Effectiveness and Safety of Sparsentan as treatment for Primary Focal Segmental Glomerulosclerosis (FSGS)
- Conditions
- Focal segmental glomerulosclerosis (FSGS)MedDRA version: 21.1Level: PTClassification code 10067757Term: Focal segmental glomerulosclerosisSystem Organ Class: 10038359 - Renal and urinary disordersTherapeutic area: Diseases [C] - Symptoms and general pathology [C23]
- Registration Number
- EUCTR2016-005141-23-IT
- Lead Sponsor
- RETROPHIN, INC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 300
1. The patient or parent/legal guardian (as appropriate) is willing and able to provide signed informed consent, and where required, the patient is willing to provide assent, prior to any screening procedures.
2. The patient has biopsy-proven primary FSGS or documentation of a genetic mutation in a podocyte protein associated with FSGS. The biopsy may have been performed at any time in the past, but will preferably include electron microscopy (EM) and immunofluorescence (IF) characteristics consistent with primary FSGS. The patient may be enrolled based on light microscopy diagnosis of FSGS in the absence of EM and/or IF analysis, provided the history (nephrotic syndrome with hypoalbuminemia, treatment with immunosuppression) and the course of the disease are indicative of primary FSGS, and potential secondary causes captured by the exclusion criteria have been carefully ruled out.
3. Sites within the US: The patient is male or female aged 8 to 75 years, inclusive.
Sites outside the US: The patient is male or female aged 18 to 75 years, inclusive.
4. The patient has a UP/C =1.5 g/g at screening.
5. The patient has an eGFR =30 mL/min/1.73 m2 at screening.
6. The patient has a mean seated blood pressure =100/60 mmHg (on a maximum of 2 antihypertensive treatments at screening, including RAAS inhibitors) and =160/100 mmHg (patients >18 years of age) or =90/60 mmHg and = the 95th percentile for age, sex, and height (patients =18 years of age).
7. Sexually active women of childbearing potential (WOCBP) must agree to the simultaneous use of 2 medically accepted methods of contraception from Day 1/Randomization until 90 days after the last dose of study medication. At least one method of contraception must be highly reliable (ie, can achieve a failure rate of <1% per year), such as stable oral, implanted, transdermal, or injected contraceptive hormones associated with inhibition of ovulation, or an intrauterine device (IUD) in place for at least 3 months. The other method of contraception must be a barrier method, such as a diaphragm with spermicide or male partner’s use of male condom with spermicide. WOCBP are defined as those who are fertile, following menarche and until becoming postmenopausal unless permanently sterile; permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as amenorrhea for more than 24 consecutive months without an alternative medical cause; women on hormone replacement therapy must have a documented plasma follicle-stimulating hormone level >40 mIU/mL. All WOCBP must have a negative pregnancy test (urine, with positive results confirmed by serum) at Visits 1 and 3 (Screening and Day 1/Randomization).
NOTE: Prior to menarche, pregnancy testing and contraceptive use is not required. However, the patient and their parent/guardian must be advised that, immediately upon menarche, the patient will be required to begin pregnancy testing and, if deemed necessary by the Investigator, initiate contraceptive use. This requirement cannot be avoided.
8. Males must be surgically sterile (more than 3 months post-vasectomy) or must agree to the use of medically accepted methods of contraception that are considered highly reliable from Day 1/Randomization until 90 days after the last dose of study medication.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this ag
1. FSGS secondary to another condition.
2. Positive findings on serological tests of primary or secondary glomerular injury.
3. Indications of relapse from complete remission.
4. History of type 1 diabetes mellitus, uncontrolled type 2 diabetes mellitus (hemoglobin A1c [HbA1c] >8%), or nonfasting blood glucose
>180 mg/dL at screening.
5. Any organ transplantation, with the exception of corneal transplants.
6. Treatment with any of the prohibited concomitant medications.
7. Treatment with rituximab, cyclophosphamide, or abatacept within =3
months prior to screening. If a patient is taking other chronic
immunosuppressive medications, the dosage must be stable for =1
month prior to randomization.
8. Documented history of heart failure and/or previous hospitalization
for heart failure or unexplained dyspnea, orthopnea, paroxysmal
nocturnal dyspnea, ascites, and/or peripheral edema.
9. Clinically significant cerebrovascular disease and/or coronary artery
disease.
10. Hemodynamically significant valvular disease.
11. Jaundice, hepatitis, or known hepatobiliary disease (excluding asymptomatic cholelithiasis), or transaminase levels >2 times the upper limit of the normal range at screening.
12. Positive at screening for the human immunodeficiency virus (HIV) or
markers indicating acute or chronic hepatitis B (HBV) infection or
hepatitis C virus (HCV) infection.
13. History of malignancy other than adequately treated basal cell or
squamous cell skin cancer or cervical carcinoma within the past 2 years.
14. A screening hematocrit value <27% or hemoglobin value <9 g/dL.
15. A screening potassium value of >5.5 mEq/L.
16. Body mass index (BMI) >40.
17. Female patient is pregnant, breastfeeding, or planning to conceive
during the study.
18. Participation in a study of another investigational product within 28
days prior to screening.
19. Prior exposure to sparsentan.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method