Sphingolipidoses clinical trials
The registry lists 23 studies for Sphingolipidoses across more than 10 countries. 5 are recruiting or about to start. 2 of them are mainly about Sphingolipidoses (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-06. Counting its 12 narrower conditions too, such as Fabry Disease: 560 studies, 119 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
National Center for Research Resources (NCRR) (3) · Masonic Cancer Center, University of Minnesota (3) · University of California, San Francisco (2) · 4D Molecular Therapeutics (2) · Bellicum Pharmaceuticals (1) · Amicus Therapeutics (1) · Fairview University Medical Center (1) · Shire (1)
What is being tested here
Drugs under test in Sphingolipidoses 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 |
|---|---|---|---|
| Alemtuzumab | 0 | 0 | 2 |
| Cyclophosphamide | 0 | 0 | 2 |
| Busulfan | 0 | 0 | 2 |
| Cyclosporine | 0 | 0 | 1 |
| Mycophenolic Acid | 0 | 0 | 1 |
| Thalidomide | 0 | 0 | 1 |
| Alendronate | 0 | 0 | 1 |
| Rivogenlecleucel | 0 | 0 | 1 |
| AP 1903 reagent | 0 | 0 | 1 |
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 2
- Dec 2026SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International) · University of California, San Francisco
- Mar 2027Krabbe Disease Global Patient Registry · State University of New York at Buffalo
Who these studies take
Of the 5 Sphingolipidoses studies recruiting now, 4 (80%) set no upper age limit and 1 (20%) stop at 75 or younger. 3 (60%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (20%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (60%) | 3 (60%) |
| 18 | 5 (100%) | 5 (100%) |
| 30 | 5 (100%) | 5 (100%) |
| 40 | 5 (100%) | 5 (100%) |
| 50 | 5 (100%) | 5 (100%) |
| 65 | 5 (100%) | 5 (100%) |
| 75 | 4 (80%) | 4 (80%) |
| 85 | 4 (80%) | 4 (80%) |
Across all 23 studies, recruiting or not: 57% stop at 75 or younger and 57% at 65; 74% take children; 0% are for women only and 9% for men only; 4% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 23 of 23. search them.
- Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.RecruitingNCT048803561 country
- Krabbe Disease Global Patient RegistryRecruitingNCT029937961 country
- Natural History of Sphingosine Phosphate Lyase Insufficiency Syndrome (SPLIS)RecruitingNCT066699491 country
- SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)RecruitingNCT048851791 country
- 4D-310 in Adults With Fabry Disease and Cardiac InvolvementRecruitingNCT05629559Phase 1/22 countries
- Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCTActive, not recruitingNCT05739643Phase 1/21 country
- MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisActive, not recruitingNCT02171104Phase 21 country
- An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry DiseaseActive, not recruitingNCT04519749Phase 1/21 country
- Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant DiseasesTerminatedNCT02231710Phase 11 country
- Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher DiseaseCompletedNCT024166617 countries
- Natural History Study of Children With Metachromatic LeukodystrophyTerminatedNCT0196365010 countries
- Effect of Glycerol Trierucate on Clinical Course of AdrenoleukodystrophyTerminatedNCT00004418Phase 21 country
- Farber Disease Natural History StudyCompletedNCT032338419 countries
- Allogeneic Bone Marrow Transplant for Inherited Metabolic DisordersCompletedNCT01043640Phase 21 country
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTerminatedNCT00668564Phase 21 country
- Randomized Study of Beta Interferon and Thalidomide in Patients With AdrenoleukodystrophyCompletedNCT00004450Not applicable
- Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's DiseaseCompletedNCT00004488Phase 22 countries
- Study of Bile Acids in Patients With Peroxisomal DisordersTerminatedNCT00004442Not applicable
- Study to Evaluate Blood Cell Lines From Patients With Gaucher DiseaseCompletedNCT003511561 country
- Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher DiseaseCompletedNCT00004294Phase 11 country
- Stem Cell Transplantation (SCT) for Genetic DiseasesCompletedNCT00004378Not applicable1 country
- Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell TransplantationStatus unknownNCT000059001 country
- Phase II Study of Glucocerebrosidase in Patients With Gaucher DiseaseStatus unknownNCT00004293Phase 21 country