Study to Evaluate the Safety, Tolerability of BCX9930 in Participants With Either Complement 3 Glomerulopathy (C3G), Immunoglobulin A Nephropathy (IgAN), or Primary Membranous Nephropathy (PMN)

Phase 2

Terminated

Conditions: Complement 3 Glomerulopathy
Immunoglobulin A Nephropathy
Membranous Nephropathy

Interventions: Drug: BCX9930

Registration Number: NCT05162066

Lead Sponsor: BioCryst Pharmaceuticals

Brief Summary: The objective of this study was to determine the safety and therapeutic potential of BCX9930 in participants with C3G, IgAN, or PMN.

Detailed Description: Not available

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 2

Inclusion Criteria

Body weight ≥ 40 kilograms (kg)
Primary diagnosis of C3G, IgAN, or PMN confirmed by central pathology review
An estimated glomerular filtration rate (eGFR) ≥ 50 milliter per minute per 1.73 meter square (mL/min/1.73 m^2) (or ≥ 30 mL/min/1.73 m^2 after Data Monitoring Committee [DMC] recommendation)
Receiving treatment with a stable, maximum recommended or maximum tolerated dose of an angiotensin-converting enzyme inhibitor (ACEi) or angiotensin receptor blocker (ARB) for at least 60 days prior to the Day 1 Visit
Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series

Exclusion Criteria

Known congenital deficiency of C1s, C1r, C1q, C2, or C4
History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation
Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition
History of malignancy within 5 years prior to the screening visit
Active serious bacterial, viral, or fungal infection or any other serious infection within 14 days of screening
Treatment with any systemic immunosuppressive or immunomodulatory therapy within 90 days OR anti-CD20 antibody therapies (eg, rituximab) within 180 days prior to the screening visit
Treatment with renin inhibitors (eg, aliskiren) or sodium-glucose-cotransporter 2 (SGLT2) inhibitors within 60 days prior to Day 1

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
IgAN cohort	BCX9930	Approximately 14 eligible participants with IgAN will be enrolled.
C3G cohort	BCX9930	Approximately 14 eligible participants with C3G will be enrolled.
PMN cohort	BCX9930	Approximately 14 eligible participants with PMN will be enrolled.

Primary Outcome Measures

Name	Time	Method
Percent Change From Baseline in 24-hour uPCR at Week 12	Baseline, 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.
Percent Change From Baseline in 24-hour uPCR at Week 24	Baseline, 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.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in Estimated Glomerular Filtration Rate	Baseline, Weeks 12, 24	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).
Number of Participants With a Treatment-emergent Adverse Event (TEAE)	From first dose up to safety follow-up period (Week 28)	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.
Percent Change From Baseline in 24-hour Urinary Protein Excretion	Baseline, Weeks 12, 24,
Number of Participants Who Discontinued Due to a TEAE	From first dose up to safety follow-up period (Week 28)	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.
Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality	From first dose up to safety follow-up period (Week 28)	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.
Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)	From first dose up to safety follow-up period (Week 28)	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: * Death * Is life-threatening (participant was at immediate risk of death at the time of the event; it does not refer to an event that hypothetically might have caused death if it were more severe) * Requires participant hospitalization (formal admission to a hospital for medical reasons) or prolongation of existing hospitalization * Results in persistent or significant disability/incapacity (ie, there was a substantial disruption of a person's ability to carry out normal life functions) * Is a congenital anomaly/birth defect
Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE	From first dose up to safety follow-up period (Week 28)	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