Niemann-Pick Disease, Type C1

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Niemann-Pick Disease, Type C1 trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingPhase 2

NCT07236190

Biomarker-based Trial of NPC-1 for Alzheimer's Pathology

This early phase, open label, single arm clinical trial will determine the intraindividual safety, tolerability and effects of NPC1 (parthenolide and ipriflavon…

United States40 participantsStarted 2026-04-01

Treatment: natural product combination-1 (NPC1)

All recent trials (18 shown)

CompletedNot Applicable

NCT05642221

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Dis…

Background: Neurocognitive disorders affect how the brain uses oxygen. They may affect mental development in children. These disorders can be studied with imag…

United States73 participantsStarted 2023-01-23

By InvitationNot Applicable

NCT03655223

Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…

United States30,000 participantsStarted 2018-10-15

Treatment: Confirmatory Testing

Niemann-Pick Disease, Type C1

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Biomarker-based Trial of NPC-1 for Alzheimer's Pathology

All recent trials (18 shown)

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Dis…

Early Check: Expanded Screening in Newborns

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Biomarker-based Trial of NPC-1 for Alzheimer's Pathology

All recent trials (18 shown)

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Dis…

Early Check: Expanded Screening in Newborns

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Ty…

Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Biomarker for Niemann Pick Type C Disease (BioNPC)

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Dis…

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1

A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C