SarcoidosisPhase 4REFSNCT01830959
Use of Roflumilast to Prevent Exacerbations in Fibrotic Sarcoidosis Patients (REFS)
A Phase 4 study testing Roflumilast in people with Sarcoidosis, run by University of Cincinnati at 1 site in United States.
Trial summary
Pulmonary sarcoidosis patients with fibrosis often develop recurrent episodes of bronchitis. These can lead to worsening of disease for both the short and long term. Roflumilast has been shown to reduce the number of acute bronchitis episodes in patients with COPD. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Sarcoidosis
- What is being tested?
- Roflumilast
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2013to Oct 2016
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with sarcoidosis as defined by the American Thoracic Society criteria
- Patients with an FEV1/FVC ratio of less than 80%
- Patients with fibrosis on chest x-ray and/or high resolution CT scan.
- Patients have had at least two exacerbations of their sarcoidosis in the prior year. An exacerbation is defined as an acute event requiring increase of prednisone with or without use of antibiotics.
- Patients must be on a stable dose of corticosteroids and other agents for their sarcoidosis at least 4 weeks prior to first visit.
- For patients on prednisone alone, the dose has to be the equivalent of 5 mg prednisone a day. For those on other immunosuppressants, they can be on any dose of prednisone.
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- Patients must be between ages of 18 and 70 years of age.
- Willing to take prednisone at increased dosage for exacerbations of their sarcoidosis.
- Patients must be able to provide written informed consent to participate in the study.
Who cannot join
- Patients with known hypersensitivity to theophylline or pentoxifylline will not be eligible. Patients with dose dependent nausea from these drugs may still participate in the trial.
- Patients will not be able to take theophylline or pentoxifylline during the time of the study. They will be allowed to take drugs for sarcoidosis including prednisone, methotrexate, azathioprine, leflunomide, hydroxychloroquine, thalidomide, infliximab, adalimumab, and rituximab.
- Patients with serum creatinine of greater than 3 mg/dL
- Patients with moderate or severe liver disease as defined Child Pugh class 3 or 4.
- Patients with unstable cardiac disease
- Patients with non cutaneous malignancy treated in the past two years.
- Patients unable to complete the questionnaires and six minute walks detailed in the study.Women of child bearing potential unable to use adequate birth control as determined by the investigator.
Trial locations and contacts
1 site in 1 country.
About this study
Pulmonary sarcoidosis patients with fibrosis often develop recurrent episodes of bronchitis. These can lead to worsening of disease for both the short and long term.
Roflumilast has been shown to reduce the number of acute bronchitis episodes in patients with COPD.
Drugs similar to Roflumilast have been shown to help sarcoidosis. The current study is to determine if Roflumilast will reduce number of episodes of bronchitis and help fibrotic sarcoidosis.
Read the detailed description
This will be a multicenter, double blind, placebo controlled study. Patients would be randomized 1:1 to receive either roflumilast 500 mcg per day or placebo added to their current treatment regimen for twelve months.
- Conditions
- Sarcoidosis
- Being tested
- Roflumilast, Placebo
- Keywords
- Sarcoidosis, fibrosis, bronchiectasis
- Medical subject headings
- Sarcoidosis, Fibrosis, Bronchiectasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RoflumilastDrug
Also called Daliresp
Roflumilast
PlaceboDrug
Placebo one a day
| Study group | Role | What they receive |
|---|---|---|
| Roflumilast | Experimental | Roflumilast Receives: Drug: Roflumilast |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction in number of episodes of acute exacerbation
Collect number of episodes of exacerbtion in one year
Measured: one year
Secondary outcomes (2)
Change in FVC
Changes in FVC over time of the study
Measured: one year
Changes in quality of life
Measure QOL using Sarcoidosis health question, fatigue assessment score, SGRQ, SF-36 Leicester cough questionnaire
Measured: One year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2013 (estimated)
- Study startApr 2013
- Primary completionMay 2016 (estimated)
- Study completionOct 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for SarcoidosisStudies of the same condition, open and under way.
