Primary IgA Nephropathy
Conditions
Keywords
IgA Nephropathy
Brief summary
The overall aim of the study is to evaluate the efficacy, safety, and tolerability of Nefecon 16 mg per day in the treatment of patients with primary IgAN (Immunoglobulin A nephropathy) at risk of progressing to end-stage renal disease (ESRD), despite maximum tolerated treatment with renin-angiotensin system (RAS) blockade using angiotensin converting enzyme inhibitors (ACEIs) or angiotensin II type I receptor blockers (ARBs).
Detailed description
This is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy, safety, and tolerability of oral Nefecon compared to matching placebo in patients with primary IgAN on a background of optimized RAS inhibitor therapy. The study will consist of 2 parts, Part A and Part B. Part A will include a 9 month blinded Treatment Period, and a 3-month Follow up Period. Part B of the study will consist of a 12-month observational Follow up Period; no study drug will be administered during Part B. Part A and B will be blinded. Safety will be monitored by an independent Data Safety Monitoring Board.
Interventions
DRUGNefecon
Nefecon 16 mg for daily administration by mouth for 9 months.
DRUGPlacebo oral capsule
Placebo capsules for daily administration by mouth for 9 months.
Sponsors
Calliditas Therapeutics AB
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Female or male patients ≥18 years 2. Biopsy-verified IgA nephropathy 3. Stable dose of RAS inhibitor therapy (ACEIs and/or ARBs) at the maximum allowed dose or Maximum Tolerated Dose (MTD) according to the 2012 KDIGO (Kidney Disease: Improving Global Outcomes) guidelines 4. Urine protein creatinine ratio ≥1 g/24hr 5. eGFR ≥35 mL/min per 1.73 m2 and ≤90 mL/min per 1.73 m2 using the Chronic Kidney Diseae Epidemiology Collaboration (CKD-EPI) formula 6. Willing and able to give informed consent
Exclusion criteria
1. Systemic diseases that may cause mesangial IgA deposition. 2. Patients who have undergone a kidney transplant. 3. Patients with acute or chronic infectious disease including hepatitis, tuberculosis, human immunodeficiency virus (HIV), and chronic urinary tract infections. 4. Patients with liver cirrhosis, as assessed by the Investigator. 5. Patients with a diagnosis of type 1 or type 2 diabetes mellitus which is poorly controlled. 6. Patients with history of unstable angina, class III or IV congestive heart failure, and/or clinically significant arrhythmia, as judged by the Investigator; 7. Patients with unacceptable blood pressure control defined as a blood pressure consistently above national guidelines for proteinuric renal disease, as assessed by the Investigator 8. Patients with diagnosed malignancy within the past 5 years.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Ratio of Urine Protein to Creatinine Ratio (UPCR) at 9 Months Compared to Baseline | 9 months | Part A primary endpoint: The ratio of Urine Protein to Creatinine Ratio (UPCR) (based on 24-hour urine collections) at 9 months following the first dose of study drug compared to baseline. Ratio being: UPCR at 9 months in g/gram divided with UPCR at Baseline in g/gram |
| Part B: Time-weighted Average of Estimated Glomerular Filtration Rate (eGFR) | Up to 2 years and 1 month | Part B Primary endpoint: Time-weighted average of estimated glomerular filtration rate (eGFR) recordings observed at each time point over 2 years. The eGFR (CKD-EPI) at 2 years (which must have been repeated to provide a second value obtained within 14 to 35 days) was the geometric mean of the 2 assessments. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part A: Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months | 9 months | Part A: Ratio of urine albumin to creatinine ratio (UACR) at 9 months compared to baseline. |
| Part B: Time to 30% Reduction in eGFR | Over 2 years | Part B: Time to 30% reduction from baseline in eGFR (CKD-EPI) confirmed by a second value. For clarity: Please note that the number of patients with a 30% reduction is presented with statistical analysis of the time to 30% reduction. |
| Part B: Time to Receiving Rescue Medication. | Over 2 years | Part B: Time from the first dose of study drug until receiving rescue medication. For clarity: Please note that the number of patients receiving rescue medication is presented with statistical analysis of the time to receiving rescue medication. |
| Part B: Ratio of UPCR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 12, 18 and 24 months | Part B: Ratio of UPCR, UACR, and eGFR (CKD-EPI) compared to baseline averaged over time points between 12 and 24 months, inclusive, following the first dose of study drug |
| Part A: Ratio of eGFR at 9 Months | 9 months | Part A: Ratio of eGFR at 9 months compared to baseline calculated using the CKD-EPI formula. |
| Part B: Ratio of eGFR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 12 to 24 months | Part B: Ratio of UPCR, UACR, and eGFR (CKD-EPI) compared to baseline averaged over time points between 12 and 24 months, inclusive, following the first dose of study drug; |
