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Safety, efficacy and pharmacokinetics of sparsentan in pediatric subjects with selected kidney diseases.
EU CTIS ID: 2023-505497-14-00
What this study is testing
1. Evaluate the safety and tolerability of sparsentan oral suspension (Population 1 and Population 2) and tablets (Population 3). 2. Assess changes in proteinuria after once-daily dosing of sparsentan oral suspension and tablets over 108 weeks.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. For All Subjects (All Three Populations) - The subject or parent/legal guardian is willing and able to provide signed informed consent/assent, the subject is willing to provide assent before any screening procedures.
- 3. For Population 3- Subject weighs ≥40 kg
- 4. For Population 3- The subject has been on ACEI and/or ARB therapy for at least 12 weeks prior to screening.
- 2. For All Subjects (All Three Populations) - The subject has an eGFR ≥30 mL/min/1.73 m2 at screening.
- 3. For All Subjects (All Three Populations) - The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height.
- 1. For Population 1 - Male or female ≥1 year at screening to <18 years of age at Day 1.
You likely can't join if
- For All Subjects (All Three Populations) 1. Weighs <7.3 kg at screening
- Female of childbearing potential who do not agree to use 1 highly reliable method of contraception from 7 days before the first dose of the study medication until 28 days after the last dose of study medication and have a - serum pregnancy test at screening and a - urine pregnancy, with + results confirmed by serum, at every study visit
- Has participated in a study of another study medication within 28 days before screening or plans to participate in such a study during the course of this study
- Has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies
- The subject has had prior exposure to sparsentan
- The subject or parent/legal guardian is unable to adhere to the requirements of the study
See the full eligibility criteria
Who can join
- 1. For All Subjects (All Three Populations) - The subject or parent/legal guardian is willing and able to provide signed informed consent/assent, the subject is willing to provide assent before any screening procedures.
- 3. For Population 3- Subject weighs ≥40 kg
- 4. For Population 3- The subject has been on ACEI and/or ARB therapy for at least 12 weeks prior to screening.
- 2. For All Subjects (All Three Populations) - The subject has an eGFR ≥30 mL/min/1.73 m2 at screening.
- 3. For All Subjects (All Three Populations) - The subject has a mean seated blood pressure between the 5th and 95th percentile for sex and height.
- 1. For Population 1 - Male or female ≥1 year at screening to <18 years of age at Day 1.
- 2. For Population 1 - Has a UP/C ≥1.5 g/g (170 mg/mmol) at screening AND one of the following: • Kidney biopsy-proven FSGS or MCD histological patterns and clinical presentation consistent with primary FSGS or MCD and qualifying proteinuria at screening despite history or ongoing treatment with corticosteroids and/or other immunosuppressive disease-modifying agents • Documentation of a genetic mutation in a podocyte protein associated with FSGS or MCD. Subjects with a documented podocytic mutation do not require kidney biopsy • Kidney biopsy-proven FSGS histological pattern with medical history and clinical presentation consistent with maladaptive cause of the lesion.
- 1. For Population 2 - Male or female ≥2 years to <18 years of age at Day 1 (Baseline).
- 2.For Population 2 - Has UP/C ≥0.6 g/g (68 mg/mmol) at screening AND one of the following: • Kidney biopsy-confirmed IgAN, IgAV or AS • Diagnosis of AS by genetic testing (pathogenic X-linked COL4A5 mutation OR autosomal-recessive mutations in both alleles of COL4A3 and/or COL4A4 OR autosomal-dominant COL4A3 and/or COL4A4 and digenic mutations [ie, simultaneous mutations in 2 of the COL4A3, COL4A4, and COL4A5 genes].
- 1. For Population 3- Male or female ≥8 years at screening and <18 years of age at Day 1 (Baseline).
- 2. For Population 3- The subject has UP/C ≥1.0 g/g (113 mg/mmol) at screening AND has kidney biopsy-confirmed IgAN
What rules you out
- For All Subjects (All Three Populations) 1. Weighs <7.3 kg at screening
- Female of childbearing potential who do not agree to use 1 highly reliable method of contraception from 7 days before the first dose of the study medication until 28 days after the last dose of study medication and have a - serum pregnancy test at screening and a - urine pregnancy, with + results confirmed by serum, at every study visit
- Has participated in a study of another study medication within 28 days before screening or plans to participate in such a study during the course of this study
- Has FSGS or MCD histological pattern secondary to viral infections, drug toxicities, or malignancies
- The subject has had prior exposure to sparsentan
- The subject or parent/legal guardian is unable to adhere to the requirements of the study
- Has immunoglobulin A (IgA) glomerular deposits not in the context of primary IgAN or IgAV (ie, secondary to another condition
- The subject has had an acute onset or presentation of glomerular disease or a diagnostic biopsy or a relapse of glomerular disease requiring new or different class of immunosuppressive treatment within 6 months before screening
- Taking chronic immunosuppressive medications and not on a stable dose for ≥1 month before screening
- Requires any of the prohibited concomitant medications
- Has undergone any organ transplantation, with the exception of corneal transplants
- Has clinically significant congenital vascular disease
- Has a documented history of congenital or acquired heart failure and/or previous hospitalization for heart failure or unexplained dyspnea, orthopnea, paroxysmal nocturnal dyspnea, ascites, and/or peripheral edema
- Has hemodynamically significant cardiac valvular disease
- For Population 3 - The subject is unable to swallow the study medication tablets whole.
- Has jaundice, hepatitis, or known hepatobiliary disease, or ALT and/or AST >2 times the UL of normal at screening
- Has a history of malignancy within the past 2 years
- Has a screening hematocrit <27% (0.27 L/L) or a hemoglobin value <9 g/dL (90 g/L)
- Has a screening potassium value >5.5 mEq/L (5.5 mmol/L)
- Has any abnormal clinical laboratory screening values that are considered to be clinically significant
- Has a history of allergy to any Ang II antagonist or ERA, including sparsentan, or has a hypersensitivity to any of the excipients
- Female is pregnant, plans to become pregnant during the course of the study, or is breastfeeding
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.