An observational, non-interventional, multicenter, multi-national study of patients with Atypical Hemolytic-Uremic Syndrome (aHUS) (Alexion)

Principal Investigator: Prof. Elena Levtchenko

aHUS is a potentially fatal condition characterized by thrombotic microangiopathy and acute kidney injury due to uncontrolled activation of the alternative pathway of complement. Treatment with eculizumab inhibits complement activation and improves prognosis of the patients. In the current study data on long-term follow-up of aHUS patients is collected.

An Observational Registry Study to Evaluate the use and Safety of Cinacalcet Among Paediatric Patients With Secondary Hyperparathyroidism (Amgen)

Principal Investigator: Prof. Elena Levtchenko

Secondary hyperparathyroidism is a frequent complication of chronic kidney disease which might be resistant to standard treatment with active vitamin D analogues. Cinacalcet is an agonist of Calcium-Sensint Receptor which is used to treat therapy-resistant hyperparathyroidism.


In deze studie worden gegevens verzameld van kinderen (3-18j) met SHPT die cinacalcet gebruiken als behandeling omdat er een gebrek is aan real-world data van pediatrisch cinacalcet gebruik. Daarnaast zijn er ook onzekerheden over de incidentie, risico factoren en therapeutisch response voor het behandelen van hypocalcaemia bij kinderen in routine klinische praktijk.

An international, multicentre, prospective, non-interventional observational registry for patients with X-linked hypophosphatemia (Kyowa Kirin)

Principal Investigator: Prof. Elena Levtchenko

X-linked hypophosphatemia is a systemic disorder due to mutations in PHEX gene leading to vitamin D resistant rickets, growth retardation and other severe bone complications due to high levels of FGF23. Data on the natural history of this disorder are limited.

Double-blind, randomized, placebo controlled, non-inferiority intervention
study to REduce STEroids in Relapsing Nephrotic syndrome (Restern, academic study coordinated by UMC Radboud, Nijmegen, The Netherlands)

Principal Investigator: Prof. Elena Levtchenko

Idiopathic nephrotic syndrome is a common condition of childhood which is treated with corticosteroid hormones. The treatment schedules with steroids are empiric and vary between different countries. The aim of this study is to test short versus standard steroid-regimen for the treatment of relapse of nephrotic syndrome.

Study of Mirabegron in children with hyperactive bladder (Astellas)

Principal Investigator: Dr. Herman

Children with an overactive bladder (OAB) have a sudden urge to urinate and often have trouble holding it up. The bladder starts to contract when there is only a small amount of urine in the bladder. These children urinate more often in smaller amounts, depending on fluid intake. It is often difficult for these children to become toilet trained at night. The cause is often unclear. There are different types of treatment: pelvic physiotherapy, urotherapy, continence nurse coaching, and medication such as anticholinergics.

Astellas: A Phase 3, Double-blind, Randomized, Multicenter, Parallel-Group, Placebo-controlled Sequential Dose Titration Study to Evaluate Efficacy, Safety, and Pharmacokinetics of Mirabegron in Pediatric Subjects from 5 to < 18 Years of Age with Overactive Bladder.

Antibiotic Prophylaxis and Renal Damage In Congenital abnormalities of the kidney and urinary Tract (academic study coordinated by Giovanni Montini and the ESCAPE study group)

Principal Investigator: Prof. Mekahli

Bacterial urinary tract infections (UTI) are common in young children. In the acute phase, kidney parenchymal damage can lead to further scarring, the development of chronic kidney damage and hypertension. This risk is largely important for hypoplastic kidneys, which are often found in combination with high-grade vesicoureteral reflux (VUR).

Last edit: 7 October 2020