RecruitingPhase 1Phase 2NCT05994534

PK and PD Study of NPI-001 and Cysteamine Bitartrate

A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients

Sponsor

Nacuity Pharmaceuticals, Inc.

Enrollment

12 participants

Start Date

Oct 29, 2023

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 10 Years

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.

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