PK and PD Study of NPI-001 and Cysteamine Bitartrate (INCA)
Cystinosis
About this trial
This is an interventional treatment trial for Cystinosis
Eligibility Criteria
Inclusion Criteria: Males or females, any race, ≥ 10 years of age. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days. Females will be nonpregnant and nonlactating, and females of childbearing potential and males will agree to use contraception as detailed in the protocol. Able to comprehend and willing to sign an informed consent /assent form and to abide by the study restrictions (travel as necessary, clinical phase 1 unit or similar for up to 3 days). Exclusion Criteria: Have undergone kidney transplantation. Are receiving dialysis treatment. History of significant hypersensitivity to NAC or any ingredient of NPI-001 oral solution. Participation in a clinical study involving administration of an investigational drug (new chemical entity) in the 30 days prior to Day 1. Inability to provide blood samples, including difficulty with venous access. Subjects who, in the opinion of the Investigator and/or Sponsor (or designee), should not participate in this study.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Active Comparator
Experimental
cysteamine
NPI-001
Single dose, tablets in current treatment dose
Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.