| Part B: Proportion of Patients Without Microhematuria | 12 to 24 months | Part B: Proportion of patients without microhematuria in at least 2 of the following time points: 12, 18, and 24 months following the first dose of study drug |
| Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | 9 months | Part B: Short Form 36 (SF-36) quality of life assessment at 9 and 24 months. The 36-Item Short Form Health Survey (SF-36) is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting and have been widely used. It consists of eight health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. Higher scores indicate better health. Scores represent the percentage of total possible score achieved. |
| Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | 24 months | Part B: Short Form 36 (SF-36) quality of life assessment at 9 and 24 months. The 36-Item Short Form Health Survey (SF-36) is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting and have been widely used. It consists of eight health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. Higher scores indicate better health. Scores represent the percentage of total possible score achieved. |
| Part B: Ratio of UACR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 12 to 24 months | Part B: Ratio of UPCR, UACR, and eGFR (CKD-EPI) compared to baseline averaged over time points between 12 and 24 months, inclusive, following the first dose of study drug. |
| Part A: Ratio of eGFR at 12 Months | 12 months | Part A: Ratio of eGFR at 12 months compared to baseline calculated using the CKD-EPI formula. |
Countries
Argentina, Australia, Belarus, Belgium, Canada, Czechia, Finland, France, Germany, Greece, Italy, Poland, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Nefecon Nefecon 16 mg once daily by mouth for 9 months.
Nefecon: Nefecon 16 mg for daily administration by mouth for 9 months. | 182 |
| Placebo Oral Capsule Placebo oral capsule once daily by mouth for 9 months.
Placebo oral capsule: Placebo capsules for daily administration by mouth for 9 months. | 182 |
| Total | 364 |
Baseline characteristics
| Characteristic | Nefecon | Placebo Oral Capsule | Total |
|---|---|---|---|
| Age, Continuous | 43.8 years STANDARD_DEVIATION 10.78 | 41.6 years STANDARD_DEVIATION 10.65 | 42.7 years STANDARD_DEVIATION 10.76 |
| Baseline Estimated glomerular filtration rate (eGFR) | 56.006 ml/min/1.73 m2 | 55.565 ml/min/1.73 m2 | 55.785 ml/min/1.73 m2 |
| Baseline Urine protein to creatinine ratio (UPCR) | 1.300 g/gram | 1.264 g/gram | 1.282 g/gram |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 43 Participants | 40 Participants | 83 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants | 5 Participants | 6 Participants |
| Race (NIH/OMB) White | 138 Participants | 137 Participants | 275 Participants |
| Sex: Female, Male Female | 65 Participants | 59 Participants | 124 Participants |
| Sex: Female, Male Male | 117 Participants | 123 Participants | 240 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 182 | 0 / 182 |
| other Total, other adverse events | 108 / 182 | 69 / 182 |
| serious Total, serious adverse events | 18 / 182 | 9 / 182 |
Outcome results
Primary
Part A: Ratio of Urine Protein to Creatinine Ratio (UPCR) at 9 Months Compared to Baseline
Part A primary endpoint: The ratio of Urine Protein to Creatinine Ratio (UPCR) (based on 24-hour urine collections) at 9 months following the first dose of study drug compared to baseline. Ratio being: UPCR at 9 months in g/gram divided with UPCR at Baseline in g/gram
Time frame: 9 months
Population: Full analysis set - global study - Part A
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part A: Ratio of Urine Protein to Creatinine Ratio (UPCR) at 9 Months Compared to Baseline | 0.69 ratio |
| Placebo Oral Capsule | Part A: Ratio of Urine Protein to Creatinine Ratio (UPCR) at 9 Months Compared to Baseline | 0.95 ratio |
p-value: 0.000396% CI: [0.61, 0.88]MMRM model
Primary
Part B: Time-weighted Average of Estimated Glomerular Filtration Rate (eGFR)
Part B Primary endpoint: Time-weighted average of estimated glomerular filtration rate (eGFR) recordings observed at each time point over 2 years. The eGFR (CKD-EPI) at 2 years (which must have been repeated to provide a second value obtained within 14 to 35 days) was the geometric mean of the 2 assessments.
Time frame: Up to 2 years and 1 month
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part B: Time-weighted Average of Estimated Glomerular Filtration Rate (eGFR) | 0.96 mL/min/1.73m2 |
| Placebo Oral Capsule | Part B: Time-weighted Average of Estimated Glomerular Filtration Rate (eGFR) | 0.87 mL/min/1.73m2 |
p-value: <0.000195% CI: [1.06, 1.15]robust regression
Secondary
Part A: Ratio of eGFR at 12 Months
Part A: Ratio of eGFR at 12 months compared to baseline calculated using the CKD-EPI formula.
