Efficacy and Safety of Nefecon in Patients With Primary IgA (Immunoglobulin A) Nephropathy (Nefigard)

Calliditas Therapeutics

Status and phase: Completed; Phase 3

Conditions: Primary IgA Nephropathy

Treatments: Drug: Placebo oral capsule; Drug: Nefecon

Study type: Interventional

Funder types: Industry

Identifiers

NCT03643965

Nef-301

Details and patient eligibility

About

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).

Full 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.

Enrollment: 365 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

365 participants in 2 patient groups, including a placebo group

Nefecon

Experimental group

Description:

Nefecon 16 mg once daily by mouth for 9 months.

Treatment:

Drug: Nefecon

Placebo oral capsule

Placebo Comparator group

Description:

Placebo oral capsule once daily by mouth for 9 months.

Treatment:

Drug: Placebo oral capsule