Pulmonary FibrosisPhase 3PANTHER-IPFNCT00650091
Evaluating the Effectiveness of Prednisone, Azathioprine, and N-acetylcysteine in Patients With IPF
A Phase 3 study testing N-acetylcysteine (NAC) in people with Pulmonary Fibrosis, run by Duke University at 26 sites in United States.
Trial summary
Idiopathic pulmonary fibrosis (IPF) is a long-term lung disease that affects an individual's ability to breathe. In this randomized, double-blind, placebo-controlled trial, we assigned patients with idiopathic pulmonary fibrosis who had mild-to-moderate lung-function impairment to one of three groups - receiving a… Read more about this study
- Who is it for?
- Ages 35 to 85 years
- Conditions the study is for
- Pulmonary Fibrosis
- What is being tested?
- N-acetylcysteine (NAC)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 264 participants
- Where
- 26 sitesUnited States
- Study dates
- Oct 2009to Jan 2014
- Phase
- Phase 3
- 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 35 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Forced vital capacity (FVC) greater than or equal to 50% of predicted value
- Diffusion capacity (DLCO) greater than or equal to 30% of predicted value
- Diagnosis of IPF by modified American Thoracic Society (ATS) criteria in the 48 months before study entry
Who cannot join
- History of clinically significant environmental exposure known to cause pulmonary fibrosis
- Diagnosis of connective tissue disease as the likely cause of the interstitial disease
- Extent of emphysema greater than the extent of fibrotic change (i.e., honeycombing, reticular changes) on high resolution computed tomography (HRCT) scan
- Forced expiratory volume in 1 second (FEV1)/FVC ratio less than 0.65 at the time of screening (post-bronchodilator)
- Partial pressure of arterial oxygen (PaO2) less than 55 mm Hg (less than 50 mm Hg at Denver study site)
- Residual volume greater than 120% predicted at the time of screening (post-bronchodilator)
Show all 34Show fewer
- Evidence of active infection
- Significant bronchodilator response on screening spirometry, defined as change in FEV1 greater than or equal to 12% and absolute change greater than 200 mL OR change in FVC greater than or equal to 12% and absolute change greater than 200 mL
- Screening and baseline FVC measurements (in liters, post-bronchodilator) differing by 11%
- Listed for lung transplantation
- History of unstable or deteriorating cardiac disease
- Heart attack, coronary artery bypass, or angioplasty in the 6 months before study entry
- Unstable angina pectoris or congestive heart failure requiring hospitalization in the 6 months before study entry
- Uncontrolled arrhythmia
- Severe uncontrolled high blood pressure
- Known HIV or hepatitis C
- Known cirrhosis and chronic active hepatitis
- Active substance and/or alcohol abuse
- Pregnant or breastfeeding
- Women of childbearing potential who are not using a medically approved means of contraception
- Any clinically relevant lab abnormalities, including the following:
- Creatinine greater than twice the upper limit of normal (ULN)
- Hematology outside of specified limits
- White blood cells less than 3,500/mm3
- Hematocrit less than 25% or greater than 59%
- Platelets less than 100,000 mm3 at the time of screening
- Any of the following liver function test criteria above specified limits
- Total bilirubin greater than twice the ULN
- Aspartate (AST) or alanine aminotransferases (ALT) greater than 1.5 the ULN
- Alkaline phosphatase greater than three times the ULN
- Albumin less than 3.0 mg/dL at the time of screening
- Known hypersensitivity to study medication
- Any condition other than IPF that, in the opinion of the site PI, is likely to result in death in the 1 year after study entry
- Any condition that, in the judgment of the PI, might cause participation in this study to be detrimental or makes the person a poor candidate for the study
Trial locations and contacts
26 sites in 1 country.