Time frame: 12 months
Population: Full analysis set - global study - Part A
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part A: Ratio of eGFR at 12 Months | 0.97 mL/min/1.73m2 |
| Placebo Oral Capsule | Part A: Ratio of eGFR at 12 Months | 0.91 mL/min/1.73m2 |
p-value: 0.010695% CI: [1.01, 1.13]robust regression
Secondary
Part A: Ratio of eGFR at 9 Months
Part A: Ratio of eGFR at 9 months compared to baseline calculated using the CKD-EPI formula.
Time frame: 9 months
Population: Full analysis set - global study - Part A
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part A: Ratio of eGFR at 9 Months | 1.00 mL/min/1.73m2 |
| Placebo Oral Capsule | Part A: Ratio of eGFR at 9 Months | 0.93 mL/min/1.73m2 |
p-value: 0.001495% CI: [1.03, 1.13]robust regression
Secondary
Part A: Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months
Part A: Ratio of urine albumin to creatinine ratio (UACR) at 9 months compared to baseline.
Time frame: 9 months
Population: Full analysis set - global study - Part A
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part A: Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months | 0.64 g/gram |
| Placebo Oral Capsule | Part A: Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months | 0.93 g/gram |
p-value: 0.000595% CI: [0.55, 0.86]MMRM model
Secondary
Part B: Proportion of Patients Without Microhematuria
Part B: Proportion of patients without microhematuria in at least 2 of the following time points: 12, 18, and 24 months following the first dose of study drug
Time frame: 12 to 24 months
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Nefecon | Part B: Proportion of Patients Without Microhematuria | 94 Participants |
| Placebo Oral Capsule | Part B: Proportion of Patients Without Microhematuria | 59 Participants |
p-value: 0.000195% CI: [1.6, 4.1]Regression, Logistic
Secondary
Part B: Ratio of eGFR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months
Part B: Ratio of UPCR, UACR, and eGFR (CKD-EPI) compared to baseline averaged over time points between 12 and 24 months, inclusive, following the first dose of study drug;
Time frame: 12 to 24 months
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part B: Ratio of eGFR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 0.93 mL/min/1.73m2 |
| Placebo Oral Capsule | Part B: Ratio of eGFR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 0.84 mL/min/1.73m2 |
p-value: <0.000195% CI: [1.06, 1.16]robust regression
Secondary
Part B: Ratio of UACR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months
Part B: Ratio of UPCR, UACR, and eGFR (CKD-EPI) compared to baseline averaged over time points between 12 and 24 months, inclusive, following the first dose of study drug.
Time frame: 12 to 24 months
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part B: Ratio of UACR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 0.52 g/gram |
| Placebo Oral Capsule | Part B: Ratio of UACR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 0.96 g/gram |
p-value: <0.000195% CI: [0.45, 0.63]MMRM model
Secondary
Part B: Ratio of UPCR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months
Part B: Ratio of UPCR, UACR, and eGFR (CKD-EPI) compared to baseline averaged over time points between 12 and 24 months, inclusive, following the first dose of study drug
Time frame: 12, 18 and 24 months
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (GEOMETRIC_LEAST_SQUARES_MEAN) |
|---|---|---|
| Nefecon | Part B: Ratio of UPCR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 0.60 g/gram |
| Placebo Oral Capsule | Part B: Ratio of UPCR Compared to Baseline Averaged Over Time Points Between 12 and 24 Months | 1.01 g/gram |
p-value: <0.000195% CI: [0.51, 0.68]MMRM model
Secondary
Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months.
Part B: Short Form 36 (SF-36) quality of life assessment at 9 and 24 months. The 36-Item Short Form Health Survey (SF-36) is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting and have been widely used. It consists of eight health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. Higher scores indicate better health. Scores represent the percentage of total possible score achieved.
