Cystinosis Clinical Trial
Official title:
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
This study will examine the safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients, aged ≥ 10 years. The ability of NPI-001 to reduce white blood cell (WBC) cystine will be assessed and compared with cysteamine. ;
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