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Wolman Disease clinical trials

View clinical trials related to Wolman Disease.

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NCT ID: NCT04652713 Completed - Obesity Clinical Trials

Breakfast for Young Women

GoDag
Start date: November 24, 2020
Phase: N/A
Study type: Interventional

Investigators will in a crossover study test the health effects of eating a dairy-based protein-rich breakfast, isocaloric carbohydrate-rich breakfast or no breakfast in young overweight women. Determination of satiety and hunger using visual analog scores (VAS), collection of blood samples and 2x 24 hours food logs will be collected.

NCT ID: NCT02372513 Completed - Clinical trials for Cholesteryl Ester Storage Disease

National Lysosomal Acid Lipase Deficiency Study

LAL-D
Start date: January 2015
Phase: N/A
Study type: Observational

Cholesteryl Ester Storage Disease (CESD) is an autosomal recessive lysosomal storage disorder (LSD) caused by mutations in the lysosomal acid lipase gene (LIPA) that markedly reduce lysosomal acid lipase (LAL) activity, leading to the accumulation of lipids, predominately cholesteryl esters and triglycerides, in various tissues and cell types. In the liver, accumulation of lipids leads to diffuse microvesicular steatosis, which progresses to fibrosis and ultimately, to micronodular cirrhosis. Patients typically present with hepatomegaly, liver dysfunction, hepatic failure and type II hyperlipidemia. Although hepatosteatosis is a typical finding, the liver biopsy diagnosis may be misclassified as non-alcoholic fatty liver disease, non-alcoholic steatohepatitis or cryptogenic liver disease. Biopsy and radiological findings are not considered diagnostic, but help to suspicion of CESD. The definitive diagnosis is based on deficient LAL activity and/or LIPA gene mutations. CESD is pan-ethnic, however, the disease incidence is unknown. The estimated incidence of the disease indicates that CESD should be largely underdiagnosed especially in European patients. Elevation of serum transaminases, and hepatomegaly are early indications of liver impairment. Therefore, CESD should be considered as a differential diagnosis in liver disease of unknown origin. To data, there is no study which evaluated the frequency of CESD in children with unexplained transaminase elevation and/or organomegaly and/or chronic liver disease. The aim of this prospective, multicenter and cross-sectional study is to investigate frequency of CESD in children with unexplained transaminase elevation and/or and/or chronic liver disease and to identify demographic and clinical features of CESD.

NCT ID: NCT02112994 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Start date: June 24, 2014
Phase: Phase 2
Study type: Interventional

This study evaluated the safety and efficacy of sebelipase alfa in a broad population of participants with lysosomal acid lipase deficiency (LAL-D).

NCT ID: NCT01884220 Completed - Wolman Disease Clinical Trials

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Start date: November 2010
Phase: N/A
Study type: Observational

The purpose of this study are: to characterize and understand the natural history of disease progression in WD and CESD, and to provide historical controls for WD and CESD for developing clinical treatment trials. The hypothesis is that the variability and clinical progression in WD and CESD is large and represents a continuum of severities from a lethal infantile to near normal adults with only "fatty livers".

NCT ID: NCT01757184 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

ARISE
Start date: January 22, 2013
Phase: Phase 3
Study type: Interventional

This Phase 3 study evaluated the efficacy and safety of 1 milligram/kilogram (mg/kg) intravenous (IV) infusions of SBC-102 (sebelipase alfa) administered every other week (qow) in participants with late onset lysosomal acid lipase deficiency (LAL-D) (cholesteryl ester storage disease [CESD]). Late-onset LAL-D is an underappreciated cause of cirrhosis, liver failure and dyslipidemia. There is currently no standard treatment for LAL-D other than supportive care. Enzyme replacement therapy may be a potential new treatment option for LAL-D participants.

NCT ID: NCT01586455 Completed - Clinical trials for Myelodysplastic Syndrome

Human Placental-Derived Stem Cell Transplantation

HPDSC
Start date: April 2013
Phase: Phase 1
Study type: Interventional

The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.

NCT ID: NCT01528917 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Start date: June 2011
Phase: N/A
Study type: Observational

This is a Natural History study to characterize key aspects of the clinical course of late onset Lysosomal Acid Lipase (LAL) Deficiency/ Cholesteryl Ester Storage Disease (CESD).

NCT ID: NCT01488097 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency

Start date: December 12, 2011
Phase: Phase 2
Study type: Interventional

This was an extension study to Study LAL-CL01 (NCT01307098). The primary objective of the study was to evaluate the long-term safety and tolerability of sebelipase alfa in participants with liver dysfunction due to lysosomal acid lipase (LAL) deficiency.

NCT ID: NCT01371825 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Start date: May 4, 2011
Phase: Phase 2/Phase 3
Study type: Interventional

This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency. Eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.

NCT ID: NCT01358370 Completed - Clinical trials for Lysosomal Acid Lipase Deficiency

A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype

Start date: November 2010
Phase: N/A
Study type: Observational

This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.