26 sites in 1 country
United States26 sites
University of Michigan
Ann Arbor, Michigan, 48109
University of Alabama - Birmingham
Birmingham, Alabama, 35294
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Medical University of South Carolina
Charleston, South Carolina, 29425
St. Luke's Hospital
Chesterfield, Missouri, 63107
University of Chicago
Chicago, Illinois, 60637
Cleveland Clinic
Cleveland, Ohio, 44195
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
National Jewish Medical and Research Center
Denver, Colorado, 80206
Duke Universtiy
Durham, North Carolina, 27705
University of California - Los Angeles
Los Angeles, California, 90095
University of Louisville
Louisville, Kentucky, 40425
University of Miami Miller School of Medicine
Miami, Florida, 33136
Vanderbilt University
Nashville, Tennessee, 37232
Yale University School of Medicine
New Haven, Connecticut, 06520
Tulane University
New Orleans, Louisiana, 70118
Weill Medical College of Cornell University
New York, New York, 10021
Mount Sinai Hospital
New York, New York, 10029
University of Pennsylvania Health System
Philadelphia, Pennsylvania, 19104
Temple University
Philadelphia, Pennsylvania, 19140
Mayo Clinic
Rochester, Minnesota, 55905
Highland Hospital - University of Rochester Medical Center
Rochester, New York, 14620
University of Utah Health Research Center
Salt Lake City, Utah, 84108
University of California - San Francisco
San Francisco, California, 94110
University of Washington
Seattle, Washington, 98165
About this study
Official title: Prednisone, Azathioprine, and N-acetylcysteine: A Study That Evaluates Response in IPF
Idiopathic pulmonary fibrosis (IPF) is a long-term lung disease that affects an individual's ability to breathe. In this randomized, double-blind, placebo-controlled trial, we assigned patients with idiopathic pulmonary fibrosis who had mild-to-moderate lung-function impairment to one of three groups - receiving a combination of prednisone, azathioprine, and NAC (combination therapy), NAC alone, or placebo - in a 1:1:1 ratio.
Read the detailed description
IPF is a disease with widespread and permanent scarring of lung tissue which eventually results in death. Individuals with IPF may experience breathing difficulties, cough, chest pain, and a decreased exercise capacity. Although the cause of IPF is unknown, it may be a result of an inflammatory response to an unknown substance. NAC, an antioxidant that is effective at loosening up mucus that forms in the lungs, may improve lung function. The purpose of this study is to evaluate the effectiveness of NAC at preventing the loss of lung function in people with IPF.
In the initial double-blind, placebo-controlled trial, subjects who have idiopathic pulmonary fibrosis with mild-to-moderate impairment in pulmonary function are randomly assigned to receive a three-drug regimen of prednisone, azathioprine, and acetylcysteine; acetylcysteine alone; or placebo.
After safety concerns were identified by the data and safety monitoring board, the three-drug regimen was stopped by the National Heart, Lung, and Blood Institute (NHLBI) on October 14, 2011, and a clinical alert was issued. After a brief period of interruption for modification of the protocol and approval by the institutional review boards, patients continued to be recruited for the acetylcysteine group and the placebo group and were followed for the pre-specified duration of 60 weeks.
Study visits will occur at baseline and Weeks 4, 15, 30, 45, and 60. At all study visits, a physical exam and blood collection will occur. At selected visits, the following study procedures will occur: lung function testing; urine collection; a 6-minute walk test, and questionnaires to assess health status, breathing, and quality of life. Participants will record medication usage and symptoms in a daily diary.
- Conditions
- Pulmonary Fibrosis
- Being tested
- N-acetylcysteine (NAC), Placebo
- Keywords
- Idiopathic Pulmonary Fibrosis, Prednisone, Azathioprine, NAC, N-acetylcysteine
- Medical subject headings
- Pulmonary Fibrosis, Idiopathic Pulmonary Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-acetylcysteine (NAC)Drug
Participants will receive 600 mg of NAC three times a day.
PlaceboDrug
Participants will receive placebo each day.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Participants will receive N-acetylcysteine (NAC) for 60 weeks. Receives: Drug: N-acetylcysteine (NAC) |
| 2 | Placebo comparator | Participants will receive placebo for 60 weeks. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Change in Forced Vital Capacity
Change from Baseline in Forced Vital Capacity at 60 weeks (units in liters)
Measured: Measured as the estimated change from baseline to Week 60
Secondary outcomes (4)
Disease Progression
The time-to-death or a 10% decline in FVC will be defined as the time-to-disease progression.
