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NCT Number: NCT05994534

PK and PD Study of NPI-001 and Cysteamine Bitartrate

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

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Cystinosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

10 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Cysteamine bitartrate

Drug

Single dose, tablets at current therapeutic dose

Other names: Cystagon

N-Acetylcysteine Amide

Drug

Single dose, oral solution

Other names: NPI-001

Primary outcomes

  1. Concentration of Cystine Levels Over Time

    Time frame: 1 day

    Cystine concentration over 6 hours

Sponsors and collaborators

Lead sponsor

Nacuity Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Acronym: INCA

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Aug 16, 2023
Registry last updated
Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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