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A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
Rochester, MN
The purpose of this study is to demonstrate that N-Acetyl-L-Leucine is effective in improving symptoms, functioning, and quality of life against the defined endpoints in patients with Niemann-Pick Disease Type C (NPC) for the purpose of establishing the benefit/risk balance of the investigational medicinal product (IB1001) in the proposed clinical setting.
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Early Access Program With Arimoclomol in US Patients With NPC
Rochester, MN
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. NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. 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.
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A Study to Establish a Registry for Niemann-Pick Disease Type A, B, and C
Rochester, MN
The purpose of this study is to establish the natural history of the 3 diseases - Niemann-Pick Type A, B, and C (their characteristics, management and outcomes).
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A Study to Establish a Rare Diseases Registry for Niemann-Pick Disease Types A, B and C
Rochester, MN
The purposes of this study are to establish the natural history of the 3 diseases including their characteristics, management and outcomes; to assess clinical effectiveness of management and quality of care; to provide an inventory of patients for recruitment to interventional studies; to establish genotype-phenotype correlations; and to support post marketing obligations for approved therapies.
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A Study to Evaluate N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
No Locations
The purpose of this study is to assess the safety and effectiveness of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
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Arimoclomol Prospective Study in Patients Diagnosed With NiemannPick Disease Type C
Rochester, MN
A prospective, randomised, double-blind, placebo controlled therapeutic study in patients with confirmed diagnosis of NiemannPick disease type C (NPC). The purpose of this study is to assess the efficacy and safety of arimoclomol (compared to placebo) when it is administered as an add-on therapy to the patient's current prescribed best standard of care; patient's standard of care may, or may not, include miglustat.