PK and PD Study of NPI-001 and Cysteamine Bitartrate

NCT05994534 | Recruiting Phase 1

• 1 other identifier: C-23-01
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Cystinosis

Outcome Measures

Primary Outcomes (1)

• Concentration of Cystine Levels Over Time

  Cystine concentration over 6 hours

  1 day

Study Arms (2)

cysteamine

ACTIVE COMPARATOR

Single dose, tablets in current treatment dose

Drug: Cysteamine Bitartrate

NPI-001

EXPERIMENTAL

Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.

Drug: N-Acetylcysteine Amide

Interventions

Cysteamine Bitartrate DRUG

Single dose, tablets at current therapeutic dose

Also known as: Cystagon

cysteamine

N-Acetylcysteine Amide DRUG

Single dose, oral solution

Also known as: NPI-001

NPI-001

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Nacuity Pharmaceuticals, Inc.: lead

Study Sites (1)

Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia

RECRUITING

MeSH Terms

Conditions

Cystinosis

Interventions

Cysteamine; N-Acetylcysteinamide

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Mercaptoethylamines; Ethylamines; Amines; Organic Chemicals; Sulfhydryl Compounds; Sulfur Compounds

Study Officials

• Hugh McCarthy, PhD, FRACP

  Sydney Children's Health Network

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jami Kern, PhD

CONTACT

+1-817-336-3000; info@nacuity.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 8, 2023
• First Posted: August 16, 2023
• Study Start: October 29, 2023
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): August 1, 2026
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)