Trial Outcomes & Findings for Efficacy and Safety in Patients With Primary IgA Nephropathy Who Have Completed Study Nef-301 (Nefigard-OLE) (NCT NCT04541043)

Last Updated: 2025-02-10

Results Overview

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

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

119 participants

Primary outcome timeframe

9 months

Results posted on

2025-02-10

Participant Flow

Participant milestones

Participant milestones
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Overall Study STARTED	45	74
Overall Study COMPLETED	43	62
Overall Study NOT COMPLETED	2	12

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=45 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=74 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Total n=119 Participants Total of all reporting groups
Age, Continuous	46 years n=5 Participants	47 years n=7 Participants	47 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants	19 Participants n=7 Participants	25 Participants n=5 Participants
Sex: Female, Male Male	39 Participants n=5 Participants	55 Participants n=7 Participants	94 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	9 Participants n=5 Participants	7 Participants n=7 Participants	16 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	36 Participants n=5 Participants	64 Participants n=7 Participants	100 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment Argentina	6 participants n=5 Participants	3 participants n=7 Participants	9 participants n=5 Participants
Region of Enrollment Czechia	2 participants n=5 Participants	13 participants n=7 Participants	15 participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants	5 participants n=7 Participants	11 participants n=5 Participants
Region of Enrollment United Kingdom	3 participants n=5 Participants	3 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment Belarus	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Spain	1 participants n=5 Participants	1 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Greece	3 participants n=5 Participants	5 participants n=7 Participants	8 participants n=5 Participants
Region of Enrollment Canada	7 participants n=5 Participants	7 participants n=7 Participants	14 participants n=5 Participants
Region of Enrollment South Korea	2 participants n=5 Participants	4 participants n=7 Participants	6 participants n=5 Participants
Region of Enrollment Sweden	1 participants n=5 Participants	2 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Turkey	0 participants n=5 Participants	2 participants n=7 Participants	2 participants n=5 Participants
Region of Enrollment Belgium	4 participants n=5 Participants	3 participants n=7 Participants	7 participants n=5 Participants
Region of Enrollment Finland	1 participants n=5 Participants	3 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Poland	0 participants n=5 Participants	3 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Italy	2 participants n=5 Participants	1 participants n=7 Participants	3 participants n=5 Participants
Region of Enrollment Australia	2 participants n=5 Participants	7 participants n=7 Participants	9 participants n=5 Participants
Region of Enrollment France	1 participants n=5 Participants	3 participants n=7 Participants	4 participants n=5 Participants
Region of Enrollment Germany	3 participants n=5 Participants	8 participants n=7 Participants	11 participants n=5 Participants
Urine Protein to Creatine Ratio (UPCR)	1.253 g/gram n=5 Participants	1.339 g/gram n=7 Participants	1.306 g/gram n=5 Participants
Baseline Estimated glomerular filtration rate (eGFR)	51.0 ml/min/1.73 m2 n=5 Participants	49.9 ml/min/1.73 m2 n=7 Participants	50.3 ml/min/1.73 m2 n=5 Participants

PRIMARY outcome

Timeframe: 9 months

Population: Number of participants analyzed reflects number of patients with available data at baseline and 9 months .

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=44 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=69 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Ratio of Urine Protein to Creatine Ratio (UPCR) at 9 Months Compared to Baseline	0.67 ratio Interval 0.56 to 0.8	0.69 ratio Interval 0.6 to 0.8

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=44 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=69 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Ratio of Estimated Glomerular Filtration Rate (eGFR) at 9 Months Compared to Baseline	0.97 ratio Interval 0.94 to 1.01	0.97 ratio Interval 0.94 to 1.0

SECONDARY outcome

Timeframe: 9 months

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.

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=44 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=69 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Ratio of Urine Albumin to Creatinine Ratio (UACR) at 9 Months Compared to Baseline	0.60 ratio Interval 0.49 to 0.75	0.65 ratio Interval 0.55 to 0.77

SECONDARY outcome

Timeframe: Baseline & 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.

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=44 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=70 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Bodily Pain	-4.518 Change in percentage of total Score Standard Deviation 10.0522	-3.756 Change in percentage of total Score Standard Deviation 9.9213
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months General Health	-4.290 Change in percentage of total Score Standard Deviation 7.4076	-3.301 Change in percentage of total Score Standard Deviation 5.8211
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Mental Component Summary	-2.282 Change in percentage of total Score Standard Deviation 7.1270	-1.061 Change in percentage of total Score Standard Deviation 6.6251
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Mental Health	-1.725 Change in percentage of total Score Standard Deviation 7.1576	-1.570 Change in percentage of total Score Standard Deviation 6.3522
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Physical Component Summary	-3.952 Change in percentage of total Score Standard Deviation 6.6367	-3.403 Change in percentage of total Score Standard Deviation 6.1115
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Physical Functioning	-2.697 Change in percentage of total Score Standard Deviation 7.4670	-1.996 Change in percentage of total Score Standard Deviation 6.6726
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Role Emotional	-2.928 Change in percentage of total Score Standard Deviation 7.2390	-0.747 Change in percentage of total Score Standard Deviation 7.4962
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Role Physical	-3.421 Change in percentage of total Score Standard Deviation 7.1914	-2.793 Change in percentage of total Score Standard Deviation 7.6901
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Social Function	-4.102 Change in percentage of total Score Standard Deviation 8.4631	-2.364 Change in percentage of total Score Standard Deviation 6.3713
Change From Baseline Short Form 36 (SF-36) Quality of Life Assessment at 12 Months Vitality	-2.633 Change in percentage of total Score Standard Deviation 7.6406	-2.376 Change in percentage of total Score Standard Deviation 7.6968

SECONDARY outcome

Timeframe: 9 months

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=44 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=68 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Number of Patients With Microhematuria at 9 Months Compared to Baseline	10 Participants	17 Participants

SECONDARY outcome

Timeframe: 12 months

Systemic immunosuppressive drugs (including glucocorticoids in some situations ), dialysis, and renal transplantation are considered as rescue medications in this study.

