Immunoglobulin A Nephropathy (IgAN) MedDRA version: 20.0 Level: PT Classification code 10021263 Term: IgA nephropathy System Organ Class: 10038359 - Renal and urinary disorders
Conditions
Sponsors
Retrophin, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion Criteria for Double-Blind Period: 1. Male or female, aged =18 years. 2. Biopsy-proven IgAN. 3. Urine total protein value =1.0 g/day at screening. 4. eGFR =30 mL/min/1.73 m2 at screening. 5. The patient has been on a stable dose of ACEI and/or ARB therapy for at least 12 weeks prior to screening. 6. At screening, systolic blood pressure =150 mmHg and diastolic blood pressure =100 mmHg. 7. Women of childbearing potential (WOCBP) must agree to the use of two forms of contraception. Inclusion Criteria for Open-Label Extension Period: Based on assessments at the Week 110 visit, a patient must meet all of the following criteria to be eligible for the open-label extension period. 1. The patient completed participation in the double-blind period, including the Week 114 visit. 2. The patient is willing and able to provide signed informed consent for participation in the open-label extension period. 3. The patient did not permanently discontinue study medication during the double-blind period. 4. WOCBP, beginning at menarche, must agree to the use of 1 highly reliable (ie, can achieve a failure rate of =65 years) yes F.1.3.1 Number of subjects for this age range 40
Exclusion criteria
Exclusion criteria: Exclusion Criteria for the Double-Blind Period: 1. IgAN secondary to another condition. 2. Cellular glomerular crescents present in >25% of glomeruli on renal biopsy within 6 months prior to screening. 3. The patient has a chronic kidney disease (CKD) in addition to IgAN. 4. Any organ transplantation, with the exception of corneal transplants. 5. Treatment with any of the prohibited concomitant medications. 6. Treatment with any systemic immunosuppressive medications (including corticosteroids) for >2 weeks within 3 months prior to screening. 7. Documented history of heart failure and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema. 8. Clinically significant cerebrovascular disease and/or coronary artery disease. 9. Jaundice, hepatitis, or known hepatobiliary disease (excluding asymptomatic cholelithiasis), or transaminase levels >2 times the upper limit of the normal range at screening. 10. History of malignancy other than adequately treated basal cell or squamous cell skin cancer or cervical carcinoma within the past 2 years. 11. A screening hematocrit value 5.5 mEq/L (5.5 mmol/L). 13. Female patient is pregnant, breastfeeding or planning to conceive during the study. 14. Participation in a study of another investigational product within 28 days prior to screening. Exclusion Criteria for the Open-Label Extension Period: Based on assessments at the Week 110 and Week 114 visits, a patient who meets any of the following criteria will be excluded from the openlabel extension period: 1. The patient has progressed to end-stage renal disease (ESRD) requiring renal replacement therapy (RRT). 2. The patient developed any criteria for discontinuation of study medication or discontinuation from the study, respectively, between Week 110 and Week 114. 3. The patient was unable to initiate, or developed contraindications to, treatment with RAAS inhibitors between Week 110 and Week 114. 4. The patient has an eGFR =20 mL/min/1.73 m2 at Week 110. 5. The female patient is pregnant or is breastfeeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The efficacy objective of the study is to determine the effect of sparsentan on proteinuria and renal function, as compared to an angiotensin receptor blocker, in patients with immunoglobulin A nephropathy (IgAN). The safety objective of the study is to assess the safety and tolerability of sparsentan by double-blind monitoring of safety endpoints. The open-label objective of the study is to assess the long-term efficacy, safety, and tolerability of open-label treatment with sparsentan in patients with IgAN.;Secondary Objective: Not applicable;Primary end point(s): Efficacy End points: The primary efficacy endpoint is the change from baseline in the urine protein/creatinine ratio (UP/C) at Week 36. Safety End points: • Descriptive statistics will be used to summarize the safety data. Open-Label Extension Period Endpoints: Endpoints for the open-label extension period include, but are not necessarily limited to: • The absolute and percent change from Week 114 in eGFR at each visit • The percent change from Week 114 in UP/C at each visit • Changes from Week 114 in QoL at each visit • Changes from Week 114 in body weight, vital signs, physical examinations, peripheral edema, and clinical laboratory parameters • Changes from Week 114 in lipid profile (total cholesterol and triglycerides, low density lipoprotein C [LDL-C], and high density lipoprotein C [HDL-C]) • The incidence of TEAEs during the open-label extension period;Timepoint(s) of evaluation of this end point: Primary efficacy endpoints will be evaluated at Week 36 or as defined above. The primary safety endpoint will be evaluated from randomization to Week 114 or as defined above. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Rate of change of eGFR;Timepoint(s) of evaluation of this end point: From baseline to week 114 | — |
Countries
Australia, Belgium, Croatia, Czech Republic, Estonia, France, Germany, Hong Kong, Italy, Korea, Republic of, Lithuania, New Zealand, Poland, Portugal, Spain, Taiwan, United Kingdom, United States
Contacts
Public ContactRetrophin Call Center
Retrophin, Inc.
Outcome results
None listed