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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04541043




Registration number
NCT04541043
Ethics application status
Date submitted
1/09/2020
Date registered
9/09/2020

Titles & IDs
Public title
Efficacy and Safety in Patients With Primary IgA Nephropathy Who Have Completed Study Nef-301 (Nefigard-OLE)
Scientific 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
Secondary ID [1] 0 0
2020-003308-14
Secondary ID [2] 0 0
Nef-301 OLE
Universal Trial Number (UTN)
Trial acronym
Nefigard-OLE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary IgA Nephropathy 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Nefecon 16mg daily

Experimental: Active Nefecon treatment - Nefecon 16 mg once daily by mouth for 9 months


Treatment: Drugs: Nefecon 16mg daily
All study patients received Nefecon 16 mg daily for 9 months.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Ratio of Urine Protein to Creatine Ratio (UPCR) at 9 Months Compared to Baseline
Assessment method [1] 0 0
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
Timepoint [1] 0 0
9 months
Primary outcome [2] 0 0
Ratio of Estimated Glomerular Filtration Rate (eGFR) at 9 Months Compared to Baseline
Assessment method [2] 0 0
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.
Timepoint [2] 0 0
9 months
Secondary outcome [1] 0 0
Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months Compared to Baseline
Assessment method [1] 0 0
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.
Timepoint [1] 0 0
9 months
Secondary outcome [2] 0 0
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months
Assessment method [2] 0 0
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.
Timepoint [2] 0 0
Baseline & 12 months
Secondary outcome [3] 0 0
Number of Patients With Microhematuria at 9 Months Compared to Baseline
Assessment method [3] 0 0
Timepoint [3] 0 0
9 months
Secondary outcome [4] 0 0
Number of Patients Receiving Rescue Treatment
Assessment method [4] 0 0
Systemic immunosuppressive drugs (including glucocorticoids in some situations ), dialysis, and renal transplantation are considered as rescue medications in this study.
Timepoint [4] 0 0
12 months
Secondary outcome [5] 0 0
Proportion of Patients on Dialysis, Undergoing Kidney Transplantation, or With eGFR <15 mL/Min Per 1.73 m2
Assessment method [5] 0 0
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
Timepoint [5] 0 0
12 months
Secondary outcome [6] 0 0
Change From Baseline Cortisol Suppression at 9 Months
Assessment method [6] 0 0
Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.
Timepoint [6] 0 0
Baseline & 9 months
Secondary outcome [7] 0 0
Change From Baseline Cortisol Suppression at 12 Months
Assessment method [7] 0 0
Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.
Timepoint [7] 0 0
Baseline & 12 months

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/11/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
26/02/2024
Sample size
Target
Accrual to date
Final
119
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
6 Investigator sites - Melbourne
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
Argentina
State/province [2] 0 0
Buenos Aires
Country [3] 0 0
Belarus
State/province [3] 0 0
Minsk
Country [4] 0 0
Belgium
State/province [4] 0 0
Brussel
Country [5] 0 0
Canada
State/province [5] 0 0
Québec
Country [6] 0 0
Czechia
State/province [6] 0 0
Praha
Country [7] 0 0
Finland
State/province [7] 0 0
Jyväskylä
Country [8] 0 0
France
State/province [8] 0 0
Saint-Priest-en-Jarez
Country [9] 0 0
Germany
State/province [9] 0 0
Aachen
Country [10] 0 0
Greece
State/province [10] 0 0
Athens
Country [11] 0 0
Italy
State/province [11] 0 0
Milano
Country [12] 0 0
Korea, Republic of
State/province [12] 0 0
Gyeonggi-do
Country [13] 0 0
Poland
State/province [13] 0 0
Lódz
Country [14] 0 0
Spain
State/province [14] 0 0
Barcelona
Country [15] 0 0
Sweden
State/province [15] 0 0
Uppsala
Country [16] 0 0
Turkey
State/province [16] 0 0
Kayseri
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Leicester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Calliditas Therapeutics AB
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Richard Philipson, MD
Address 0 0
Calliditas Therapeutics AB
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol Study Protocol and Statistical Analysis Plan
Statistical analysis plan Study Protocol and Statistical Analysis Plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT04541043