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A Exploratory Trial of WTX221 in Refractory Gout Patients

Primary Purpose

Gout Tophus

Status
Not yet recruiting
Phase
Early Phase 1
Locations
Study Type
Interventional
Intervention
WTX221
Sponsored by
RenJi Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gout Tophus

Eligibility Criteria

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

Inclusion Criteria: Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form, and be able to complete all trial processes as required by the protocol; Subject is ≥ 18 years of age; Patients with refractory gout: multiple tophi, more than 2 sites; Subjects (males and females) must agree to be free of childbearing potential and willing to use effective contraception with their partner and have no plans to donate sperm or eggs during the trial and for at least 1 year after the infusion. Exclusion Criteria: Patients with allergic diseases or specific allergic history, or possible allergic to the study drug and any of its components or related preparations; Patients unable to communicate or cooperate with medical staff due to neurological, mental illness or language disorder, which affects patient compliance; Having any past or present medical history that may affect the safety of the trial or the in vivo process of the drug, especially history of cardiovascular, liver, kidney, digestive tract, lung, spleen, nerve, blood, tumor, immune or metabolic disorders that are considered clinically significant by the investigator; Participated in other drug or medical device clinical trials within 3 months before screening; Patients who have received intravenous anti-infective treatment or active infection within 14 days before administration; Patients with history of allogeneic organ, bone marrow transplantation or stem cell transplantation; Patients with laboratory abnormalities that are clinically significant: The investigator believes that it will prevent the subject from following and completing any other circumstances of the study protocol;

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm 3

    Arm Type

    Experimental

    Experimental

    Experimental

    Arm Label

    Treatment group A

    Treatment group B

    Treatment group C

    Arm Description

    WTX221 low dose

    WTX221middle dose

    WTX221 high dose

    Outcomes

    Primary Outcome Measures

    To evaluate the safety and tolerability of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout.
    Adverse Events and Serious Adverse Events

    Secondary Outcome Measures

    To evaluate the pharmacokinetic (PK) profile of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout;
    Functional protein content in blood (PK)
    To evaluate the pharmacodynamic (PD) profile of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout
    plasma uric acid(UA) level
    To evaluate the preliminary efficacy of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout
    plasma UA level < 360 umol/L
    To evaluate immunogenicity (ADA) levels of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout
    ADA incidence rate

    Full Information

    First Posted
    March 23, 2023
    Last Updated
    April 4, 2023
    Sponsor
    RenJi Hospital
    Collaborators
    Westlake Therapeutics
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05800041
    Brief Title
    A Exploratory Trial of WTX221 in Refractory Gout Patients
    Official Title
    A Exploratory Trial to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD), Immunogenicity, and Preliminary Efficacy of WTX221 Infusion in Patients With Refractory Gout
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2023
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    April 10, 2023 (Anticipated)
    Primary Completion Date
    July 30, 2023 (Anticipated)
    Study Completion Date
    February 1, 2024 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    RenJi Hospital
    Collaborators
    Westlake Therapeutics

    4. Oversight

    Studies a U.S. FDA-regulated Drug Product
    No
    Studies a U.S. FDA-regulated Device Product
    No
    Product Manufactured in and Exported from the U.S.
    No
    Data Monitoring Committee
    Yes

