Phase 2
Completed N=70
NAC Phase IIB: A Multi-Center, Phase IIB, Randomized, Placebo-controlled, Double-Blind Study Of The Effects Of N-Acetylcysteine On Redox Changes and Lung Inflammation In Cystic Fibrosis Patients
Source: ClinicalTrials.gov NCT00809094 ↗Enrolled (actual)
70
Serious AEs
45.7%
Results posted
Mar 2013
Primary outcomePrimary: Change in the Logarithm of the Level of Human Neutrophil Elastase (HNE) Activity Measured in Sputum — 0.03; -0.17 log10 mcg/mL — p=0.14
Summary
This Phase IIB proof-of-concept study would examine the effects of an investigational product called N-acetylcysteine (NAC) on the basic processes that cause inflammation in CF lung disease. We hope to learn more about the causes of lung disease in cystic fibrosis by studying the characteristics of the inflammation in the lungs of patients who have CF.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in the Logarithm of the Level of Human Neutrophil Elastase (HNE) Activity Measured in Sputum |
0.03; -0.17 | 0.14 |
| SECONDARY Change in FEV1 (Percent of Predicted for Age) |
1.055; -5.62 | — |
| SECONDARY FEV1 (L) |
2.5; -4.35 | — |
| SECONDARY FEF 25-75% (L/Sec) |
0.08; -0.13 | — |
| SECONDARY FEF 25-75% (Percent of Predicted) |
1.33; -3.81 | — |
Eligibility Criteria
Inclusion Criteria
- Male or female 7 years of age or older
- Diagnosis of CF based upon the following criteria:
- One or more clinical features characteristic of CF AND (b or c)
- Positive sweat test > 60 mEq/L by quantitative pilocarpine iontophoresis
- A genotype with two identifiable mutations consistent with CF
- Written informed consent (and assent when applicable) obtained from subject or subject's legal representative
- Clinically stable with no evidence of acute upper or lower respiratory tract infection within 4 weeks prior to enrollment
- Stable mild or moderately severe lung disease defined by an FEV1 > or = 40% and or = 18 years, females > or = 16 years) standardized equations
- Able to tolerate sputum induction with 3% hypertonic saline and to expectorate
- Able to perform repeatable, consistent efforts in pulmonary function testing
- Weight > or = 25 kg at time of enrollment
- Females of child bearing potential must be willing to use birth control (IUD, oral, transdermal, or parenteral contraceptives; abstinence)
Exclusion Criteria
- Clinically significant liver enzymes (AST, ALT or GGT) > 2.5 times the upper limit of normal at screening
- History of ABPA, unless have evidence of a stable IgE ( 12 years of age
- Vitamin C: more than 0.5 gm/day
- More than two alcoholic drinks per day
- Known hypersensitivity to oral PharmaNAC®
- Current cigarette consumption
- Pregnant or breastfeeding
- Subject unlikely to complete the study as determined by the Investigator
- Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the subject
- Participation in trials for other anti-inflammatory or therapeutic investigational drugs within 6 weeks prior to enrollment
Data sourced from ClinicalTrials.gov (NCT00809094). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.