Wolman Disease: all 33 studies
Page 1 of 1. Open to participants first (5), then studies with results posted, then the rest; newest registration first within each.
- The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary IncontinenceRecruitingNCT06287658Not applicable
- Registry of Patients Diagnosed With Lysosomal Storage DiseasesRecruitingNCT05619900
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT05368038
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)RecruitingNCT04532047Phase 1
- Lysosomal Acid Lipase (LAL) Deficiency RegistryRecruitingNCT01633489
- Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyTerminatedNCT02193867Phase 2Results posted
- Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT02112994Phase 2Results posted
- Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT01757184Phase 3Results posted
- Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal DisordersCompletedNCT01626092Not applicableResults posted
- Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase DeficiencyCompletedNCT01488097Phase 2Results posted
- Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyCompletedNCT01371825Phase 2/3Results posted
- Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT01307098Phase 1/2Results posted
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTerminatedNCT00668564Phase 2Results posted
- HSCT for High Risk Inherited Inborn ErrorsCompletedNCT00383448Phase 2Results posted
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/3Results posted
- Lysosomal Acid Lipase Deficiency in Risk GroupsTerminatedNCT07455864
- Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
- Prevalence and Risk Factors of Women Mental Health DisordersStatus unknownNCT04792671
- Maximal Fat Oxidation and Fuel Use During ExerciseCompletedNCT04678713Not applicable
- Breakfast for Young WomenCompletedNCT04652713Not applicable
- Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)Status unknownNCT03984149
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
- Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe ObesityStatus unknownNCT03564002
- Screening for Lysosomal Acid Lipase DeficiencyTerminatedNCT02926872
- Biomarker for Wolman Disease (BioWolman)WithdrawnNCT02383641
- An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase DeficiencyNo longer availableNCT02376751
- A Study to Identify and Characterize LAL-D Patients in High-risk PopulationsTerminatedNCT02345421
- Wolman/CESD Natural History Chart Review and Longitudinal Follow-UpCompletedNCT01884220
- Identification of Undiagnosed Lysosomal Acid Lipase DeficiencyStatus unknownNCT01716728Not applicable
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 1
- An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease PhenotypeCompletedNCT01528917
- A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman PhenotypeCompletedNCT01358370
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT00005900