N Acetyl Cysteine for Cystinosis Patients

NCT ID: NCT01614431

Last Updated: 2012-06-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-03-31
• Study Completion Date: 2012-05-31

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.

Conditions

Renal Disease; Cystinosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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)

Group Type: EXPERIMENTAL

N acetyl cysteine

Intervention Type: DRUG

N acetyl cysteine to cystinosis patients with CKD stages 1 to 4

N acetyl cysteine

Intervention Type: DRUG

N acetyl cysteine for cystinosis patients CKD stages 1 to 4

Interventions

N acetyl cysteine

N acetyl cysteine to cystinosis patients with CKD stages 1 to 4

Intervention Type: DRUG

N acetyl cysteine

N acetyl cysteine for cystinosis patients CKD stages 1 to 4

Intervention Type: DRUG

Other Intervention Names

N acety cysteine

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

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fundação de Amparo à Pesquisa do Estado de São Paulo

OTHER_GOV

Sponsor Role: collaborator

University of Sao Paulo

OTHER

Sponsor Role: lead

Responsible Party

Maria Helena Vaisbich

Clinical Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Maria Helena Vaisbich, Doctor

Role: PRINCIPAL_INVESTIGATOR

University of Sao Paulo

Other Identifiers

Cystinosis and NAC

Identifier Type: -

Identifier Source: org_study_id