15 completed, 6 unknown, 5 terminated, 3 recruiting. 8 Phase 2, 5 Not applicable, 3 Phase 1, 3 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Completed 6, Terminated 2 |
| Not applicable | 5 | Completed 3, Unknown 2 |
| Phase 1 | 3 | Completed 2, Recruiting 1 |
| Phase 3 | 3 | Completed 3 |
Follow Wolman Disease: new trials by email Literature Review of Wolman Disease trials Research Report on Wolman Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Lysosomal Acid Lipase Deficiency in Risk Groups Lysosomal Acid Lipase Deficiency | Terminated | AstraZeneca | 25 | 2026-02-25 | 2026-07-08 | |
| The Effect of Kegel Exercise and Ba Duan Jin Applications on Premenopausal Women With Urinary Incontinence Training on Kegel exercise and Ba Duan Jin applications online, training on Kegel exercises and Ba Duan Jin applications after the research is completed - Urinary Incontinence, Menopause | Not applicable | Unknown | Ondokuz Mayıs University | 60 | 2024-04-01 | 2024-04-03 |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Registry of Patients Diagnosed With Lysosomal Storage Diseases There is no intervention - Mucopolysaccharidosis I, Mucopolysaccharidosis II | Recruiting | University of California, San Francisco | 250 | 2022-05-31 | 2026-04-08 | |
| PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) Aldurazyme (laronidase) - MPS I, MPS II | Phase 1 | Recruiting | University of California, San Francisco | 10 | 2021-07-01 | 2026-03-17 |
| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program Confirmatory Testing - Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2 | Enrolling by invitation | Albert Einstein College of Medicine | 100000 | 2021-05-10 | 2026-07-28 | |
| Prevalence and Risk Factors of Women Mental Health Disorders Sociodemographic variables, Family affluence scale (FAS), Edinburgh postnatal depression scale (EPDS) - Psychiatric Problem, Wolman Disease | Unknown | Assiut University | 200 | 2021-03-01 | 2021-03-11 | |
| Breakfast for Young Women Protein-rich breakfast, Carbohydrate-rich breakfast, No breakfast - Obesity, Wolman Disease | Not applicable | Completed | University of Aarhus | 30 | 2020-11-24 | 2024-05-09 |
| Maximal Fat Oxidation and Fuel Use During Exercise investigate the nutritional determinants of MFO in moderate to well-train individuals, Investigate the physiological determinants of MFO in moderate to well-train individuals - Obesity | Not applicable | Completed | University of Copenhagen | 40 | 2020-01-01 | 2024-02-15 |
| Early Check: Expanded Screening in Newborns Confirmatory Testing - Spinal Muscular Atrophy, Fragile X Syndrome | Active not recruiting | RTI International | 30000 | 2018-10-15 | 2026-07-01 | |
| Biomarker for Wolman Disease (BioWolman) Acid Lipase Deficiency, Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type | Withdrawn | CENTOGENE GmbH Rostock | 2018-08-20 | 2023-02-13 | ||
| Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects) Observational study - Lysosomal Acid Lipase Deficiency | Unknown | Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) | 1000 | 2017-09-01 | 2022-07-29 | |
| Screening for Lysosomal Acid Lipase Deficiency There is no intervention in the study - Lysosomal Acid Lipase Deficiency | Terminated | Alexion Pharmaceuticals, Inc. | 22 | 2016-11-01 | 2017-06-16 | |
| Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity Lysosomal Acid Lipase Deficiency, Obesity | Unknown | University Of Perugia | 60 | 2016-10-01 | 2020-03-09 | |
| A Study to Identify and Characterize LAL-D Patients in High-risk Populations Lysosomal Acid Lipase Deficiency | Terminated | Alexion Pharmaceuticals, Inc. | 640 | 2014-12-01 | 2016-05-24 | |
| Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Completed | Alexion Pharmaceuticals, Inc. | 31 | 2014-06-24 | 2019-12-04 |
| Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency Sebelipase Alfa - Lysosomal Acid Lipase Deficiency | Phase 2 | Terminated | Alexion Pharmaceuticals, Inc. | 10 | 2014-06-06 | 2019-11-18 |
| Lysosomal Acid Lipase (LAL) Deficiency Registry Lysosomal Acid Lipase Deficiency, Cholesterol Ester Storage Disease | Recruiting | Alexion Pharmaceuticals, Inc. | 300 | 2013-05-30 | 2026-03-06 | |
| Human Placental-Derived Stem Cell Transplantation Human Placental Derived Stem Cell - Mucopolysaccharidosis I, Mucopolysaccharidosis VI | Phase 1 | Completed | New York Medical College | 43 | 2013-04-01 | 2022-10-25 |
| Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency Sebelipase Alfa, Placebo - Lysosomal Acid Lipase Deficiency | Phase 3 | Completed | Alexion Pharmaceuticals, Inc. | 66 | 2013-01-22 | 2020-12-29 |
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Cholesterol Ester Storage Disease (12) · Leukodystrophy, Metachromatic (9) · Lysosomal Storage Diseases (9) · Mucopolysaccharidosis VI (8) · Gaucher Disease (7) · Leukodystrophy, Globoid Cell (7) · Adrenoleukodystrophy (6) · Metabolism, Inborn Errors (6) · Mucopolysaccharidosis I (6) · Mucopolysaccharidosis VII (6) · Niemann-Pick Diseases (6) · Mucopolysaccharidosis IV (5) · Fucosidosis (4) · Gangliosidosis, GM1 (4)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (33) · Nutritional and Metabolic Diseases (33) · Infant, Newborn, Diseases (32) · Lipid Metabolism Disorders (32) · Lipid Metabolism, Inborn Errors (32) · Lipidoses (32) · Genetic Diseases, Inborn (31) · Metabolic Diseases (31) · Metabolism, Inborn Errors (27) · Lysosomal Storage Diseases (24) · Cholesterol Ester Storage Disease (21) · Brain Diseases (13) · Brain Diseases, Metabolic (13) · Brain Diseases, Metabolic, Inborn (13) · Central Nervous System Diseases (13)
Sebelipase alfa (8) · Alemtuzumab (3) · Bone Marrow Transplantation (2) · Busulfan (2) · Clofarabine (2) · Cyclophosphamide (2) · Hematopoietic Stem Cell Transplantation (2) · Melphalan (2) · Stem Cell Transplantation (2) · Whole-Body Irradiation (2) · Antilymphocyte Serum (1) · Cyclosporine (1) · GAA protein, human (1) · GALNS protein, human (1) · Hydroxyurea (1)
Alexion Pharmaceuticals, Inc. (12) · Masonic Cancer Center, University of Minnesota (4) · University of California, San Francisco (2) · Albert Einstein College of Medicine (1) · Assiut University (1) · AstraZeneca (1) · CENTOGENE GmbH Rostock (1) · Centre Hospitalier Universitaire de Liege (1) · Children's Hospital Medical Center, Cincinnati (1) · Fairview University Medical Center (1) · Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) (1) · Massachusetts General Hospital (1) · New York Medical College (1) · Ondokuz Mayıs University (1) · RTI International (1)
United States (23) · United Kingdom (9) · Italy (8) · France (7) · Canada (5) · Czechia (5) · Germany (4) · Russia (4) · Australia (3) · Belgium (3) · Croatia (3) · Denmark (3) · Mexico (3) · Spain (3) · Turkey (Türkiye) (3)
Paul Orchard, MD (3) · Curt Scharfe, MD, PhD (1) · Florian Abel, MD (1) · Gregory A Grabowski, MD (1) · K. Scott Baker (1) · Katherine B Sims, MD (1) · Laurent Servais (1) · Maurizio Averna (1) · Melissa Wasserstein, MD (1) · Mette Hansen, Assoc Prof (1) · Mitchell S Cairo, MD (1) · Peter Bauer, Prof. (1)
PMID 21586746, PMID 23348766, PMID 15324318, PMID 19948615, PMID 20872155, PMID 21637107, PMID 23510062, PMID 24993530, PMID 25896571, PMID 26252914, PMID 28320214, PMID 28374935, PMID 28657663, PMID 30337566, PMID 31249784, PMID 31315600, PMID 32102934, PMID 32657505, PMID 34774639, PMID 35142618, PMID 35442238, PMID 37222260, PMID 37955580, PMID 39891377, PMID 40781810, PMID 9918480
2019 (1), R01HD073292 (1), U54NS065768 (1)