The 10% decline in FVC from enrollment must be confirmed on 2 consecutive visits no less than 6 weeks apart. For subjects with 2 consecutive visits with a 10% decline in FVC, the time-to-disease progression will be defined as the time interval between enrollment and the initial visit with a 10% FVC decline.
Measured: Measured at Week 60
Acute Exacerbations
The following 3 criteria will define acute exacerbations in subjects with acute worsening of their respiratory conditions:
- Clinical (all of the following required): A) Unexplained worsening of dyspnea or cough within 30 days, triggering unscheduled medical care (e.g., emergency room, clinic, study visit, hospitalization). B) No clinical suspicion or overt evidence of cardiac event, pulmonary embolism, or deep venous thrombosis to explain acute worsening of dyspnea. C) No pneumothorax.
Measured: Measured at Week 60
Respiratory Infections
Measured: Measured at Week 60
Number of Participants With Maintained Forced Vital Capacity Response
Maintained forced vital capacity response was a binary variable taking on a value of 1 for participants with higher FVC % predicted at week 60 compared to baseline.
Measured: Measured at Week 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2008 (estimated)
- Study startOct 2009
- Primary completionOct 2013
- Study completionJan 2014
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.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Pulmonary FibrosisStudies of the same condition, open and under way.
- More trials run by Duke 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 264 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Marvin I Schwarz, MD, Study chair, University of Colorado, DenverKevin Brown, MD, Principal investigator, National Jewish HealthRob Kaner, MD, Principal investigator, Weill Medical College at Cornell UniversityTalmadge King, MD, Principal investigator, University of California, San FranciscoJoe Lasky, MD, Principal investigator, Tulane UniversityJames Loyd, MD, Principal investigator, Vanderbilt UniversityFernando Martinez, MD, Principal investigator, University of MichiganImre Noth, MD, Principal investigator, University of Chicago
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00650091
- Sponsor's protocol ID
- Pro00020066
- Other IDs
- U10HL080413-03
- Acronym
- PANTHER-IPF
- Status last verified
- Feb 2015
- First submitted
- 28 Mar 2008
- Last updated
- 2 Jun 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Allen RJ, Stockwell A, Oldham JM, Guillen-Guio B, Schwartz DA, Maher TM, Flores C, Noth I, Yaspan BL, Jenkins RG, Wain LV; International IPF Genetics Consortium. Genome-wide association study across five cohorts identifies five novel loci associated with idiopathic pulmonary fibrosis. Thorax. 2022 Aug;77(8):829-833. doi: 10.1136/thoraxjnl-2021-218577. Epub 2022 Jun 10.
PubMed 35688625Andrade J, Schwarz M, Collard HR, Gentry-Bumpass T, Colby T, Lynch D, Kaner RJ; IPFnet Investigators. The Idiopathic Pulmonary Fibrosis Clinical Research Network (IPFnet): diagnostic and adjudication processes. Chest. 2015 Oct;148(4):1034-1042. doi: 10.1378/chest.14-2889.
PubMed 26111071Durheim MT, Collard HR, Roberts RS, Brown KK, Flaherty KR, King TE Jr, Palmer SM, Raghu G, Snyder LD, Anstrom KJ, Martinez FJ; IPFnet investigators. Association of hospital admission and forced vital capacity endpoints with survival in patients with idiopathic pulmonary fibrosis: analysis of a pooled cohort from three clinical trials. Lancet Respir Med. 2015 May;3(5):388-96. doi: 10.1016/S2213-2600(15)00093-4. Epub 2015 Apr 15.
PubMed 25890798Idiopathic Pulmonary Fibrosis Clinical Research Network; Martinez FJ, de Andrade JA, Anstrom KJ, King TE Jr, Raghu G. Randomized trial of acetylcysteine in idiopathic pulmonary fibrosis. N Engl J Med. 2014 May 29;370(22):2093-101. doi: 10.1056/NEJMoa1401739. Epub 2014 May 18.
