N Acetyl Cysteine for Cystinosis Patients

University of Sao Paulo

Status and phase: Completed; Phase 4

Conditions: Cystinosis; Renal Disease

Treatments: Drug: N acetyl cysteine

Study type: Interventional

Funder types: Other

Identifiers

NCT01614431

Cystinosis and NAC

Details and patient eligibility

About

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

Full 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.

Enrollment: 23 patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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23 participants in 1 patient group

N acetyl cysteine

Experimental group

Description:

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

Treatment:

Drug: N acetyl cysteine

Drug: N acetyl cysteine