RecruitingPhase 1/2

PK and PD Study of NPI-001 and Cysteamine Bitartrate

Australia12 participantsStarted 2023-10-29

Plain-language summary

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

Age range10 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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).

✕. 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.

Concentration of Cystine Levels Over Time

Timeframe: 1 day

NCT IDNCT05994534

SponsorNacuity Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2026-06

Jami Kern, PhD