Time frame: 24 months
Population: Full analysis set - global study - Part B
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Bodily Pain | 53.385 Percentage of total possible score | Standard Deviation 9.6352 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | General Health | 47.038 Percentage of total possible score | Standard Deviation 9.345 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Mental Component Summary | 51.246 Percentage of total possible score | Standard Deviation 8.1362 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Mental Health | 51.691 Percentage of total possible score | Standard Deviation 8.2104 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Physical Component Summary | 51.443 Percentage of total possible score | Standard Deviation 7.52 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Physical Functioning | 52.665 Percentage of total possible score | Standard Deviation 7.6364 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Role Emotional | 50.322 Percentage of total possible score | Standard Deviation 8.469 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Role Physical | 50.948 Percentage of total possible score | Standard Deviation 7.8921 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Social Function | 51.949 Percentage of total possible score | Standard Deviation 7.9677 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Vitality | 53.291 Percentage of total possible score | Standard Deviation 8.8558 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Role Physical | 50.722 Percentage of total possible score | Standard Deviation 8.8255 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Bodily Pain | 53.208 Percentage of total possible score | Standard Deviation 9.9811 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Physical Functioning | 53.059 Percentage of total possible score | Standard Deviation 6.9006 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | General Health | 47.214 Percentage of total possible score | Standard Deviation 9.9564 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Vitality | 52.364 Percentage of total possible score | Standard Deviation 10.0023 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Mental Component Summary | 49.743 Percentage of total possible score | Standard Deviation 10.2184 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Role Emotional | 49.333 Percentage of total possible score | Standard Deviation 10.0789 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Mental Health | 50.294 Percentage of total possible score | Standard Deviation 10.0007 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Social Function | 51.471 Percentage of total possible score | Standard Deviation 8.9304 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 24 Months. | Physical Component Summary | 51.993 Percentage of total possible score | Standard Deviation 7.1783 |
Secondary
Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months.
Part B: Short Form 36 (SF-36) quality of life assessment at 9 and 24 months. The 36-Item Short Form Health Survey (SF-36) is a set of generic, coherent, and easily administered quality-of-life measures. These measures rely upon patient self-reporting and have been widely used. It consists of eight health concepts: physical functioning, bodily pain, role limitations due to physical health problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. Higher scores indicate better health. Scores represent the percentage of total possible score achieved.
Time frame: 9 months
Population: Full analysis set - global study - Part B
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Bodily Pain | 54.353 Percentage of total possible score | Standard Deviation 9.0306 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | General Health | 47.058 Percentage of total possible score | Standard Deviation 9.6373 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Mental Component Summary | 49.564 Percentage of total possible score | Standard Deviation 9.2834 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Mental Health | 50.226 Percentage of total possible score | Standard Deviation 8.9539 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Physical Component Summary | 52.257 Percentage of total possible score | Standard Deviation 7.2188 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Physical Functioning | 52.722 Percentage of total possible score | Standard Deviation 6.737 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Role Emotional | 49.574 Percentage of total possible score | Standard Deviation 9.4322 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Role Physical | 51.028 Percentage of total possible score | Standard Deviation 8.0207 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Social Function | 50.941 Percentage of total possible score | Standard Deviation 8.3606 |
| Nefecon | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Vitality | 51.826 Percentage of total possible score | Standard Deviation 10.0909 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Role Physical | 52.693 Percentage of total possible score | Standard Deviation 6.66 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Bodily Pain | 54.659 Percentage of total possible score | Standard Deviation 9.2387 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Physical Functioning | 54.276 Percentage of total possible score | Standard Deviation 5.2243 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | General Health | 48.014 Percentage of total possible score | Standard Deviation 10.012 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Vitality | 53.490 Percentage of total possible score | Standard Deviation 9.3987 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Mental Component Summary | 49.760 Percentage of total possible score | Standard Deviation 8.8974 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Role Emotional | 50.393 Percentage of total possible score | Standard Deviation 8.3001 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Mental Health | 50.099 Percentage of total possible score | Standard Deviation 8.8483 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Social Function | 51.707 Percentage of total possible score | Standard Deviation 7.9259 |
| Placebo Oral Capsule | Part B: Short Form 36 (SF-36) Quality of Life Assessment at 9 Months. | Physical Component Summary | 53.732 Percentage of total possible score | Standard Deviation 6.5876 |
Secondary
Part B: Time to 30% Reduction in eGFR
Part B: Time to 30% reduction from baseline in eGFR (CKD-EPI) confirmed by a second value. For clarity: Please note that the number of patients with a 30% reduction is presented with statistical analysis of the time to 30% reduction.
Time frame: Over 2 years
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Nefecon | Part B: Time to 30% Reduction in eGFR | 21 Participants |
| Placebo Oral Capsule | Part B: Time to 30% Reduction in eGFR | 39 Participants |
p-value: 0.002895% CI: [0.26, 0.75]Cox proportional hazards model
Secondary
Part B: Time to Receiving Rescue Medication.
Part B: Time from the first dose of study drug until receiving rescue medication. For clarity: Please note that the number of patients receiving rescue medication is presented with statistical analysis of the time to receiving rescue medication.
Time frame: Over 2 years
Population: Full analysis set - global study - Part B
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Nefecon | Part B: Time to Receiving Rescue Medication. | 15 Participants |
| Placebo Oral Capsule | Part B: Time to Receiving Rescue Medication. | 20 Participants |
p-value: 0.264795% CI: [0.34, 1.33]Cox proportional hazards model