PK and PD Study of NPI-001 and Cysteamine Bitartrate

Phase 1

Recruiting

• Conditions: Cystinosis
• Interventions: Drug: Cysteamine Bitartrate; Drug: N-Acetylcysteine Amide

• Registration Number: NCT05994534
• Lead Sponsor: Nacuity Pharmaceuticals, Inc.

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.

Study Timeline

• Study First Submitted: 2023-08-08
• Study First Submitted QC: 2023-08-08
• Study First Posted: 2023-08-16
• Study Start: 2023-10-29
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-12
• Last Update Posted: 2024-11-14
• Primary Completion: 2025-04-21
• Study Completion: 2025-06-21

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

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).

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
cysteamine	Cysteamine Bitartrate	Single dose, tablets in current treatment dose
NPI-001	N-Acetylcysteine Amide	Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.

Primary Outcome Measures

Name	Time	Method
Concentration of Cystine Levels Over Time	1 day	Cystine concentration over 6 hours

Trial Locations

Locations (1)

Children's Hospital at Westmead; 🇦🇺 Westmead, New South Wales, Australia