Niemann-Pick Disease Clinical Trials This 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.

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
Condition: Niemann-Pick Disease, Type C1 Interventions: Drug: Hydroxypropyl-beta-cyclodextrin; Drug: Placebo Sponsor: Cyclo Therapeutics, Inc. Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - April 27, 2021 Category: Research Source Type: clinical trials

A master protocol to investigate a novel therapy acetyl- l -leucine for three ultra-rare neurodegenerative diseases: Niemann-Pick type C, the GM2 gangliosidoses, and ataxia telangiectasia
DiscussionThe novel CI-CS assessment, developed through a collaboration of all stakeholders, is advantageous in that it better ensures the primary endpoint is functionally relevant for each patient, is able to capture small but meaningful clinical changes critical to the patients ’ quality of life (fine-motor skills; gait), and blinds the primary outcome assessment. The results of these three trials will inform whether N-acetyl-l-leucine is an effective treatment for NPC, GM2, and A-T and can also serve as a new therapeutic paradigm for the development of future treatments for other orphan diseases.Trial registrationThe ...
Source: Trials - January 22, 2021 Category: Research Source Type: clinical trials

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick
Condition: Niemann-Pick Diseases Intervention: Sponsors: National Niemann-Pick Disease Foundation; Engage Health, Inc.; Icahn School of Medicine at Mount Sinai Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - July 14, 2020 Category: Research Source Type: clinical trials