Treatment Approaches for Alport Syndrome.
Michelle N Rheault
https://pubmed.ncbi.nlm.nih.gov/40938675Actively Recruiting
Phase 2
Age: 1Year - 17Years
All Genders
ID05003986
A Phase 2, Open-Label, Single-Arm Study to Evaluate Sparsentan Safety, Efficacy, and Pharmacokinetics in Children With Selected Proteinuric Glomerular Diseases
Led by Travere Therapeutics, Inc. · Updated on 2026-05-12
67
Participants Needed
47
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The trial investigates the safety, effectiveness, and tolerability of sparsentan in children aged 1 to under 18 years old who have specific proteinuric glomerular diseases, including Focal Segmental Glomerulosclerosis (FSGS), Minimal Change Disease (MCD), Immunoglobulin A Nephropathy (IgAN), IgA Vasculitis (IgAV), and Alport Syndrome (AS). The study is a phase 2, open-label, multicenter trial involving approximately 67 pediatric participants divided into three populations based on diagnosis and age groups. Participants receive sparsentan either as an oral suspension or tablets depending on their population group and age. Population 1 receives 800 mg sparsentan oral suspension, Population 2 receives 400 mg sparsentan oral suspension, and Population 3 receives 400 mg sparsentan tablets. The study spans 112 weeks, with pharmacokinetic evaluations at various time points specific to each population. The trial includes multiple cohorts based on age ranges within each population. During the study, participants will have regular visits for safety and efficacy assessments, including monitoring of adverse events and changes in proteinuria measured by urine protein/creatinine ratio. Pharmacokinetic samples are collected at scheduled visits. Other measures include urine albumin/creatinine ratio, estimated glomerular filtration rate, and remission status of proteinuria. The total participation time is approximately 108 weeks after the last patient visit, with ongoing safety monitoring and evaluations throughout the study period.
CONDITIONS
Brief Title
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
Who Can Participate
Age: 1Year - 17Years
All Genders
Eligibility Criteria
You may qualify if you...
- Participant or parent/legal guardian provides signed informed consent/assent.
- Estimated glomerular filtration rate (eGFR) is at least 30 mL/min/1.73 m2 at screening.
- Mean seated blood pressure is between the 5th and 95th percentile for sex and height.
- For Population 1: Age 1 to under 18 years; UP/C at least 1.5 g/g; diagnosed with FSGS or MCD by biopsy or genetic mutation.
- For Population 2: Age 2 to under 18 years; UP/C at least 0.6 g/g; diagnosed with IgAN, IgAV, or Alport Syndrome by biopsy or genetic testing.
- For Population 3: Age 8 to under 18 years; UP/C at least 1.0 g/g; biopsy-confirmed IgAN; weight at least 40 kg; on ACEI and/or ARB therapy for at least 12 weeks prior to screening.
You will not qualify if you...
- Weight less than 7.3 kg at screening.
- FSGS or MCD caused by viral infections, drug toxicity, or malignancies.
- IgA deposits due to secondary conditions like lupus or liver disease.
- Acute glomerular disease onset, biopsy, or relapse requiring new immunosuppressive treatment within 6 months before screening.
- Unstable chronic immunosuppressive medication use in the month before screening.
- Use of prohibited concomitant medications as defined by the protocol.
- History of organ transplantation except corneal transplants.
- History of moderate to severe heart failure or related symptoms.
- Significant cardiac valve or congenital vascular disease.
- Liver disease or elevated liver enzymes more than twice normal.
- History of cancer in the past 2 years.
- Low hemoglobin or hematocrit values at screening.
- High potassium level above 5.5 mEq/L at screening.
- Clinically significant abnormal lab values per investigator judgment.
- Allergy or hypersensitivity to sparsentan or its components.
- Female participants who are pregnant, breastfeeding, or not using reliable contraception if of childbearing potential.
- Participation in another study medication trial within 28 days prior to screening.
- Prior exposure to sparsentan.
- Inability to comply with study requirements.
- For Population 3: inability to swallow tablets whole.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Treatment
Duration - 112 weeks
Participants receive sparsentan according to their assigned population and age cohort to evaluate safety, efficacy, and pharmacokinetics.
