Niemann-Pick Disease, Type C clinical trials
The registry lists 37 studies for Niemann-Pick Disease, Type C across more than 10 countries. 8 are recruiting or about to start. 31 of them are mainly about Niemann-Pick Disease, Type C (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.
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Who runs them
Azafaros B.V. (5) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (5) · Mandos LLC (4) · Cyclo Therapeutics, Inc. (4) · ZevraDenmark (3) · IntraBio Inc (2) · Actelion (2) · Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company (2)
What is being tested here
Drugs under test in Niemann-Pick Disease, Type C studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
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 3
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Jun 2027A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease · Azafaros B.V.
- Sep 2027Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Who these studies take
Of the 5 Niemann-Pick Disease, Type C studies recruiting now, 4 (80%) set no upper age limit and 0 (0%) stop at 75 or younger. 5 (100%) 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 | 5 (100%) | 5 (100%) |
| 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 | 5 (100%) | 5 (100%) |
| 85 | 5 (100%) | 5 (100%) |
Across all 37 studies, recruiting or not: 41% stop at 75 or younger and 38% at 65; 89% take children; 0% are for women only and 0% for men only; 11% 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.
Newly registered1
Studies
Recruiting first, then most recently updated. Showing 37 of 37. search them.
- Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type CRecruitingNCT003443311 country
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)RecruitingNCT07082725Phase 315 countries
- A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 GangliosidosisRecruitingNCT07054515Phase 317 countries
- Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal DisordersNot yet recruitingNCT07885917Phase 2/3
- Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type CRecruitingNCT055881671 country
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT053680381 country
- A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type CRecruitingNCT05163288Phase 38 countries
- Early Access Program With Arimoclomol in US Patients With NPCAvailableNCT043166371 country
- A Study to Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseActive, not recruitingNCT07399704Phase 21 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C DiseaseCompletedNCT05758922Phase 21 country
- Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type CCompletedNCT03471143Phase 1/21 country
- Safety and Efficacy of Miglustat in Chinese NPC PatientsCompletedNCT03910621Phase 41 country
- Miglustat in Niemann-Pick Type C DiseaseCompletedNCT00517153Phase 2
- Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type CCompletedNCT02612129Phase 2/39 countries
- Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1Active, not recruitingNCT04860960Phase 313 countries
- Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous SystemTerminatedNCT03643562Phase 31 country
- Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1TerminatedNCT03879655Phase 2/31 country
- N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)CompletedNCT03759639Phase 25 countries
- Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) DiseaseTerminatedNCT04958642Phase 2/39 countries
- VTS-270 to Treat Niemann-Pick Type C1 (NPC1) DiseaseCompletedNCT02534844Phase 2/39 countries
- Biomarker for Niemann Pick Type C Disease (BioNPC)WithdrawnNCT013066043 countries
- Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1TerminatedNCT03887533Phase 1/21 country
- Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C PatientsCompletedNCT02912793Phase 1/23 countries
- Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 DiseaseCompletedNCT01747135Phase 11 country
- Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type CStatus unknownNCT03893071Phase 11 country
- Complement Activation in the Lysosomal Storage DisordersWithdrawnNCT041896011 country
- Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)CompletedNCT02939547Phase 11 country
- Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) DiseaseWithdrawnNCT03687476Phase 2
- Study of Lithium Carbonate to Treat Niemann-Pick Type C1 DiseaseStatus unknownNCT03201627Early Phase 11 country
- Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1CompletedNCT02124083Phase 1/21 country
- Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismTerminatedNCT00668564Phase 21 country
- A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type CCompletedNCT024350308 countries
- Screening of Niemann-Pick Disease, Type C in a Psychiatric PopulationTerminatedNCT02841358Not applicable1 country
- Longitudinal Study of Cognition With Niemann-Pick Disease, Type CCompletedNCT018999501 country
- Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl CysteineCompletedNCT00975689Phase 1/21 country
- Application of Miglustat in Patients With Niemann-Pick Type CCompletedNCT01760564Phase 31 country