Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders...
BCR-JAK2 Fusion Protein ExpressionBlasts 20 Percent or Less of Peripheral Blood White Cells11 moreThis phase II trial studies how well ruxolitinib works in treating patients with hypereosinophilic syndrome or primary eosinophilic disorders.
S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis
AmorphousEosinophilic13 moreThis phase II trial studies how well isatuximab works in treating patients with primary amyloidosis that has come back or does not respond to treatment. Monoclonal antibodies, such as isatuximab, may interfere with the ability of cancer cells to grow and spread.
Nivolumab in Treating Patients With Primary Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis,...
HepatomegalyMyelofibrosis Transformation in Essential Thrombocythemia4 moreThis phase II trial studies how well nivolumab works in treating patients with primary myelofibrosis, post-essential thrombocythemia myelofibrosis, or post-polycythemia vera myelofibrosis. Immunotherapy with monoclonal antibodies, such as nivolumab, may help the body's immune system attack the cancer, and may interfere with the ability of tumor cells to grow and spread.
Lanreotide as Treatment of Polycystic Livers
Polycystic Liver DiseaseHepatomegaly3 moreTo assess the efficacy of lanreotide in controling total liver volume in patients with polycystic livers this study will be performed. A minimum of 38 patients will be recruited and randomized (1:1) to receive either verum or placebo. Lanreotide is already used in other disease states and found to be safe and non-toxic.
Octreotide in Severe Polycystic Liver Disease
Polycystic KidneyAutosomal Dominant6 moreThis study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection or liver transplantation. A total of 42 patients will be recruited -14 who will receive placebo and 28 the study drug. Preliminary evidence indicates that this drug is safe and non-toxic in other disease states. Treatment with this drug holds promise not only for individuals with liver involvement, but also for many more patients with polycystic kidney disease.
Open-Label Extension of LOCKCYST Trial
Polycystic Liver DiseaseHepatomegaly3 morePositive data originating from two polycystic liver patients treated with somatostatin analogues, showed a volume reduction of 38.3% and 14.9%. These two patients had complicated polycystic livers and no other therapeutic options were available. Patients who participated in LOCKCYST trial are able to benefit from active treatment. Participants will be actively treated for 24 weeks.
Biomarker for Gaucher Disease: BioGaucher (BioGaucher)
SplenomegalyHepatomegalyDevelopment of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Gaucher Disease from blood (plasma)
Biomarker for Sly Disease (MPS VII) (BioSly)
Developmental DelaySkeletal Abnormalities2 moreDevelopment of a new MS-based biomarker for the early and sensitive diagnosis of Sly disease from blood (plasma)
Biomarker for Farber Disease (BioFarber)
Farber's LipogranulomatosisCeramidase Deficiency2 moreDevelopment of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Farber disease from the blood
Prevalence of Hepatomegaly in Children and Adolescents With Type 1 Diabetes Mellitus
HepatomegalyTo assess the prevalence of hepatomegaly in children and adolescents with type 1 diabetes mellitus, and to find out it᾿s underlying etiology.