Clincosm LogoSign in Get a demo

INCA: PK and PD Study of NPI-001 and Cysteamine Bitartrate

Sponsor
Nacuity Pharmaceuticals, Inc. (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05994534
Collaborator
(none)
12
Enrollment
2
Arms
7
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
  • Drug: Cysteamine Bitartrate
  • Drug: N-Acetylcysteine Amide
 Phase 1/Phase 2

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 white blood cell (WBC) cystine will be assessed and compared with cysteamine.

Study Design

Study Type:
Interventional
Anticipated Enrollment :
12 participants
Allocation:
Non-Randomized
Intervention Model:
Crossover Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
Anticipated Study Start Date :
Oct 1, 2023
Anticipated Primary Completion Date :
Dec 21, 2023
Anticipated Study Completion Date :
Apr 30, 2024

Arms and Interventions

Arm Intervention/Treatment
Active Comparator: cysteamine

Single dose, tablets in current treatment dose

Drug: Cysteamine Bitartrate
Single dose, tablets at current therapeutic dose
Other Names:
  • Cystagon

    		•
    Experimental: NPI-001

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

    Drug: N-Acetylcysteine Amide
    Single dose, oral solution
    Other Names:
  • NPI-001

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Concentration of Cystine Levels Over Time [1 day]

      White Blood Cell cystine concentration over 6 hours

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    10 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    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.

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Nacuity Pharmaceuticals, Inc.

    Investigators

    • Principal Investigator: Hugh McCarthy, PhD, FRACP, Sydney Children's Health Network

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Nacuity Pharmaceuticals, Inc.
    ClinicalTrials.gov Identifier:
    NCT05994534
    Other Study ID Numbers:
    • C-23-01
    First Posted:
    Aug 16, 2023
    Last Update Posted:
    Aug 18, 2023
    Last Verified:
    Aug 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    No
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Cystinosis
    Acetylcysteine
    N-monoacetylcystine
    Cysteamine

    Study Results

    No Results Posted as of Aug 18, 2023