Niemann-Pick Disease Clinical Trials Niemann-Pick Disease RSS feedThis is an RSS file. You can use it to subscribe to this data in your favourite RSS reader or to display this data on your own website or blog.

A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
Conditions: Niemann-Pick Diseases; Acid Sphingomyelinase Deficiency Interventions: Drug: Olipudase alfa Sponsors: Sanofi; Pulse Infoframe Ltd. Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 5, 2024 Category: Research Source Type: clinical trials

N-acetyl-L-leucine for Niemann-Pick type C: a multinational double-blind randomized placebo-controlled crossover study
DiscussionPre-clinical as well as observational and phase IIb clinical trials have previously demonstrated that IB1001 rapidly improved symptoms, functioning, and quality of life for pediatric and adult NPC patients and is safe and well tolerated. In this placebo-controlled cross-over trial, the risk/benefit profile of IB1001 for NPC will be evaluated. It will also give information about the applicability of IB1001 as a therapeutic paradigm for other rare and common neurological disorders.Trial registrationsThe trial (IB1001-301) has been registered atwww.clinicaltrials.gov (NCT05163288) andwww.clinicaltrialsregister.eu (E...
Source: Trials - May 29, 2023 Category: Research Source Type: clinical trials

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
Conditions:   GM2 Gangliosidosis;   Niemann-Pick Disease, Type C Interventions:   Drug: AZ-3102 (Dose 1);   Drug: Placebo;   Drug: AZ-3102 (Dose 2) Sponsor:   Azafaros A.G. Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - March 8, 2023 Category: Research Source Type: clinical trials

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Condition:   Niemann-Pick Disease, Type C Intervention:   Sponsor:   Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - October 20, 2022 Category: Research Source Type: clinical trials