patient affected by alkaptonuria-ocronosis
Conditions
Interventions
Trade Name: FLUIMUCIL 200
Pharmaceutical Form: Granules
INN or Proposed INN: Acetylcysteine
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Trade Name: CEBION
Sponsors
AZIENDA OSPEDALIERA SENESE
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Inclusion Criteria: - informed consent - Established diagnosis of alkaptonuria - Age:18-75 Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Exclusion criteria - NAC intolerance - History of malignacy or neoplastic disease - pregnant or lactanting females - chronic or acute renal disease - values of aspartate aminotrasferase (AST), alanine aminotrasferase (ALT) or bilirubin > 2 x upper limit of normal (ULN) -history of excessive alcohol consuption or chronic alcohol induced disease -patients who were treated with ascorbic acid or other antioxidant agents in the previous 30 days - disorders that may interfere with the interpretation of the outcome data
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluation of NAC effectiveness to inhibit HGA oxidation and its polymerization within connective tissue in patients affected by Ochronosis;Secondary Objective: Evaluation of NAC treatment on 1)plasmatic and urinary parameters of oxidative stress 2) radiologic assessment of joint damage;Primary end point(s): Evaluation of NAC effect on plasmatic and urinary levels of homogentisic acid in patients with homogentisic acid oxidase (HGO) deficiency. | — |
Countries
Italy
Outcome results
None listed