PK and PD Study of NPI-001 and Cysteamine Bitartrate

Last updated: November 12, 2024
Sponsor: Nacuity Pharmaceuticals, Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

Kidney Failure (Pediatric)

Kidney Failure

Treatment

Cysteamine Bitartrate

N-Acetylcysteine Amide

Clinical Study ID

NCT05994534
C-23-01
  • Ages > 10
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Males or females, any race, ≥ 10 years of age.

  2. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2days.

  3. Females will be nonpregnant and nonlactating, and females of childbearing potentialand males will agree to use contraception as detailed in the protocol.

  4. Able to comprehend and willing to sign an informed consent /assent form and to abideby the study restrictions (travel as necessary, clinical phase 1 unit or similar forup to 3 days).

Exclusion

Exclusion Criteria:

  1. Have undergone kidney transplantation.

  2. Are receiving dialysis treatment.

  3. History of significant hypersensitivity to NAC or any ingredient of NPI-001 oralsolution.

  4. Participation in a clinical study involving administration of an investigationaldrug (new chemical entity) in the 30 days prior to Day 1.

  5. Inability to provide blood samples, including difficulty with venous access.

  6. Subjects who, in the opinion of the Investigator and/or Sponsor (or designee),should not participate in this study.

Study Design

Total Participants: 12
Treatment Group(s): 2
Primary Treatment: Cysteamine Bitartrate
Phase: 1/2
Study Start date:
October 29, 2023
Estimated Completion Date:
June 21, 2025

Study 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 cystine will be assessed and compared with cysteamine.

Connect with a study center

  • Children's Hospital at Westmead

    Westmead, New South Wales 2145
    Australia

    Active - Recruiting

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