Cystic FibrosisPhase 2NCT00809094
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
A Phase 2 study testing N-acetylcysteine (NAC) in people with Cystic Fibrosis, run by Stanford University at 11 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 7 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- N-acetylcysteine (NAC)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 11 sitesUnited States
- Study dates
- Nov 2008to Feb 2011
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
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- 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 = 85% predicted for age based on the Wang (males < 18 years, females < 16 years) or Hankinson (males > 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)
Who cannot join
- 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 (< 5% increase compared to previous test) for 6 months prior to enrollment
- Current or history of rheumatic or collagen vascular disorders
- Use of NSAIDS other than for chronic therapy within 1 week prior to enrollment
- Initiation of chronic therapy with ibuprofen, azithromycin, TOBI® or Aztreonam within 6 weeks prior to enrollment
- Consumption or inhalation of antioxidants (including NAC, GSH, Immunocal, Nacystelyn, pentoxyfilline) within 6 weeks prior to enrollment
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- Use of oral or IV corticosteroids within 4 weeks prior to enrollment
- Use of acetaminophen within 3 days prior to enrollment
- Unable to forego during the study:
- Vitamin E: more than 400 IU/day for subjects < or = 12 years of age and 800 IU/day for subjects > 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
Trial locations and contacts
11 sites in 1 country.
11 sites in 1 country
United States11 sites
University of Alabama at Birmingham
Birmingham, Alabama, 35294
National Jewish Hospital
Denver, Colorado
Duke Children
Durham, North Carolina
Shands at the University of Florida
Gainesville, Florida
The PennState Milton S Hersey Medical Ctr
Hershey, Pennsylvania
Yale New Haven Hospital
New Haven, Connecticut
Columbia University Medical Ctr
New York, New York
The Children
Philadelphia, Pennsylvania
Children
Pittsburgh, Pennsylvania
University of Utah, Primary Children
Salt Lake City, Utah
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: 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
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.
- Conditions
- Cystic Fibrosis
- Being tested
- N-acetylcysteine (NAC), Placebo
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetylcysteine (NAC)Drug
Also called PharmaNAC
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo was administered oral tablet TID for 24 weeks. Receives: Drug: Placebo |
| N-Acetylcysteine | Active comparator | Participants received 900 mg of oral N-acetylcysteine TID for 24 weeks. Receives: Drug: N-acetylcysteine (NAC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the Logarithm of the Level of Human Neutrophil Elastase (HNE) Activity Measured in Sputum
(change in log10 HNE in the active treatment group) - (change in log10 HNE in the placebo group)
Measured: From enrollment to end of the 24-week trial
Secondary outcomes (4)
Change in FEV1 (Percent of Predicted for Age)
Change in forced expiratory volume in 1 second as compared to normals for age (percent of predicted)
Measured: From enrollment to the end of the 24-week trial
FEV1 (L)
Forced expiratory volume in 1 second (Liters)
Measured: Baseline to end of study (24 weeks)
FEF 25-75% (L/Sec)
Difference in mid-expiratory flow rates between 25 to 75% of the vital capacity, in L/sec measured at the beginning of the study to the end of the study.
Measured: Baseline to end of study (24 weeks)
FEF 25-75% (Percent of Predicted)
Difference in the forced expiratory flow rate in mid-exhalation as a percent of predicted to standard values measured from baseline to the end of study (24 weeks).
Measured: Baseline to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2008 (estimated)
- Study startNov 2008
- Primary completionFeb 2011
- Study completionFeb 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Carol K. Conrad, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00809094
- Sponsor's protocol ID
- SU-12112008-1378
- Status last verified
- Apr 2013
- First submitted
- 15 Dec 2008
- Last updated
- 22 Apr 2013
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 22 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT00809094
- Acronym / sponsor ID
- SU-12112008-1378
- Conditions
- Cystic Fibrosis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Nov 2008
- Primary completion
- Feb 2011 (actual)
- Study completion
- Feb 2011 (actual)
- First posted
- 17 Dec 2008 (estimated)
- Results
- Posted 15 Mar 2013
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo was administered oral tablet TID for 24 weeks. |
| N-Acetylcysteine | Active comparator | Drug: N-acetylcysteine (NAC)Participants received 900 mg of oral N-acetylcysteine TID for 24 weeks. |
Who may qualify
- Ages
- 7 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- Male or female 7 years of age or older
- Diagnosis of CF based upon the following criteria:
- 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 = 85% predicted for age based on the Wang (males < 18 years, females < 16 years) or Hankinson (males > or = 18 years, females > or = 16 years) standardized equations
Key exclusion criteria (18 listed)
- 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 (< 5% increase compared to previous test) for 6 months prior to enrollment
- Current or history of rheumatic or collagen vascular disorders
- Use of NSAIDS other than for chronic therapy within 1 week prior to enrollment
- Initiation of chronic therapy with ibuprofen, azithromycin, TOBI® or Aztreonam within 6 weeks prior to enrollment
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| N-acetylcysteine (NAC)Also: PharmaNAC | Drug | N-Acetylcysteine |
| Placebo | Drug | Placebo |
Primary outcome
Change in the Logarithm of the Level of Human Neutrophil Elastase (HNE) Activity Measured in Sputum
Time frame: From enrollment to end of the 24-week trial
(change in log10 HNE in the active treatment group) - (change in log10 HNE in the placebo group)
4 secondary outcomes
Change in FEV1 (Percent of Predicted for Age)
Time frame: From enrollment to the end of the 24-week trial
FEV1 (L)
Time frame: Baseline to end of study (24 weeks)
FEF 25-75% (L/Sec)
Time frame: Baseline to end of study (24 weeks)
FEF 25-75% (Percent of Predicted)
Time frame: Baseline to 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Apr 2013.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carol K. Conrad principal investigator
Stanford University
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing N-acetylcysteine (NAC) in people with Cystic Fibrosis, run by Stanford University at 11 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Apr 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Mar 2013). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the Logarithm of the Level of Human Neutrophil Elastase (HNE) Activity Measured in Sputum (time frame: From enrollment to end of the 24-week trial). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Apr 2013.