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=45 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=74 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Number of Patients Receiving Rescue Treatment	1 Participants	0 Participants

SECONDARY outcome

Timeframe: 12 months

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

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=45 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=74 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Proportion of Patients on Dialysis, Undergoing Kidney Transplantation, or With eGFR <15 mL/Min Per 1.73 m2	2 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline & 9 months

Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=44 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=66 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Change From Baseline Cortisol Suppression at 9 Months	-24.122 ug/day) Standard Deviation 25.1690	-32.126 ug/day) Standard Deviation 30.6978

SECONDARY outcome

Timeframe: Baseline & 12 months

Cortisol suppression at 9 and 12 months, measured as urinary cortisol excretion over 24 hours compared to baseline.

Outcome measures

Outcome measures
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=41 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=63 Participants Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Change From Baseline Cortisol Suppression at 12 Months	-7.660 ug/day Standard Deviation 27.4262	-11.954 ug/day Standard Deviation 25.8052

Adverse Events

Retreatment - Previously Treated With Nefecon in Nef-301 Study

Serious events: 5 serious events

Other events: 32 other events

Deaths: 0 deaths

Delayed Treatment - Previously Treated With Placebo in Nef-301 Study

Serious events: 5 serious events

Other events: 46 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=45 participants at risk Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=74 participants at risk Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Blood and lymphatic system disorders Lymphadenopathy	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	0.00% 0/74 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Blood and lymphatic system disorders Monoclonal B-cell lymphocytosis	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	1.4% 1/74 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Injury, poisoning and procedural complications Joint dislocation	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	0.00% 0/74 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Injury, poisoning and procedural complications Scapula fracture	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	1.4% 1/74 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Nervous system disorders Central nervous system vasculitis	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	0.00% 0/74 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Nervous system disorders Headache	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	1.4% 1/74 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Cardiac disorders Atrial fibrillation	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	0.00% 0/74 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Eye disorders Chorioretinopathy	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	1.4% 1/74 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Gastrointestinal disorders Vomiting	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	1.4% 1/74 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Musculoskeletal and connective tissue disorders Osteoarthritis	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	0.00% 0/74 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Breast cancer	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	1.4% 1/74 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Renal and urinary disorders Haematuria	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	0.00% 0/74 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.

Other adverse events

Additional Information

Clinical Program Leader

Calliditas Therapeutics

Phone: 0737456451

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Retreatment - Previously Treated With Nefecon in Nef-301 Study n=45 participants at risk Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.	Delayed Treatment - Previously Treated With Placebo in Nef-301 Study n=74 participants at risk Nef-301-OLE study is an extension study to the Nef-301 study. Study results are analyzed based on treatment in the previous Nef-301 study where patients were randomized to receive either Nefecon or Placebo. Thus, in this Nef-301-OLE study the patients are either retreated with Nefecon or receiving Nefecon for the first time.
Infections and infestations Corona virus infection	26.7% 12/45 • Number of events 12 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	17.6% 13/74 • Number of events 14 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Infections and infestations Folliculitis	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	5.4% 4/74 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Infections and infestations Nasopharyngitis	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	5.4% 4/74 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Infections and infestations Upper respiratory tract infection	6.7% 3/45 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	2.7% 2/74 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Gastrointestinal disorders Nausea	6.7% 3/45 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	2.7% 2/74 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Vascular disorders Hypertension	17.8% 8/45 • Number of events 9 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	16.2% 12/74 • Number of events 13 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Skin and subcutaneous tissue disorders Acne	6.7% 3/45 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	8.1% 6/74 • Number of events 7 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Investigations Weight increased	6.7% 3/45 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	10.8% 8/74 • Number of events 8 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
General disorders Fatigue	8.9% 4/45 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	2.7% 2/74 • Number of events 2 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
General disorders Oedema peripheral	2.2% 1/45 • Number of events 1 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	13.5% 10/74 • Number of events 12 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
General disorders Pyrexia	0.00% 0/45 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	5.4% 4/74 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Musculoskeletal and connective tissue disorders Arthralgia	8.9% 4/45 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	4.1% 3/74 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Musculoskeletal and connective tissue disorders Back pain	6.7% 3/45 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	4.1% 3/74 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Musculoskeletal and connective tissue disorders Muscle spasms	13.3% 6/45 • Number of events 8 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	6.8% 5/74 • Number of events 6 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Psychiatric disorders Insomnia	6.7% 3/45 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	8.1% 6/74 • Number of events 6 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Nervous system disorders Headache	8.9% 4/45 • Number of events 6 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	4.1% 3/74 • Number of events 3 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Renal and urinary disorders Proteinuria	8.9% 4/45 • Number of events 4 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	6.8% 5/74 • Number of events 5 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.
Endocrine disorders Cushingoid	4.4% 2/45 • Number of events 2 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.	8.1% 6/74 • Number of events 6 • All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug. All-Cause Mortality, SAEs and non-serious AEs were assessed from date of first dose of study treatment until 12 months after the first dose of study drug.