Overview
Safety, pharmacokinetics, and pharmacodynamics of NPI-001 oral solution in cystinosis patients compared with cysteamine.
Description
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.
Eligibility
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.