An observational, non-interventional, multicenter, multi-national study of patients with Atypical Hemolytic-Uremic Syndrome (aHUS) (Alexion)
Principal Investigator: prof. Mekahli
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. Mekahli
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.
An international, multicentre, prospective, non-interventional observational registry for patients with X-linked hypophosphatemia (Kyowa Kirin)
Principal Investigator: prof. Mekahli
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. Mekahli
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.
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).
Multicentre, ambispective, observational, earl life study to assess the Quality of Life effectiveness of ER oral cysteamine therapy (Procysbi®) in Belgian patients suffering from nephropathic cystinosis (“PROREAL”)
Principal Investigator: prof. Mekahli
Nephropathic cystinosis is a rare autosomal disease characterized by a deficiency of the cystine lysosomal transporter protein, cystinosin, resulting in intracellular accumulation of cystine crystals in all tissues and organs, and subsequently leading to tissue damage; the most affected organs are kidney, liver, eye, thyroid gland, and brain.
Prevention of relapses with Levamisole as adjuvant therapy to corticosteroids in children with first episode of idiopathic nephrotic syndrome (LEARNS, academic study coordinated by AMC, Amsterdam, The Netherlands)
Principal Investigator: prof. Mekahli
Idiopathic nephrotic syndrome (INS) is a relatively rare disease, predominantly in children and adolescents. After initial treatment with corticosteroids, the vast majority achieves remission. Unfortunately, relapse rates are high (80%), resulting in repeated and high doses of corticosteroids that have major physical and psychological side effects. Previous randomized controlled studies (RCTs) showed promising results when levamisole, an antihelminthic drug, was added as adjuvant therapy to corticosteroids in children with frequently relapsing INS in reducing the occurrence of relapses.