Niemann-pick Disease Type
📧 Sign up to get email alerts when something new happens for Niemann-pick Disease Type.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
-
CLINICAL_TRIALS trial_result · 2025-12-12Clinical Trial Update: Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C [Phase 1, Phase 2]
Phase 1/2 trial evaluating intravenous VTS-270 for infantile liver disease in Niemann-Pick Disease, Type C has completed and results have be…
-
CLINICAL_TRIALS trial_result · 2025-03-30Clinical Trial Update: Safety and Efficacy of Miglustat in Chinese NPC Patients [Phase 4]
Phase 4 study in Chinese Niemann-Pick Disease Type C patients evaluated miglustat for safety and efficacy. The trial has completed.
-
CLINICAL_TRIALS trial_result · 2025-02-04Clinical Trial Update: Miglustat in Niemann-Pick Type C Disease [Phase 2]
Phase 2 trial of miglustat for Niemann-Pick disease type C has completed.
-
CLINICAL_TRIALS trial_result · 2024-01-17Clinical Trial Update: Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System [Phase 3]
Phase 3 trial of Adrabetadex for Niemann-Pick Type C disease has been terminated; results posted.
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS trial_result · 2023-12-26Clinical Trial Update: Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1 [Phase 2, Phase 3]
Open-label study of VTS-270 in neurologic manifestations of Niemann-Pick disease type C1 was terminated; results have been posted.
-
CLINICAL_TRIALS trial_result · 2023-11-28Clinical Trial Update: N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) [Phase 2]
Phase 2 trial update for N-Acetyl-L-Leucine in Niemann-Pick Disease, Type C (NPC) completed with results posted.
-
CLINICAL_TRIALS trial_result · 2023-08-29Clinical Trial Update: Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease [Phase 2, Phase 3]
Clinical trial update for adrabetadex in Niemann-Pick disease type C1 (NPC1) has terminated after Phase 2/3; results posted were in 2023.
-
CLINICAL_TRIALS trial_result · 2023-06-09Clinical Trial Update: Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C [Phase 2, Phase 3]
A prospective Phase 2/3 trial of arimoclomol in Niemann-Pick Disease Type C has completed and results have been posted.
-
CLINICAL_TRIALS trial_result · 2023-02-22Clinical Trial Update: VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease [Phase 2, Phase 3]
Clinical trial update for VTS-270 in Niemann-Pick Disease Type C1 (NPC1) across Phase 2 and Phase 3, with completed status and results poste…
-
CLINICAL_TRIALS trial_result · 2022-05-24Clinical Trial Update: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency [Phase 2, Phase 3]
Phase 2/3 trials evaluated the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyeli…
-
CLINICAL_TRIALS trial_result · 2021-08-10Clinical Trial Update: Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients [Phase 1, Phase 2]
A completed Phase 1/2 clinical trial evaluating intravenous Trappsol Cyclo (HPBCD) for Niemann-Pick disease type C1, focusing on safety and …
-
CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease [Phase 1]
Phase 1 clinical trial completed evaluating saccadic eye movements in patients with Niemann-Pick disease type C.
-
CLINICAL_TRIALS trial_result · 2017-05-18Clinical Trial Update: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C
Prospective non-therapeutic observational study in patients with Niemann-Pick Disease Type C; the trial status is completed.
-
CLINICAL_TRIALS trial_result · 2017-01-13Clinical Trial Update: Screening of Niemann-Pick Disease, Type C in a Psychiatric Population [NA]
A clinical trial screening Niemann-Pick disease type C in a psychiatric adult population (NCT02841358) sponsored by University Hospital, Gre…
-
CLINICAL_TRIALS trial_result · 2016-01-06Clinical Trial Update: Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
This entry notes the completion of a longitudinal cognitive study in Niemann-Pick disease, type C. No treatment results are provided in the …
-
CLINICAL_TRIALS trial_result · 2013-01-04Clinical Trial Update: Application of Miglustat in Patients With Niemann-Pick Type C [Phase 3]
Phase 3 trial update of miglustat in patients with Niemann-Pick disease type C; the study is reported as completed.
-
CLINICAL_TRIALS trial_result · 2012-07-12Clinical Trial Update: Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating the safety and potential efficacy of N-acetylcysteine in Niemann-Pick disease Type C has completed, wi…