posted | Mean | Standard Deviation | percent change | Baseline, Weeks 12, 24, |
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| Secondary | Change From Baseline in Estimated Glomerular Filtration Rate | eGFR was calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula for participants ≥ 18 years old and by the bedside Schwartz formula for participants ≤ 18 years old. eGFR was reported in milliliter per minute per 1.73 per square meter (mL/min/1.73m^2). | Safety population with available data was analyzed | Posted | Mean | Standard Deviation | mL/min/1.73m^2 | Baseline, Weeks 12, 24 |
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| Secondary | Number of Participants With a Treatment-emergent Adverse Event (TEAE) | An Adverse event (AE) was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug. | Safety population | Posted | Count of Participants | Participants | From first dose up to safety follow-up period (Week 28) |
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| Secondary | Number of Participants Who Discontinued Due to a TEAE | An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug. | Safety population | Posted | Count of Participants | Participants | From first dose up to safety follow-up period (Week 28) |
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| Secondary | Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE) | An AE was any untoward medical occurrence in a clinical study participant. TEAEs were defined, within a dosing group, as AEs that started on or after the first dose of study treatment through 30 days after the last dose of study drug. A serious adverse event (SAE) was an adverse event/reaction that results in any of the following outcomes:
| Safety population | Posted | Count of Participants | Participants | From first dose up to safety follow-up period (Week 28) |
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| Secondary | Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE | An AE was any untoward medical occurrence has no causal relationship with the study drug or with the clinical study itself. It was an unfavorable & unintended sign, symptom, syndrome, or illness that developed or worsened during clinical study. TEAEs: AEs that started on or after first dose of treatment through 30 days after the last dose of study drug. All AEs were graded using the CTCAE Grades 1 through 5. Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL) Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL Grade 4: Life-threatening consequences; urgent intervention indicated Grade 5: Death Participants with Grades 3 or 4 AEs were reported | Safety population | Posted | Count of Participants | Participants | From first dose up to safety follow-up period (Week 28) |
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| Secondary | Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality | Treatment-emergent Laboratory Abnormality were defined as an event that started on or after the first dose of study treatment through 30 days after the last dose of study drug. CTCAE Grades for laboratory abnormalities include: Grade 1: Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2: Moderate; minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental ADL. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL. Grade 4: Life-threatening consequences; urgent intervention indicated. Grade 5: Death Participants with Grades 3 or 4 laboratory abnormality were reported. | Safety population | Posted | Count of Participants | Participants | From first dose up to safety follow-up period (Week 28) |
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From first dose up to safety follow-up period (Week 28)
Safety population
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
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| EG000 | BCX9930 | Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26). | 0 | 2 | 0 | 2 | 2 | 2 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eyelid oedema | Eye disorders | MedDRA v24.0 | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA v24.0 | Systematic Assessment |
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| Hypertransaminasaemia | Hepatobiliary disorders | MedDRA v24.0 | Systematic Assessment |
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| Asymptomatic bacteriuria | Infections and infestations | MedDRA v24.0 | Systematic Assessment |
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| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA v24.0 | Systematic Assessment |
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| Hypotension | Vascular disorders | MedDRA v24.0 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA v24.0 | Systematic Assessment |
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The sponsor decided to prematurely terminate the study due to changes in the competitive landscape. Due to low enrolment in the study (N=2) and early termination of both participants, very limited data were available for analyses. Therefore, only key efficacy endpoints (24-hour uPCR, 24-hour urine protein excretion, and eGFR) and safety were analyzed and reported.
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| Title | Organization | Phone | Extension | |
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| Study Director | BioCryst Pharmaceuticals Inc | +1 919-859-1302 | clinicaltrials@biocryst.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Feb 14, 2023 | Feb 26, 2024 | SAP_001.pdf |
| ID | Term |
|---|---|
| D015432 | Glomerulonephritis, Membranoproliferative |
| D005922 | Glomerulonephritis, IGA |
| D015433 | Glomerulonephritis, Membranous |
| ID | Term |
|---|---|
| D005921 | Glomerulonephritis |
| D009393 | Nephritis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D007154 | Immune System Diseases |
| D001327 | Autoimmune Diseases |
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| Unknown or Not Reported |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Percent Change at Week 12 |
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| Percent Change at Week 24 |
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