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Treatment With Metformin in Chinese Children With Alport Syndrome

Primary Purpose

Alport Syndrome, Metformin

Status
Not yet recruiting
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Metformin
Placebo
Sponsored by
Peking University First Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Alport Syndrome

Eligibility Criteria

10 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: (1) The diagnostic of Alport syndrome-hematuria with or without proteinuria meets one or more of the following 4 items: i) Immunofluorescence staining of the α5 (IV) chain of the glomerular basement membrane was abnormal; ii) electron microscopy of renal biopsy showed diffuse changes in the glomerular basement membrane; iii) There was one pathogenic variant in the COL4A5 gene or two pathogenic variants in the COL4A3/COL4A4 gene; iv) Family history of Alport syndrome. (2) Age is older than or equal to 10 years old. (3) The 24-hour urine protein quantification was greater than 150mg and the urine microalbumin-creatinine ratio was greater than 30 mg/g. (4) The estimated glomerular filtration rate (24-hour creatinine clearance) was greater than 45 mL/min/1.73m2. (5) Accept the maximum tolerated dose of renin-angiotensin-aldosterone system blockers (Blood pressure in the clinic was in the 50th percentile of the same age, same sex and same height but greater than 90/60mmHg, blood potassium was normal, and the increase of blood creatinine did not exceed 30%) for at least 3 months. Exclusion Criteria: (1) Vitamin B12 deficiency was uncorrected. (2) Treated with Furosemide, amiloride, and nifedipine. (3) Hypertension, chronic liver disease, chronic heart disease, and chronic kidney disease resulting from causes except Alport syndrome. (4) History of hyperlactatemia. (5) Allergy to metformin. (6) kidney transplant. (7) Poor compliance. (8) Type 1 diabetes.

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Active Comparator

    Placebo Comparator

    Arm Label

    Metformin intervention treatment group

    The placebo treatment group

    Arm Description

    Outcomes

    Primary Outcome Measures

    Efficacy of metformin for Alport syndrome
    either decrease in proteinuria or steady eGFR from baseline. To assess the decrease in proteinuria or steady eGFR from baseline to month 12 or month 24 under metformin treatment compared to placebo
    Safety of metformin for Alport syndrome
    The occurrence of adverse events. Safety will be assessed by monitoring adverse drug events, physical examinations and clinical laboratory test through 24 months

    Secondary Outcome Measures

    Full Information

    First Posted
    December 8, 2022
    Last Updated
    December 16, 2022
    Sponsor
    Peking University First Hospital
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05655728
    Brief Title
    Treatment With Metformin in Chinese Children With Alport Syndrome
    Official Title
    Efficacy and Safety of Metformin in Alport Syndrome: A Randomized, Double-blind, Placebo-controlled Study in China
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    December 2022
    Overall Recruitment Status
    Not yet recruiting
    Study Start Date
    January 3, 2023 (Anticipated)
    Primary Completion Date
    January 31, 2025 (Anticipated)
    Study Completion Date
    April 20, 2025 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Peking University First Hospital

    4. Oversight

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

    5. Study Description

    Brief Summary
    This single-center, prospective, double-blind randomized placebo-controlled trial will evaluate the efficacy and safety of metformin in Chinese children with Alport syndrome who have received (and continue to receive)) ACEi/ARB.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Alport Syndrome, Metformin

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Parallel Assignment
    Masking
    ParticipantCare ProviderInvestigatorOutcomes Assessor
    Allocation
    Randomized
    Enrollment
    78 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Metformin intervention treatment group
    Arm Type
    Active Comparator
    Arm Title
    The placebo treatment group
    Arm Type
    Placebo Comparator
    Intervention Type
    Drug
    Intervention Name(s)
    Metformin
    Intervention Description
    The participants will receive treatment of metformin (initial dose of 500 mg/day, within 2 weeks reaching the maximum tolerated dose [maximum: 1500 mg/day]).
    Intervention Type
    Other
    Intervention Name(s)
    Placebo
    Intervention Description
    The participants will receive treatment of placebo.
    Primary Outcome Measure Information:
    Title
    Efficacy of metformin for Alport syndrome
    Description
    either decrease in proteinuria or steady eGFR from baseline. To assess the decrease in proteinuria or steady eGFR from baseline to month 12 or month 24 under metformin treatment compared to placebo
    Time Frame
    24 months
    Title
    Safety of metformin for Alport syndrome
    Description
    The occurrence of adverse events. Safety will be assessed by monitoring adverse drug events, physical examinations and clinical laboratory test through 24 months
    Time Frame
    24 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    10 Years
    Maximum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: (1) The diagnostic of Alport syndrome-hematuria with or without proteinuria meets one or more of the following 4 items: i) Immunofluorescence staining of the α5 (IV) chain of the glomerular basement membrane was abnormal; ii) electron microscopy of renal biopsy showed diffuse changes in the glomerular basement membrane; iii) There was one pathogenic variant in the COL4A5 gene or two pathogenic variants in the COL4A3/COL4A4 gene; iv) Family history of Alport syndrome. (2) Age is older than or equal to 10 years old. (3) The 24-hour urine protein quantification was greater than 150mg and the urine microalbumin-creatinine ratio was greater than 30 mg/g. (4) The estimated glomerular filtration rate (24-hour creatinine clearance) was greater than 45 mL/min/1.73m2. (5) Accept the maximum tolerated dose of renin-angiotensin-aldosterone system blockers (Blood pressure in the clinic was in the 50th percentile of the same age, same sex and same height but greater than 90/60mmHg, blood potassium was normal, and the increase of blood creatinine did not exceed 30%) for at least 3 months. Exclusion Criteria: (1) Vitamin B12 deficiency was uncorrected. (2) Treated with Furosemide, amiloride, and nifedipine. (3) Hypertension, chronic liver disease, chronic heart disease, and chronic kidney disease resulting from causes except Alport syndrome. (4) History of hyperlactatemia. (5) Allergy to metformin. (6) kidney transplant. (7) Poor compliance. (8) Type 1 diabetes.

    12. IPD Sharing Statement

    Plan to Share IPD
    No

    Learn more about this trial

    Treatment With Metformin in Chinese Children With Alport Syndrome

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