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

NCT04541043 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: Nef-301 OLE2020-003308-14
• Study Type: interventional
• Target: 119
• Locations: 17 countries
• Sites: 17

Brief Summary

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.

Conditions

Primary IgA Nephropathy

Outcome Measures

Primary Outcomes (2)

• 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

  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.

  9 months

Secondary Outcomes (7)

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

  9 months

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

  Baseline & 12 months

• Number of Patients With Microhematuria at 9 Months Compared to Baseline

  9 months

• Number of Patients Receiving Rescue Treatment

  12 months

• Proportion of Patients on Dialysis, Undergoing Kidney Transplantation, or With eGFR <15 mL/Min Per 1.73 m2

  12 months

Study Arms (1)

Active Nefecon treatment

EXPERIMENTAL

Nefecon 16 mg once daily by mouth for 9 months

Drug: Nefecon 16mg daily

Interventions

Nefecon 16mg daily DRUG

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

Active Nefecon treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients that completed study Nef-301
• 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
• Willing and able to provide written informed consent.
• UPCR equal to or more than 0.8 g/gram
• eGFR equal to or more than 30 mL/min per 1.73 m2 using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula.

You may not qualify if:

• Systemic diseases that may cause mesangial IgA deposition.
• Patients who have undergone a kidney transplant;
• Patients with presence of other glomerulopathies and/or nephrotic syndrome
• Patients with acute, chronic, or latent infectious disease including hepatitis, tuberculosis (TB), human immunodeficiency virus (HIV), and chronic urinary tract infections;
• Patients with liver cirrhosis, as assessed by the Investigator;
• Patients with a diagnosis of type 1 or type 2 diabetes mellitus which is poorly controlled
• Patients with history of unstable angina, class III or IV congestive heart failure, and/or clinically significant arrhythmia, as judged by the Investigator;
• Patients with unacceptable blood pressure control defined as a blood pressure consistently above national guidelines for proteinuric renal disease, as assessed by the Investigator.
• Patients with diagnosed malignancy within the past 5 years.

Sponsors & Collaborators

• Calliditas Therapeutics AB: lead

Study Sites (18)

13 Investigator sites

Palo Alto, California, 94304, United States

Location

4 Investigator sites

Buenos Aires, Argentina

Location

6 Investigator sites

Melbourne, Australia

Location

3 Investigator sites

Minsk, Belarus

Location

4 Investigator sites

Brussels, Belgium

Location

7 Investigator sites

Québec, Canada

Location

6 Investigator sites

Prague, Czechia

Location

2 Investigator sites

Jyväskylä, Finland

Location

2 Investigator sites

Saint-Priest-en-Jarez, France

Location

5 Investigator sites

Aachen, Germany

Location

5 Investigator sites

Athens, Greece

Location

2 Investigator sites

Milan, Italy

Location

2 Investigator sites

Lodz, Poland

Location

4 Investigator sites

Gyeonggi-do, South Korea

Location

4 Investigator sites

Barcelona, Spain

Location

3 Investigator sites

Uppsala, Sweden

Location

3 Investigator sites

Kayseri, Turkey (Türkiye)

Location

6 Investigator sites

Leicester, United Kingdom

Location

MeSH Terms

Conditions

Glomerulonephritis, IGA

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Clinical Program Leader
• Organization: Calliditas Therapeutics

kristin.onnestam@calliditas.com

0737456451

Study Officials

• Richard Philipson, MD

  Calliditas Therapeutics AB

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is an open label, single-arm study with active treatment

Study Record Dates

• First Submitted: September 1, 2020
• First Posted: September 9, 2020
• Study Start: November 17, 2020
• Primary Completion: February 26, 2024
• Study Completion: February 26, 2024
• Last Updated: February 10, 2025
• Results First Posted: January 3, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); DE (1); KR (1); PL (1); BE (1); ES (1); AR (1); FI (1); US (1); TU (1); CA (1); GB (1); IT (1); CZ (1); FR (1); GR (1); SE (1)