PubMed 24836309Idiopathic Pulmonary Fibrosis Clinical Research Network; Raghu G, Anstrom KJ, King TE Jr, Lasky JA, Martinez FJ. Prednisone, azathioprine, and N-acetylcysteine for pulmonary fibrosis. N Engl J Med. 2012 May 24;366(21):1968-77. doi: 10.1056/NEJMoa1113354. Epub 2012 May 20.
PubMed 22607134
Study at a glance
From the registry record, last updated by the sponsor on 2 Jun 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prednisone, Azathioprine, and N-acetylcysteine: A Study That Evaluates Response in IPF
- ClinicalTrials.gov ID
- NCT00650091
- Acronym / sponsor ID
- PANTHER-IPF / Pro00020066
- Conditions
- Pulmonary Fibrosis
- Keywords
- Idiopathic Pulmonary Fibrosis, Prednisone, Azathioprine, NAC, N-acetylcysteine
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 264 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Oct 2009
- Primary completion
- Oct 2013 (actual)
- Study completion
- Jan 2014 (actual)
- First posted
- 1 Apr 2008 (estimated)
- Results
- Posted 2 Jun 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Idiopathic pulmonary fibrosis (IPF) is a long-term lung disease that affects an individual's ability to breathe. In this randomized, double-blind, placebo-controlled trial, we assigned patients with idiopathic pulmonary fibrosis who had mild-to-moderate lung-function impairment to one of three groups - receiving a combination of prednisone, azathioprine, and NAC (combination therapy), NAC alone, or placebo - in a 1:1:1 ratio.
Detailed description
IPF is a disease with widespread and permanent scarring of lung tissue which eventually results in death. Individuals with IPF may experience breathing difficulties, cough, chest pain, and a decreased exercise capacity. Although the cause of IPF is unknown, it may be a result of an inflammatory response to an unknown substance. NAC, an antioxidant that is effective at loosening up mucus that forms in the lungs, may improve lung function. The purpose of this study is to evaluate the effectiveness of NAC at preventing the loss of lung function in people with IPF.
In the initial double-blind, placebo-controlled trial, subjects who have idiopathic pulmonary fibrosis with mild-to-moderate impairment in pulmonary function are randomly assigned to receive a three-drug regimen of prednisone, azathioprine, and acetylcysteine; acetylcysteine alone; or placebo.
After safety concerns were identified by the data and safety monitoring board, the three-drug regimen was stopped by the National Heart, Lung, and Blood Institute (NHLBI) on October 14, 2011, and a clinical alert was issued. After a brief period of interruption for modification of the protocol and approval by the institutional review boards, patients continued to be recruited for the acetylcysteine group and the placebo group and were followed for the pre-specified duration of 60 weeks.
