Efficacy and Safety in Patients With Primary IgA Nephropathy Who Have Completed Study Nef-301 (Nefigard-OLE)

Calliditas Therapeutics AB119 enrolled

Overview

This is a Phase 3b, multicenter, open-label extension (OLE) study to evaluate the efficacy and safety of Nefecon treatment in patients with IgAN who have completed the Phase 3 Study Nef 301 and continue to be treated with a stable dose of RAS inhibitor therapy (ACEIs and/or ARBs). Patients who previously received Nefecon in Study Nef-301 will receive retreatment, whereas patients who previously received placebo in Study Nef-301 will be treatment naïve to Nefecon. During Study Nef-301 OLE, the patients and Investigators will remain blinded to treatment given in Study Nef-301. During Study Nef-301 OLE, patients will receive Nefecon for a 9-month period. The dose may be reduced if clinically relevant adverse events (AEs) develop during the 9-month Treatment Period that the Investigator considers related to the study drug and that mandate dose reduction. Patients will remain on a stable dose of RAS inhibitor therapy (ACEIs and/or ARBs) throughout the study. The patient will come for a follow-up visit at 12 months after first dose.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

119

Conditions

Primary IgA Nephropathy

Interventions

Nefecon 16mg dailyDRUG

All study patients received Nefecon 16 mg daily for 9 months.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Patients that completed study Nef-301 2. On a stable dose of RAS inhibitor therapy (ACEIs and/or ARBs) at the maximum allowed dose or maximum tolerated dose according to the 2012 KDIGO guidelines 3. Willing and able to provide written informed consent. 4. UPCR equal to or more than 0.8 g/gram 5. eGFR equal to or more than 30 mL/min per 1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula. Exclusion Criteria: 1. Systemic diseases that may cause mesangial IgA deposition. 2. Patients who have undergone a kidney transplant; 3. Patients with presence of other glomerulopathies and/or nephrotic syndrome 4. Patients with acute, chronic, or latent infectious disease including hepatitis, tuberculosis (TB), human immunodeficiency virus (HIV), and chronic urinary tract infections; 5. Patients with liver cirrhosis, as assessed by the Investigator; 6. Patients with a diagnosis of type 1 or type 2 diabetes mellitus which is poorly controlled 7. Patients with history of unstable angina, class III or IV congestive heart failure, and/or clinically significant arrhythmia, as judged by the Investigator; 8. Patients with unacceptable blood pressure control defined as a blood pressure consistently above national guidelines for proteinuric renal disease, as assessed by the Investigator. 9. Patients with diagnosed malignancy within the past 5 years.

Locations (18)

13 Investigator sites

Palo Alto, California, United States

4 Investigator sites

Buenos Aires, Argentina

6 Investigator sites

Melbourne, Australia

Outcomes

Primary Outcomes

Ratio of Urine Protein to Creatine Ratio (UPCR) at 9 Months Compared to Baseline

The outcome is measured as UPCR based on 24 hour urine collections at 9 months following the first dose of Nefecon compared to baseline Ratio being: UPCR at 9 months in g/gram divided with UPCR at Baseline in g/gram

Time frame: 9 months

Ratio of Estimated Glomerular Filtration Rate (eGFR) at 9 Months Compared to Baseline

The outcome is measured as ratio of eGFR in mL/min/1.73 m2 (calculated using the CKD-EPI formula) at 9 months following the first dose of Nefecon compared to baseline. I.e. eGFR at 9 months divided by eGFR at Baseline.

Time frame: 9 months

Secondary Outcomes

Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months Compared to Baseline

Ratio of urine albumin to creatinine ratio (UACR) measured by 24h urine sampling at 9 months compared to baseline. I.e. UACR at 9 months divided by eGFR at Baseline.

Time frame: 9 months

Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months

Short Form 36 (SF-36) quality of life assessment at 12 months compared to baseline, i.e. change from baseline. 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, i.e. 0 is the minimum and 100 is the maximum score.

Time frame: Baseline & 12 months

Number of Patients With Microhematuria at 9 Months Compared to Baseline

Data from ClinicalTrials.gov

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