Multiple visits across 112 weeks, including baseline, Day 2, Week 4, 8, 12, 24, 36, 48, 60, 72, 84, 96, and 108 visits depending on population and assessments
Follow-up
Duration - Up to 4 weeks after treatment ends
Participants are monitored for long-term safety and efficacy outcomes after treatment completion.
1 to 2 visits depending on participant status
Trial Site Locations
Total: 47 locations
1
Cedars-Sinai Medical Center
Los Angeles, California, United States, 90048
Actively Recruiting
2
Nemours Children's Hospital
Wilmington, Delaware, United States, 19803
Actively Recruiting
3
University of Miami, Leonard M. Miller School of Medicine
Miami, Florida, United States, 33136
Actively Recruiting
4
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
5
University of Iowa, Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
Withdrawn
6
Floating Hospital for Children at Tufts Medical Center
Boston, Massachusetts, United States, 02111
Withdrawn
7
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109-5008
Actively Recruiting
8
University of Minnesota, Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
9
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States, 64108
Actively Recruiting
10
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
Actively Recruiting
11
Jersey Shore University Medical Center
Neptune City, New Jersey, United States, 07753
Actively Recruiting
12
Cohen Children's Medical Center
New Hyde Park, New York, United States, 11042
Actively Recruiting
13
Fink Children's Ambulatory Care Center
New York, New York, United States, 10016
Withdrawn
14
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
15
Atrium Health Levine Children's Hospital
Charlotte, North Carolina, United States, 28203
Completed
16
Duke Molecular Physiology Institute
Durham, North Carolina, United States, 22710
Actively Recruiting
17
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
18
University of Oklahoma Health Sciences Center (OUHSC)
Oklahoma City, Oklahoma, United States, 73104
Actively Recruiting
19
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
20
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, United States, 19134
Withdrawn
21
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
22
UT Health - John P. and Kathrine G. McGovern Medical School
Houston, Texas, United States, 77030
Withdrawn
23
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
24
Uniklinik Köln, Klinik und Poliklinik für Kinder- und Jugendmedizin
Cologne, Germany, 50937
Actively Recruiting
25
Universitätsklinikum Hamburg-Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
26
Universitätsklinikum Heidelberg - Angelika Lautenschläger - Kinderklinik
Heidelberg, Germany, 69120
Actively Recruiting
27
Policlinico Bari Ospedale Pediatrico Giovanni XXIII
Bari, Italy, 70126
Actively Recruiting
28
IRCCS Istituto Giannina Gaslini
Genova, Italy, 16147
Actively Recruiting
29
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy, 20122
Actively Recruiting
30
Azienda Ospedale Università di Padova
Padova, Italy, 35128
Actively Recruiting
31
Ospedale Pediatrico Bambino Gesù
Roma, Italy, 00165
Actively Recruiting
32
Emma Kinderziekenhuis
Amsterdam, Netherlands, 1105 AZ
Actively Recruiting
33
Radboud Universitair Medisch Centrum
Nijmegen, Netherlands, 6525 GA
Withdrawn
34
Uniwersytecki Szpital Dziecięcy w Krakowie
Krakow, Poland, 30-663
Actively Recruiting
35
Instytut Centrum Zdrowia Matki Polki
Lodz, Poland, 93-338
Withdrawn
36
Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, Poland, 04-730
Withdrawn
37
Hospital Universitari Vall d'Hebrón
Barcelona, Spain, 08035
Actively Recruiting
38
Hospital Universitario 12 de Octubre
Madrid, Spain, 28041
Actively Recruiting
39
Hospital Universitario La Paz
Madrid, Spain, 28046
Actively Recruiting
40
Hospital Universitario Virgen del Rocío
Seville, Spain, 41013
Actively Recruiting
41
Drottning Silvias Barn- och Ungdomssjukhus
Gothenburg, Sweden, 416 85
Actively Recruiting
42
Karolinska Universitetssjukhuset Huddinge
Stockholm, Sweden, 141 86
Actively Recruiting
43
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children
Bristol, United Kingdom, BS2 8BJ
Actively Recruiting
44
NHS Greater Glasgow and Clyde, Royal Hospital for Children
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
45
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom, L12 2AP
Actively Recruiting
46
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom, WC1N3JH
Actively Recruiting
47
Manchester University NHS Foundation Trust
Manchester, United Kingdom, M13 9WL
Actively Recruiting
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Research Team
T
Travere Call Center
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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