Study visits will occur at baseline and Weeks 4, 15, 30, 45, and 60. At all study visits, a physical exam and blood collection will occur. At selected visits, the following study procedures will occur: lung function testing; urine collection; a 6-minute walk test, and questionnaires to assess health status, breathing, and quality of life. Participants will record medication usage and symptoms in a daily diary.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Drug: N-acetylcysteine (NAC)Participants will receive N-acetylcysteine (NAC) for 60 weeks. |
| 2 | Placebo comparator | Drug: PlaceboParticipants will receive placebo for 60 weeks. |
Who may qualify
- Ages
- 35 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Forced vital capacity (FVC) greater than or equal to 50% of predicted value
- Diffusion capacity (DLCO) greater than or equal to 30% of predicted value
- Diagnosis of IPF by modified American Thoracic Society (ATS) criteria in the 48 months before study entry
Key exclusion criteria (34 listed)
- History of clinically significant environmental exposure known to cause pulmonary fibrosis
- Diagnosis of connective tissue disease as the likely cause of the interstitial disease
- Extent of emphysema greater than the extent of fibrotic change (i.e., honeycombing, reticular changes) on high resolution computed tomography (HRCT) scan
- Forced expiratory volume in 1 second (FEV1)/FVC ratio less than 0.65 at the time of screening (post-bronchodilator)
- Partial pressure of arterial oxygen (PaO2) less than 55 mm Hg (less than 50 mm Hg at Denver study site)
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)Participants will receive 600 mg of NAC three times a day. | Drug | 1 |
| PlaceboParticipants will receive placebo each day. | Drug | 2 |
Primary outcome
Overall Change in Forced Vital Capacity
Time frame: Measured as the estimated change from baseline to Week 60
Change from Baseline in Forced Vital Capacity at 60 weeks (units in liters)
4 secondary outcomes
Disease Progression
Time frame: Measured at Week 60
Acute Exacerbations
Time frame: Measured at Week 60
Respiratory Infections
Time frame: Measured at Week 60
Number of Participants With Maintained Forced Vital Capacity Response
Time frame: Measured at Week 60
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jun 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35688625Allen RJ, Stockwell A, Oldham JM, Guillen-Guio B, Schwartz DA, Maher TM, Flores C, Noth I, Yaspan BL, Jenkins RG, Wain LV; International IPF Genetics Consortium. Genome-wide association study across five cohorts identifies five novel loci associated with idiopathic pulmonary fibrosis. Thorax. 2022 Aug;77(8):829-833. doi: 10.1136/thoraxjnl-2021-218577. Epub 2022 Jun 10.
- Derived from this study: PubMed 26111071Andrade J, Schwarz M, Collard HR, Gentry-Bumpass T, Colby T, Lynch D, Kaner RJ; IPFnet Investigators. The Idiopathic Pulmonary Fibrosis Clinical Research Network (IPFnet): diagnostic and adjudication processes. Chest. 2015 Oct;148(4):1034-1042. doi: 10.1378/chest.14-2889.
- Derived from this study: PubMed 25890798Durheim MT, Collard HR, Roberts RS, Brown KK, Flaherty KR, King TE Jr, Palmer SM, Raghu G, Snyder LD, Anstrom KJ, Martinez FJ; IPFnet investigators. Association of hospital admission and forced vital capacity endpoints with survival in patients with idiopathic pulmonary fibrosis: analysis of a pooled cohort from three clinical trials. Lancet Respir Med. 2015 May;3(5):388-96. doi: 10.1016/S2213-2600(15)00093-4. Epub 2015 Apr 15.
- Derived from this study: PubMed 24836309Idiopathic Pulmonary Fibrosis Clinical Research Network; Martinez FJ, de Andrade JA, Anstrom KJ, King TE Jr, Raghu G. Randomized trial of acetylcysteine in idiopathic pulmonary fibrosis. N Engl J Med. 2014 May 29;370(22):2093-101. doi: 10.1056/NEJMoa1401739. Epub 2014 May 18.
- Derived from this study: PubMed 22607134Idiopathic Pulmonary Fibrosis Clinical Research Network; Raghu G, Anstrom KJ, King TE Jr, Lasky JA, Martinez FJ. Prednisone, azathioprine, and N-acetylcysteine for pulmonary fibrosis. N Engl J Med. 2012 May 24;366(21):1968-77. doi: 10.1056/NEJMoa1113354. Epub 2012 May 20.
- Clinical Alert: Commonly used three-drug regimen for idiopathic pulmonary fibrosis found harmfulLink provided by the sponsor.
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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.
Marvin I Schwarz, MD study chair
University of Colorado, Denver
Kevin Brown, MD principal investigator
National Jewish Health
Rob Kaner, MD principal investigator
Weill Medical College at Cornell University
26 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing N-acetylcysteine (NAC) in people with Pulmonary Fibrosis, run by Duke University at 26 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 3 inclusion and 34 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 2 Jun 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 2 Jun 2015). 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?
Overall Change in Forced Vital Capacity (time frame: Measured as the estimated change from baseline to Week 60). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00650091, which also lists 5 linked publications.
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 2 Jun 2015.