Niemann-Pick Diseases clinical trials
The registry lists 28 studies for Niemann-Pick Diseases across more than 10 countries. 7 are recruiting or about to start. 10 of them are mainly about Niemann-Pick Diseases (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-15. Counting its 2 narrower conditions too, such as Niemann-Pick Disease, Type C: 71 studies, 14 open.
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Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
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Who runs them
Sanofi (4) · CENTOGENE GmbH Rostock (2) · O & O Alpan LLC (2) · Alexion Pharmaceuticals, Inc. (2) · Wladimir MAUHIN, Dr (2) · Genzyme, a Sanofi Company (2) · Fundación Española de Hematología y Hemoterapía (2) · New York Medical College (1)
What is being tested here
Drugs under test in Niemann-Pick Diseases studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| Olipudase alfa | 0 | 0 | 2 |
| Sebelipase alfa | 0 | 1 | 2 |
| Busulfan | 0 | 1 | 1 |
| Cyclophosphamide | 0 | 1 | 1 |
| Antilymphocyte Serum | 0 | 1 | 1 |
| Miglustat | 0 | 0 | 1 |
Who these studies take
Of the 6 Niemann-Pick Diseases studies recruiting now, 3 (50%) set no upper age limit and 2 (33%) stop at 75 or younger. 4 (67%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (17%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 2 (33%) | 2 (33%) |
| 18 | 4 (67%) | 4 (67%) |
| 30 | 5 (83%) | 5 (83%) |
| 40 | 5 (83%) | 5 (83%) |
| 50 | 5 (83%) | 5 (83%) |
| 65 | 4 (67%) | 4 (67%) |
| 75 | 4 (67%) | 4 (67%) |
| 85 | 4 (67%) | 4 (67%) |
Across all 28 studies, recruiting or not: 36% stop at 75 or younger and 36% at 65; 79% take children; 0% are for women only and 0% for men only; 18% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Started recruiting2
Studies
Recruiting first, then most recently updated. Showing 28 of 28. search them.
- Study of the Prevalence of Acid Sphingomyelinase Deficiency/Niemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung DiseaseRecruitingNCT06869499Not applicable1 country
- National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and ChildrenRecruitingNCT069852121 country
- A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)RecruitingNCT061925761 country
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT053680381 country
- Retinal Hyperspectral Imaging in Neurodegenerative DiseasesRecruitingNCT07545473Not applicable1 country
- GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple MyelomaRecruitingNCT059925321 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in FranceCompletedNCT06949358Phase 21 country
- PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"CompletedNCT056411031 country
- Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)Active, not recruitingNCT072748261 country
- A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged SpleenCompletedNCT048459581 country
- Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in FranceCompletedNCT053592761 country
- Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic DisordersTerminatedNCT01372228Phase 1/21 country
- Biomarker for Niemann Pick Type C Disease (BioNPC)WithdrawnNCT013066043 countries
- Human Placental-Derived Stem Cell TransplantationCompletedNCT01586455Phase 11 country
- Induced Pluripotent Stem Cells for Niemann Pick DiseaseCompletedNCT038837501 country
- Molecular and Cellular Mechanisms of Lysosomal Storage DiseasesStatus unknownNCT020003101 country
- Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-PickCompletedNCT044698941 country
- Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyTerminatedNCT02193867Phase 24 countries
- Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyCompletedNCT01371825Phase 2/35 countries
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/31 country
- Saccadic Eye Movements in Patients With Niemann-Pick Type C DiseaseCompletedNCT00316498Phase 12 countries
- Investigating Lysosomal Storage Diseases in Minority GroupsStatus unknownNCT021202351 country
- Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsCompletedNCT00730314Phase 1/21 country
- Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency PatientsCompletedNCT01722526Phase 12 countries
- Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)TerminatedNCT00410566Phase 11 country
- PET Scan of Brain Metabolism in Relation to Age and DiseaseCompletedNCT000019721 country
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT000059001 country