Early Access Program With Arimoclomol in US Patients With NPC

Status Available

Clinical Trial Summary

NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT04316637
Study type	Expanded Access
Source	ZevraDenmark
Contact	Zevra Therapeutics Medical Information
Phone	+1-888-289-5607
Email	medicalaffairs@zevra.com
Status	Available
Phase

View Details

See also
Status	Clinical Trial	Phase
Completed	`NCT03759639` - N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)	Phase 2
Withdrawn	`NCT04189601` - Complement Activation in the Lysosomal Storage Disorders
Completed	`NCT02435030` - A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C	N/A
Completed	`NCT02534844` - VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease	Phase 2/Phase 3
Recruiting	`NCT05588167` - Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Withdrawn	`NCT03687476` - Safety and Tolerability Study of VTS-270 in Pediatric Participants With Niemann-Pick Type C (NPC) Disease	Phase 2
Enrolling by invitation	`NCT05368038` - ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Completed	`NCT01899950` - Longitudinal Study of Cognition With Niemann-Pick Disease, Type C	N/A
Terminated	`NCT00668564` - Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism	Phase 2
Active, not recruiting	`NCT05163288` - A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C	Phase 3
Active, not recruiting	`NCT02612129` - Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C	Phase 2/Phase 3
Recruiting	`NCT03471143` - Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C	Phase 1/Phase 2
Terminated	`NCT04958642` - Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease	Phase 2/Phase 3
Terminated	`NCT03879655` - Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1	Phase 2/Phase 3
Completed	`NCT00975689` - Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine	Phase 1/Phase 2
Withdrawn	`NCT01306604` - Biomarker for Niemann Pick Type C Disease (BioNPC)
Recruiting	`NCT00344331` - Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C
Active, not recruiting	`NCT05758922` - Phase 2 Study Evaluating the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Oral AZ-3102 in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease	Phase 2
Completed	`NCT03910621` - Safety and Efficacy of Miglustat in Chinese NPC Patients	Phase 4