    5. Study Description

    Brief Summary
    This trial is a single-center, open-label designed investigator-initiated clinical study (IIT) to investigate the safety, tolerability, PK, PD, immunogenicity, and preliminary efficacy of multiple doses of WTX221 infusion combined with a single dose of the immunosuppressive agent cyclophosphamide (CTX) to induce immune tolerance. The trial consisted of a Screening Period (14 days), a First Dose Period (30 days), a Second Dose Period (30 days), and a Safety Follow-up Period (150 days) for a total of 224 days.
    Detailed Description
    Refractory gout is usually associated with persistently swollen and tender joints, chronic pain, reduced functional status, impaired quality of life, and joint destruction,most of the patients can't tolerate urate-lowering therapy(ULT)or receive adequate dose of ULT treatment recommended to control Serum uric acid (SUA) to target level. WTX221 is designed to induce antigen-specific immune tolerance. WTX221 is made of urate oxidase(UOX) molecules conjugated on the surface of Red blood cells(RBCs), which can induce antigen-specific immune tolerance to UOX in vivo by making use of it's natural biologic characteristics. WTX221 infusion combined with a single dose CTX to induce immune tolerance. The trial consisted of a Screening Period (14 days), a First Dose Period (30 days), a Second Dose Period (30 days), and a Safety Follow-up Period (150 days) for a total of 224 days.

    6. Conditions and Keywords

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

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Early Phase 1
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    Non-Randomized
    Enrollment
    7 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Treatment group A
    Arm Type
    Experimental
    Arm Description
    WTX221 low dose
    Arm Title
    Treatment group B
    Arm Type
    Experimental
    Arm Description
    WTX221middle dose
    Arm Title
    Treatment group C
    Arm Type
    Experimental
    Arm Description
    WTX221 high dose
    Intervention Type
    Drug
    Intervention Name(s)
    WTX221
    Other Intervention Name(s)
    cyclophosphamide
    Intervention Description
    Drug: WTX221 WTX221 infusion once every 30 days for two doses in total Drug: CTX Co-administration of CTX and WTX221 at the initial infusion
    Primary Outcome Measure Information:
    Title
    To evaluate the safety and tolerability of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout.
    Description
    Adverse Events and Serious Adverse Events
    Time Frame
    from the first WTX221 infusion to 210 days
    Secondary Outcome Measure Information:
    Title
    To evaluate the pharmacokinetic (PK) profile of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout;
    Description
    Functional protein content in blood (PK)
    Time Frame
    from the first WTX221 infusion to 210 days
    Title
    To evaluate the pharmacodynamic (PD) profile of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout
    Description
    plasma uric acid(UA) level
    Time Frame
    from the first WTX221 infusion to 210 days
    Title
    To evaluate the preliminary efficacy of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout
    Description
    plasma UA level < 360 umol/L
    Time Frame
    from the first WTX221 infusion to 60days Number of gout flares in subjects (within 60 days);
    Title
    To evaluate immunogenicity (ADA) levels of multiple doses of WTX221 infusion combined with a single dose of cyclophosphamide in patients with refractory gout
    Description
    ADA incidence rate
    Time Frame
    from the first WTX221 infusion to 210days

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form, and be able to complete all trial processes as required by the protocol; Subject is ≥ 18 years of age; Patients with refractory gout: multiple tophi, more than 2 sites; Subjects (males and females) must agree to be free of childbearing potential and willing to use effective contraception with their partner and have no plans to donate sperm or eggs during the trial and for at least 1 year after the infusion. Exclusion Criteria: Patients with allergic diseases or specific allergic history, or possible allergic to the study drug and any of its components or related preparations; Patients unable to communicate or cooperate with medical staff due to neurological, mental illness or language disorder, which affects patient compliance; Having any past or present medical history that may affect the safety of the trial or the in vivo process of the drug, especially history of cardiovascular, liver, kidney, digestive tract, lung, spleen, nerve, blood, tumor, immune or metabolic disorders that are considered clinically significant by the investigator; Participated in other drug or medical device clinical trials within 3 months before screening; Patients who have received intravenous anti-infective treatment or active infection within 14 days before administration; Patients with history of allogeneic organ, bone marrow transplantation or stem cell transplantation; Patients with laboratory abnormalities that are clinically significant: The investigator believes that it will prevent the subject from following and completing any other circumstances of the study protocol;
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Ting Lee, phD
    Phone
    021-68383204
    Email
    leeting007@163.com

    12. IPD Sharing Statement

    Plan to Share IPD
    No

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    A Exploratory Trial of WTX221 in Refractory Gout Patients

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