NCT01614431

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.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
23

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Mar 2011

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2011

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2012

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2012

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

May 19, 2012

Completed
20 days until next milestone

First Posted

Study publicly available on registry

June 8, 2012

Completed
Last Updated

June 20, 2012

Status Verified

June 1, 2012

Enrollment Period

10 months

First QC Date

May 19, 2012

Last Update Submit

June 19, 2012

Conditions

Renal DiseaseCystinosis

Keywords

renal functionbefore and after N acetyl cysteineCystinosis patients

Outcome Measures

Primary Outcomes (2)

  • creatinine clearance

    creatinine clearance will be evaluate 6 months beforw and at baseline and after 3 months with NAC

    change in creatinine clearance evaluated 6 months before , 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

    change in cystatin C from baseline and after 3 months with NAC

Study Arms (1)

N acetyl cysteine

EXPERIMENTAL

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

Interventions

N acetyl cysteineDRUG

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

Also known as: N acety cysteine
N acetyl cysteine

Eligibility Criteria

Age1 Year - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

  • patients with CKD stage 5

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Kidney DiseasesCystinosis

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Urologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesLysosomal Storage DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

CysteineAmino Acids, SulfurSulfur CompoundsOrganic ChemicalsAmino AcidsAmino Acids, Peptides, and Proteins

Study Officials

  • Maria Helena Vaisbich, Doctor

    University of Sao Paulo

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Clinical Professor

Study Record Dates

First Submitted

May 19, 2012

First Posted

June 8, 2012

Study Start

March 1, 2011

Primary Completion

January 1, 2012

Study Completion

May 1, 2012

Last Updated

June 20, 2012

Record last verified: 2012-06