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N Acetyl Cysteine for Cystinosis Patients

Primary Purpose

Renal Disease, Cystinosis

Status
Completed
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
N acetyl cysteine
N acetyl cysteine
Sponsored by
University of Sao Paulo
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Renal Disease focused on measuring renal function, before and after N acetyl cysteine, Cystinosis patients

Eligibility Criteria

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

Inclusion Criteria:

  • cystinosis patients age under 18 years with good compliance to the treatment with Chronic Kidney Disease 1 to 4 according to KDOQI

Exclusion Criteria:

  • patients with CKD stage 5

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    N acetyl cysteine

    Arm Description

    NAC will be given to cystinosis patients and we will observe the renal function status and a marker of oxidative stress (TBARS)

    Outcomes

    Primary Outcome Measures

    creatinine clearance
    creatinine clearance will be evaluate 6 months beforw and at baseline and after 3 months with NAC
    cystatin c
    Cystinosis patients will be evaluated 6 months before and at baseline and after 3 months after the use of NAC

    Secondary Outcome Measures

    Full Information

    First Posted
    May 19, 2012
    Last Updated
    June 19, 2012
    Sponsor
    University of Sao Paulo
    Collaborators
    Fundação de Amparo à Pesquisa do Estado de São Paulo
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    1. Study Identification

    Unique Protocol Identification Number
    NCT01614431
    Brief Title
    N Acetyl Cysteine for Cystinosis Patients
    Official Title
    N Acetyl Cysteine Can Decrease the Progression of Renal Disease in Cystinosis Patients
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    June 2012
    Overall Recruitment Status
    Completed
    Study Start Date
    March 2011 (undefined)
    Primary Completion Date
    January 2012 (Actual)
    Study Completion Date
    May 2012 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    University of Sao Paulo
    Collaborators
    Fundação de Amparo à Pesquisa do Estado de São Paulo

    4. Oversight

    5. Study Description

    Brief Summary
    This study intends to verify the interference of N acetyl cysteine in the progression of chronic kidney disease in patients with Nephropathic Cystinosis.
    Detailed Description
    Patients with Nephropathic Cystinosis have an increased oxidative stress and go to end-stage renal disease, even when all steps of the treatment are done. Therefore, this study is conducted to verify the interference of the stress oxidative in the progression of the renal disease with the use of an oxidant drug, N acetyl cysteine (NAC). The investigators selected patients with good compliance of the treatment. In these patients the investigators evaluate the serum creatinine, creatinine clearance estimated by Schwartz Formula, cystatin C and a marker of oxidative stress, TBARS ( thiobarbituric acid substances). The variables are analyzed 6 months before the introduction of NAC and the three months after NAC.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Renal Disease, Cystinosis
    Keywords
    renal function, before and after N acetyl cysteine, Cystinosis patients

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    23 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    N acetyl cysteine
    Arm Type
    Experimental
    Arm Description
    NAC will be given to cystinosis patients and we will observe the renal function status and a marker of oxidative stress (TBARS)
    Intervention Type
    Drug
    Intervention Name(s)
    N acetyl cysteine
    Other Intervention Name(s)
    N acety cysteine
    Intervention Description
    N acetyl cysteine to cystinosis patients with CKD stages 1 to 4
    Intervention Type
    Drug
    Intervention Name(s)
    N acetyl cysteine
    Intervention Description
    N acetyl cysteine for cystinosis patients CKD stages 1 to 4
    Primary Outcome Measure Information:
    Title
    creatinine clearance
    Description
    creatinine clearance will be evaluate 6 months beforw and at baseline and after 3 months with NAC
    Time Frame
    change in creatinine clearance evaluated 6 months before , at baseline and after 3 months with NAC
    Title
    cystatin c
    Description
    Cystinosis patients will be evaluated 6 months before and at baseline and after 3 months after the use of NAC
    Time Frame
    change in cystatin C from baseline and after 3 months with NAC

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    1 Year
    Maximum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: cystinosis patients age under 18 years with good compliance to the treatment with Chronic Kidney Disease 1 to 4 according to KDOQI Exclusion Criteria: patients with CKD stage 5
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Maria Helena Vaisbich, Doctor
    Organizational Affiliation
    University of Sao Paulo
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

    N Acetyl Cysteine for Cystinosis Patients

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