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Niemann-Pick Diseases clinical trials

View clinical trials related to Niemann-Pick Diseases.

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NCT ID: NCT04958642 Terminated - Clinical trials for Niemann-Pick Disease, Type C

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

Start date: December 23, 2015
Phase: Phase 2/Phase 3
Study type: Interventional

Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.

NCT ID: NCT03887533 Terminated - Clinical trials for Niemann-Pick Disease, Type C1

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

Start date: January 6, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

Background: For people who have Niemann-Pick disease, type C1 (NPC1), cholesterol and other fats have trouble moving out of liver and other tissue cells. This makes the cells sick. Researchers want to find out if a drug called VTS-270 can help. Objective: To test if VTS-270 is safe and effective in treating chronic liver disease associated with NPC1. Eligibility: People ages 3-60 with NPC1 Design: Participants may be screened by phone or under another protocol. Participants will have visits once a month for 12 months. If they have intrathecal injections, the study may last 15 months or more. The first visit will last about 5 days. Others will last 2-3 days. Participants will get VTS-270 injected into a vein at each visit. They can also choose to have intrathecal injections. These are like spinal taps. Some visits will also include: Physical exam Urine tests Blood tests. A small tube or needle will be inserted into the participants vein to collect blood. The small tube will also be used to give the VTS-270. Hearing tests: For one test, participants will have electrodes taped to their head. These will record brain waves. Breathing tests Ultrasound of abdomen: Sounds waves will take pictures of the participant s body. Chest x-ray: This is a picture of the lungs.

NCT ID: NCT03879655 Terminated - Clinical trials for Niemann-Pick Disease, Type C

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

Start date: December 2, 2019
Phase: Phase 2/Phase 3
Study type: Interventional

This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B [NCT02534844] and Part C [NCT04958642]) with neurologic manifestations of Niemann-Pick Type C1 (NPC1) disease.

NCT ID: NCT03643562 Terminated - Clinical trials for Niemann-Pick Type C Disease

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

Start date: June 18, 2018
Phase: Phase 3
Study type: Interventional

This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks. Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg. Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.

NCT ID: NCT02841358 Terminated - Clinical trials for Psychiatric Adults Patients

Screening of Niemann-Pick Disease, Type C in a Psychiatric Population

NPCPsy
Start date: December 2013
Phase: N/A
Study type: Interventional

Niemann-Pick disease, Type C is a rare genetic disorder characterized by a failing in intracellular cholesterol transport, inducing an accumulation of sphingolipids in the brain. Neurological signs are at the forefront of the disease. There are also psychiatric signs of psychotic kind among 28 to 45 % of patients according to studies, and a thirty cases were published. These signs can be concomitant with neurological signs or precede them. Is is likely that psychotic disorders are the first signs of a Niemann-Pick disease not yet non encore diagnosed for some patients. Yet, no prevalence study for this disease in a psychiatric population of patients currently exists. In response to this problem this study proposes to search patients whose disease could be of organic origin or patients whose disease is suspected, based on clinical data. The diagnosis will be confirmed certified with a genetic and/or biochemical test.

NCT ID: NCT01372228 Terminated - Sandhoff Disease Clinical Trials

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Start date: April 2011
Phase: Phase 1/Phase 2
Study type: Interventional

The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.

NCT ID: NCT00668564 Terminated - Clinical trials for Niemann-Pick Disease, Type C

Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Start date: March 2008
Phase: Phase 2
Study type: Interventional

The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).

NCT ID: NCT00410566 Terminated - Clinical trials for Niemann-Pick Disease

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

Start date: December 2006
Phase: Phase 1
Study type: Interventional

The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.