Primary IgA NephropathyPhase 3Nefigard-OLENCT04541043
Efficacy and Safety in Patients With Primary IgA Nephropathy Who Have Completed Study Nef-301 (Nefigard-OLE)
A Phase 3 study testing Nefecon 16mg daily in people with Primary IgA Nephropathy, run by Calliditas Therapeutics AB at 18 sites in 18 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary IgA Nephropathy
- What is being tested?
- Nefecon 16mg daily
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 119 participants
- Where
- 18 sitesin 18 countries
- Study dates
- 17 Nov 2020to 26 Feb 2024
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
18 sites in 18 countries.
18 sites in 18 countries
About this study
Official title: An Open-Label Extension (OLE) Study to Evaluate the Efficacy and Safety of Nefecon Treatment in Patients With IgA Nephropathy Who Have Completed Study Nef-301
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.
Read the detailed description
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.
- Conditions
- Primary IgA Nephropathy
- Being tested
- Nefecon 16mg daily
- Medical subject headings
- Glomerulonephritis, IGA
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nefecon 16mg dailyDrug
All study patients received Nefecon 16 mg daily for 9 months.
| Study group | Role | What they receive |
|---|---|---|
| Active Nefecon treatment | Experimental | Nefecon 16 mg once daily by mouth for 9 months Receives: Drug: Nefecon 16mg daily |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 9 months
Secondary outcomes (7)
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.
Measured: 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.
Measured: Baseline & 12 months
Number of Patients With Microhematuria at 9 Months Compared to Baseline
Measured: 9 months
Number of Patients Receiving Rescue Treatment
Systemic immunosuppressive drugs (including glucocorticoids in some situations ), dialysis, and renal transplantation are considered as rescue medications in this study.
Measured: 12 months
Proportion of Patients on Dialysis, Undergoing Kidney Transplantation, or With eGFR <15 mL/Min Per 1.73 m2
Looking at number of patients with end stage kidney disease defined as being on dialysis, undergoing kidney transplantation, or having eGFR <15 mL/min per 1.73 m2
Measured: 12 months
Change From Baseline Cortisol Suppression at 9 Months
Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.
Measured: Baseline & 9 months
Change From Baseline Cortisol Suppression at 12 Months
Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.
Measured: Baseline & 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2020
- Study start17 Nov 2020
- Primary completion26 Feb 2024
- Study completion26 Feb 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Primary IgA NephropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- This is an open label, single-arm study with active treatment
- Masking
- None (open label)
- Enrolment
- 119 participants (actual)
Sponsor and investigators
- Lead sponsor
- Calliditas Therapeutics AB
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Richard Philipson, MD, Study director, Calliditas Therapeutics AB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04541043
- Sponsor's protocol ID
- Nef-301 OLE
- Other IDs
- 2020-003308-14
- Acronym
- Nefigard-OLE
- Status last verified
- Dec 2024
- First submitted
- 1 Sep 2020
- Last updated
- 10 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension (OLE) Study to Evaluate the Efficacy and Safety of Nefecon Treatment in Patients With IgA Nephropathy Who Have Completed Study Nef-301
- ClinicalTrials.gov ID
- NCT04541043
- Acronym / sponsor ID
- Nefigard-OLE / Nef-301 OLE
- Conditions
- Primary IgA Nephropathy
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 119 participants (actual)
- Lead sponsor
- Calliditas Therapeutics ABIndustry (pharma, biotech, devices)
- Study start
- 17 Nov 2020 (actual)
- Primary completion
- 26 Feb 2024 (actual)
- Study completion
- 26 Feb 2024 (actual)
- First posted
- 9 Sep 2020 (actual)
- Results
- Posted 3 Jan 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- This is an open label, single-arm study with active treatment
| Arm | Type | Receives |
|---|---|---|
| Active Nefecon treatment | Experimental | Drug: Nefecon 16mg dailyNefecon 16 mg once daily by mouth for 9 months |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (9 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nefecon 16mg dailyAll study patients received Nefecon 16 mg daily for 9 months. | Drug | Active Nefecon treatment |
Primary outcome
Ratio of Urine Protein to Creatine Ratio (UPCR) at 9 Months Compared to Baseline
Time frame: 9 months
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
Ratio of Estimated Glomerular Filtration Rate (eGFR) at 9 Months Compared to Baseline
Time frame: 9 months
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.
7 secondary outcomes
Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months Compared to Baseline
Time frame: 9 months
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months
Time frame: Baseline & 12 months
Number of Patients With Microhematuria at 9 Months Compared to Baseline
Time frame: 9 months
Number of Patients Receiving Rescue Treatment
Time frame: 12 months
Proportion of Patients on Dialysis, Undergoing Kidney Transplantation, or With eGFR <15 mL/Min Per 1.73 m2
Time frame: 12 months
Change From Baseline Cortisol Suppression at 9 Months
Time frame: Baseline & 9 months
Change From Baseline Cortisol Suppression at 12 Months
Time frame: Baseline & 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Richard Philipson, MD study director
Calliditas Therapeutics AB
18 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nefecon 16mg daily in people with Primary IgA Nephropathy, run by Calliditas Therapeutics AB at 18 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Argentina, Australia, Belarus, Belgium and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Jan 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ratio of Urine Protein to Creatine Ratio (UPCR) at 9 Months Compared to Baseline (time frame: 9 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Feb 2025.