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PK and PD Study of NPI-001 and Cysteamine Bitartrate (INCA)

Primary Purpose

Cystinosis

Status
Not yet recruiting
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Cysteamine Bitartrate
N-Acetylcysteine Amide
Sponsored by
Nacuity Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystinosis

Eligibility Criteria

10 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Active Comparator

    Experimental

    Arm Label

    cysteamine

    NPI-001

    Arm Description

    Single dose, tablets in current treatment dose

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

    Outcomes

    Primary Outcome Measures

    Concentration of Cystine Levels Over Time
    White Blood Cell cystine concentration over 6 hours

    Secondary Outcome Measures

    Full Information

    First Posted
    August 8, 2023
    Last Updated
    August 15, 2023
    Sponsor
    Nacuity Pharmaceuticals, Inc.
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05994534
    Brief Title
    PK and PD Study of NPI-001 and Cysteamine Bitartrate
    Acronym
    INCA
    Official Title
    A Phase 1/2 Pharmacokinetic and Pharmacodynamic Study of NPI-001 Oral Solution Compared to Cysteamine Bitartrate in Cystinosis Patients
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    October 1, 2023 (Anticipated)
    Primary Completion Date
    December 21, 2023 (Anticipated)
    Study Completion Date
    April 30, 2024 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Nacuity Pharmaceuticals, Inc.

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Data Monitoring Committee
    No

    5. Study Description

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

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cystinosis

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1, Phase 2
    Interventional Study Model
    Crossover Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    12 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    cysteamine
    Arm Type
    Active Comparator
    Arm Description
    Single dose, tablets in current treatment dose
    Arm Title
    NPI-001
    Arm Type
    Experimental
    Arm Description
    Single dose, NPI-001 (N-acetylcysteine amide) oral solution at molar equivalent of current cysteamine dose.
    Intervention Type
    Drug
    Intervention Name(s)
    Cysteamine Bitartrate
    Other Intervention Name(s)
    Cystagon
    Intervention Description
    Single dose, tablets at current therapeutic dose
    Intervention Type
    Drug
    Intervention Name(s)
    N-Acetylcysteine Amide
    Other Intervention Name(s)
    NPI-001
    Intervention Description
    Single dose, oral solution
    Primary Outcome Measure Information:
    Title
    Concentration of Cystine Levels Over Time
    Description
    White Blood Cell cystine concentration over 6 hours
    Time Frame
    1 day

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    10 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    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.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Jami Kern, PhD
    Phone
    +1-817-336-3000
    Email
    info@nacuity.com
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Hugh McCarthy, PhD, FRACP
    Organizational Affiliation
    Sydney Children's Health Network
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    No
    IPD Sharing Plan Description
    Individual participant data will not be made available to other researchers.

    Learn more about this trial

    PK and PD Study of NPI-001 and Cysteamine Bitartrate

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