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 cystine will be assessed and compared with cysteamine.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Single dose, tablets at current therapeutic dose
Single dose, oral solution
Children's Hospital at Westmead
Westmead, New South Wales, Australia
RECRUITINGConcentration of Cystine Levels Over Time
Cystine concentration over 6 hours
Time frame: 1 day
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