N Acetyl Cysteine for Cystinosis Patients

This study has been completed.
Sponsor:
Collaborator:
Fundação de Amparo à Pesquisa do Estado de São Paulo
Information provided by (Responsible Party):
Maria Helena Vaisbich, University of Sao Paulo
ClinicalTrials.gov Identifier:
NCT01614431
First received: May 19, 2012
Last updated: June 19, 2012
Last verified: June 2012
  Purpose

This study intends to verify the interference of N acetyl cysteine in the progression of chronic kidney disease in patients with Nephropathic Cystinosis.


Condition Intervention Phase
Renal Disease
Cystinosis
Drug: N acetyl cysteine
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: N Acetyl Cysteine Can Decrease the Progression of Renal Disease in Cystinosis Patients

Resource links provided by NLM:


Further study details as provided by University of Sao Paulo:

Primary Outcome Measures:
  • creatinine clearance [ Time Frame: change in creatinine clearance evaluated 6 months before , at baseline and after 3 months with NAC ] [ Designated as safety issue: Yes ]
    creatinine clearance will be evaluate 6 months beforw and at baseline and after 3 months with NAC

  • cystatin c [ Time Frame: change in cystatin C from baseline and after 3 months with NAC ] [ Designated as safety issue: Yes ]
    Cystinosis patients will be evaluated 6 months before and at baseline and after 3 months after the use of NAC


Enrollment: 23
Study Start Date: March 2011
Study Completion Date: May 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: N acetyl cysteine
NAC will be given to cystinosis patients and we will observe the renal function status and a marker of oxidative stress (TBARS)
Drug: N acetyl cysteine
N acetyl cysteine to cystinosis patients with CKD stages 1 to 4
Other Name: N acety cysteine
Drug: N acetyl cysteine
N acetyl cysteine for cystinosis patients CKD stages 1 to 4
Other Name: N acetyl cysteine

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.

  Eligibility

Ages Eligible for Study:   1 Year to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT01614431

Sponsors and Collaborators
University of Sao Paulo
Fundação de Amparo à Pesquisa do Estado de São Paulo
Investigators
Principal Investigator: Maria Helena Vaisbich, Doctor University of Sao Paulo
  More Information

No publications provided

Responsible Party: Maria Helena Vaisbich, Clinical Professor, University of Sao Paulo
ClinicalTrials.gov Identifier: NCT01614431     History of Changes
Other Study ID Numbers: Cystinosis and NAC
Study First Received: May 19, 2012
Last Updated: June 19, 2012
Health Authority: Brazil: Ethics Committee

Keywords provided by University of Sao Paulo:
renal function
before and after N acetyl cysteine
Cystinosis patients

Additional relevant MeSH terms:
Cystinosis
Kidney Diseases
Nephrotic Syndrome
Urologic Diseases
Lysosomal Storage Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases
Nephrosis
Acetylcysteine
N-monoacetylcystine
Antiviral Agents
Anti-Infective Agents
Therapeutic Uses
Pharmacologic Actions
Expectorants
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antidotes

ClinicalTrials.gov processed this record on July 22, 2014