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
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
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
NCT ID
NCT01614431
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
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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