Trial Outcomes & Findings for 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) (NCT NCT05162066)
NCT ID: NCT05162066
Last Updated: 2024-05-02
Results Overview
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.
TERMINATED
PHASE2
2 participants
Baseline, Week 12
2024-05-02
Participant Flow
Participants with either complement 3 glomerulopathy (C3G), immunoglobulin A nephropathy (IgAN), or primary membranous nephropathy (PMN) were planned to be enrolled in the study.
At the time the study was discontinued, only participants with historical C3G were enrolled.
Participant milestones
| Measure |
BCX9930 - C3G Cohort
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
BCX9930 - IgAN Cohort
Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).
|
BCX9930 - PMN Cohort
Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|---|---|
|
Overall Study
STARTED
|
2
|
0
|
0
|
|
Overall Study
COMPLETED
|
0
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
0
|
0
|
Reasons for withdrawal
| Measure |
BCX9930 - C3G Cohort
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
BCX9930 - IgAN Cohort
Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).
|
BCX9930 - PMN Cohort
Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
0
|
|
Overall Study
Perceived lack of efficacy
|
1
|
0
|
0
|
Baseline Characteristics
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)
Baseline characteristics by cohort
| Measure |
BCX9930 - C3G Cohort
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
BCX9930 - IgAN Cohort
Participants with complement IgAN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).
|
BCX9930 - PMN Cohort
Participants with complement PMN were to receive BCX9930 tablet orally for up to Day 186 (Week 26).
|
Total
n=2 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
35.5 years
STANDARD_DEVIATION 16.26 • n=93 Participants
|
—
|
—
|
35.5 years
STANDARD_DEVIATION 16.26 • n=483 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=93 Participants
|
—
|
—
|
1 Participants
n=483 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=93 Participants
|
—
|
—
|
1 Participants
n=483 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=93 Participants
|
—
|
—
|
2 Participants
n=483 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=93 Participants
|
—
|
—
|
2 Participants
n=483 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
—
|
—
|
0 Participants
n=483 Participants
|
|
24-hour Urine Protein-to-creatinine Ratio (uPCR)
|
380.5 milligram per millimole
STANDARD_DEVIATION 137.89 • n=93 Participants
|
—
|
—
|
380.5 milligram per millimole
STANDARD_DEVIATION 137.89 • n=483 Participants
|
PRIMARY outcome
Timeframe: Baseline, Week 12Population: Safety population with available data was analyzed
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.
Outcome measures
| Measure |
BCX9930
n=1 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Percent Change From Baseline in 24-hour uPCR at Week 12
|
-33.2 percent change
Standard Deviation NA
Standard deviation was not calculated as only one participant was analyzed
|
PRIMARY outcome
Timeframe: Baseline, Week 24Population: Safety population with available data was analyzed
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.
Outcome measures
| Measure |
BCX9930
n=1 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Percent Change From Baseline in 24-hour uPCR at Week 24
|
-8.8 percent change
Standard Deviation NA
Standard deviation was not calculated as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24,Population: Safety population with available data was analyzed
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Percent Change From Baseline in 24-hour Urinary Protein Excretion
Percent Change at Week 12
|
-40.9 percent change
Standard Deviation NA
Standard deviation was not calculated as only one participant was analyzed
|
|
Percent Change From Baseline in 24-hour Urinary Protein Excretion
Percent Change at Week 24
|
-25.8 percent change
Standard Deviation NA
Standard deviation was not calculated as only one participant was analyzed
|
SECONDARY outcome
Timeframe: Baseline, Weeks 12, 24Population: Safety population with available data was analyzed
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).
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Change From Baseline in Estimated Glomerular Filtration Rate
Change at Week 12
|
-6.0 mL/min/1.73m^2
Standard Deviation NA
Standard deviation was not calculated as only one participant was analyzed
|
|
Change From Baseline in Estimated Glomerular Filtration Rate
Change at Week 24
|
-5.0 mL/min/1.73m^2
Standard Deviation NA
Standard deviation was not calculated as only one participant was analyzed
|
SECONDARY outcome
Timeframe: From first dose up to safety follow-up period (Week 28)Population: Safety population
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.
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Number of Participants With a Treatment-emergent Adverse Event (TEAE)
|
2 Participants
|
SECONDARY outcome
Timeframe: From first dose up to safety follow-up period (Week 28)Population: Safety population
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.
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Number of Participants Who Discontinued Due to a TEAE
|
1 Participants
|
SECONDARY outcome
Timeframe: From first dose up to safety follow-up period (Week 28)Population: Safety population
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
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Number of Participants Who Experienced a Treatment-emergent Serious Adverse Event (TESAE)
|
0 Participants
|
SECONDARY outcome
Timeframe: From first dose up to safety follow-up period (Week 28)Population: Safety population
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
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Number of Participants Who Experienced a Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 or 4 TEAE
|
1 Participants
|
SECONDARY outcome
Timeframe: From first dose up to safety follow-up period (Week 28)Population: Safety population
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.
Outcome measures
| Measure |
BCX9930
n=2 Participants
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Number of Participants Who Experienced a CTCAE Treatment-emergent Grade 3 or 4 Laboratory Abnormality
|
2 Participants
|
Adverse Events
BCX9930
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
BCX9930
n=2 participants at risk
Participants with complement C3G received BCX9930 tablet orally for up to Day 186 (Week 26).
|
|---|---|
|
Eye disorders
Eyelid oedema
|
50.0%
1/2 • Number of events 2 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
1/2 • Number of events 2 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
1/2 • Number of events 2 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Hepatobiliary disorders
Hypertransaminasaemia
|
50.0%
1/2 • Number of events 5 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Infections and infestations
Asymptomatic bacteriuria
|
50.0%
1/2 • Number of events 1 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
50.0%
1/2 • Number of events 1 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
50.0%
1/2 • Number of events 1 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Skin and subcutaneous tissue disorders
Rash
|
50.0%
1/2 • Number of events 1 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
Vascular disorders
Hypotension
|
50.0%
1/2 • Number of events 1 • From first dose up to safety follow-up period (Week 28)
Safety population
|
|
General disorders
Oedema peripheral
|
50.0%
1/2 • Number of events 2 • From first dose up to safety follow-up period (Week 28)
Safety population
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place