Actively Recruiting
A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)
Led by Otsuka Pharmaceutical Development & Commercialization, Inc. · Updated on 2026-02-17
20
Participants Needed
23
Research Sites
290 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
To evaluate the safety of tolvaptan in pediatric subjects with ARPKD
CONDITIONS
Official Title
A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 28 days to less than 18 years with clinical features consistent with ARPKD
- Parent or legal guardian able to provide written informed consent
- Participant able to provide written assent if required by local laws
- Ability to comply with all trial requirements as judged by the investigator
You will not qualify if you...
- Premature birth at or before 32 weeks gestational age for infants 28 days to less than 12 weeks old
- Anuria or receiving any form of renal replacement therapy or history of kidney transplant
- Syndromic conditions associated with renal cysts other than ARPKD
- Abnormal liver function tests with ALT or AST above 1.2 times the upper limit of normal
- Presence of splenomegaly or portal hypertension
- Parents with renal cystic disease
- Use of chronic diuretics that cannot be adjusted after starting tolvaptan
- Inability to monitor fluid balance
- Risk or presence of sodium and potassium electrolyte imbalances
- Risk or presence of significant hypovolemia
- Clinically significant anemia
- Platelet count below 50,000 per microliter
- Severe systolic dysfunction with ejection fraction below 14%
- Serum sodium levels below 130 mmol/L or above 145 mmol/L
- Use of other experimental medications
- Requirement for ventilator support
- Use of medications inducing CYP3A4
- Active infections requiring treatment that interferes with study drug dosing
- Females who are breastfeeding or pregnant
- History of substance abuse within the last 6 months
- Bladder dysfunction or difficulty voiding
- Use of vasopressin agonists like desmopressin
- History of persistent noncompliance with important medical therapies
- Use of therapies likely to affect PKD cysts such as tolvaptan or similar agents
- Received or scheduled for liver transplant
- History of cholangitis within last 6 months
- Clinically significant portal hypertension findings such as varices or hypersplenism indicated by thrombocytopenia
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 23 locations
1
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
2
Emory University Hospital
Atlanta, Georgia, United States, 30322
Withdrawn
3
Northwestern University Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Neonatology
Chicago, Illinois, United States, 60611
Withdrawn
4
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202-5119
Withdrawn
5
Children's Hospital - New Orleans
New Orleans, Louisiana, United States, 70118
Withdrawn
6
Johns Hopkins Pediatric Specialty Clinic
Baltimore, Maryland, United States, 21287
Actively Recruiting
7
C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109-5000
Actively Recruiting
8
Mayo Clinic - Rochester
Rochester, Minnesota, United States, 55905
Actively Recruiting
9
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
Actively Recruiting
10
Cleveland Clinic
Cleveland, Ohio, United States, 44195
Actively Recruiting
11
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15213
Withdrawn
12
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Withdrawn
13
Université Catholique De Louvain And Cliniques St Luc
Brussels, Brussels Capital, Belgium, 1200
Actively Recruiting
14
Universitair Ziekenhuis Gent
Ghent, Oost-Vlaanderen, Belgium, 9000
Actively Recruiting
15
UZ Leuven
Leuven, Vlaams Brabant, Belgium, 3000
Actively Recruiting
16
Universitätsklinikum Köln
Cologne, North Rhine-Westphalia, Germany, 50937
Actively Recruiting
17
Instytut "Pomnik - Centrum Zdrowia Dziecka"
Warsaw, Masovian Voivodeship, Poland, 04-730
Withdrawn
18
Uniwersytecki Dzieciecy Szpital Kliniczny im. L. Zamenhofa
Bialystok, Poland, 15-274
Withdrawn
19
Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)
Esplugues de Llobregat, Barcelona, Spain, 8950
Actively Recruiting
20
Hospital Universitari Parc Tauli
Sabadell, Barcelona, Spain, 08208
Withdrawn
21
Hospital Universitari Vall D Hebron
Barcelona, Spain, 8035
Actively Recruiting
22
Hospital Universitario Virgen del Rocío Avenida Manuel Siurot
Seville, Spain, 41013
Withdrawn
23
Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom, WC1N 3JH
Actively Recruiting
Research Team
O
Otsuka Call Center
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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