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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05994534

Acronym

INCA

Enrollment

Registered

2023-08-16

Start date

2023-10-29

Completion date

2026-08-01

Last updated

2026-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystinosis

Brief summary

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

Detailed 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.

Interventions

DRUGCysteamine Bitartrate

Single dose, tablets at current therapeutic dose

DRUGN-Acetylcysteine Amide

Single dose, oral solution

Study design

Allocation

NON_RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

10 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Males or females, any race, ≥ 10 years of age. 2. Diagnosis of nephropathic cystinosis and able to cease cysteamine therapy for 2 days. 3. Females will be nonpregnant and nonlactating, and females of childbearing potential and 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 abide by the study restrictions (travel as necessary, clinical phase 1 unit or similar for up to 3 days).

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 oral solution. 4. Participation in a clinical study involving administration of an investigational drug (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.

Design outcomes

Primary

Measure	Time frame	Description
Concentration of Cystine Levels Over Time	1 day	Cystine concentration over 6 hours

Countries

Australia

Contacts

PRINCIPAL_INVESTIGATORHugh McCarthy, PhD, FRACP

Sydney Children's Health Network

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 1, 2026