- More trials run by University of CincinnatiThe 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- The Cleveland Clinic, University of Pittsburgh, University of Illinois Chicago, Henry Ford Health System, Albany Medical College
- Responsible party
- Principal investigator, Robert P Baughman, University of Cincinnati
- Investigators
- Robert P Baughman, MD, Study chair, University of Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01830959
- Sponsor's protocol ID
- REFS-1
- Other IDs
- WIRB Pr #: 20130426
- Acronym
- REFS
- Status last verified
- Dec 2015
- First submitted
- 10 Apr 2013
- Last updated
- 8 Dec 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Baughman RP, Judson MA, Culver DA, Birring SS, Parambil J, Zeigler J, Lower EE. Roflumilast (Daliresp(R)) to reduce acute pulmonary events in fibrotic sarcoidosis: a multi-center, double blind, placebo controlled, randomized clinical trial. Sarcoidosis Vasc Diffuse Lung Dis. 2021;38(3):e2021035. doi: 10.36141/svdld.v38i3.11684. Epub 2021 Sep 30.
PubMed 34744427
Study at a glance
From the registry record, last updated by the sponsor on 8 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Roflumilast to Prevent Exacerbations in Fibrotic Sarcoidosis Patients (REFS)
- ClinicalTrials.gov ID
- NCT01830959
- Acronym / sponsor ID
- REFS / REFS-1
- Conditions
- Sarcoidosis
- Keywords
- Sarcoidosis, fibrosis, bronchiectasis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- University of CincinnatiUniversity, hospital or other
- Collaborators
- The Cleveland Clinic, University of Pittsburgh, University of Illinois Chicago, Henry Ford Health System, Albany Medical College
- Study start
- Apr 2013
- Primary completion
- May 2016 (estimated)
- Study completion
- Oct 2016 (estimated)
- First posted
- 12 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Pulmonary sarcoidosis patients with fibrosis often develop recurrent episodes of bronchitis. These can lead to worsening of disease for both the short and long term.
Roflumilast has been shown to reduce the number of acute bronchitis episodes in patients with COPD.
Drugs similar to Roflumilast have been shown to help sarcoidosis. The current study is to determine if Roflumilast will reduce number of episodes of bronchitis and help fibrotic sarcoidosis.
Detailed description
This will be a multicenter, double blind, placebo controlled study. Patients would be randomized 1:1 to receive either roflumilast 500 mcg per day or placebo added to their current treatment regimen for twelve months.
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 |
|---|---|---|
| Roflumilast | Experimental | Drug: RoflumilastRoflumilast |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients with sarcoidosis as defined by the American Thoracic Society criteria
- Patients with an FEV1/FVC ratio of less than 80%
- Patients with fibrosis on chest x-ray and/or high resolution CT scan.
- Patients have had at least two exacerbations of their sarcoidosis in the prior year. An exacerbation is defined as an acute event requiring increase of prednisone with or without use of antibiotics.
- Patients must be on a stable dose of corticosteroids and other agents for their sarcoidosis at least 4 weeks prior to first visit.
Key exclusion criteria (7 listed)
- Patients with known hypersensitivity to theophylline or pentoxifylline will not be eligible. Patients with dose dependent nausea from these drugs may still participate in the trial.
- Patients will not be able to take theophylline or pentoxifylline during the time of the study. They will be allowed to take drugs for sarcoidosis including prednisone, methotrexate, azathioprine, leflunomide, hydroxychloroquine, thalidomide, infliximab, adalimumab, and rituximab.
- Patients with serum creatinine of greater than 3 mg/dL
- Patients with moderate or severe liver disease as defined Child Pugh class 3 or 4.
- Patients with unstable cardiac disease
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 |
|---|---|---|
| RoflumilastAlso: DalirespRoflumilast | Drug | Roflumilast |
| PlaceboPlacebo one a day | Drug | Placebo |
Primary outcome
Reduction in number of episodes of acute exacerbation
Time frame: one year
Collect number of episodes of exacerbtion in one year
2 secondary outcomes
Change in FVC
Time frame: one year
Changes in quality of life
Time frame: One year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Dec 2015.
- Derived from this study: PubMed 34744427Baughman RP, Judson MA, Culver DA, Birring SS, Parambil J, Zeigler J, Lower EE. Roflumilast (Daliresp(R)) to reduce acute pulmonary events in fibrotic sarcoidosis: a multi-center, double blind, placebo controlled, randomized clinical trial. Sarcoidosis Vasc Diffuse Lung Dis. 2021;38(3):e2021035. doi: 10.36141/svdld.v38i3.11684. Epub 2021 Sep 30.
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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.
Robert P Baughman, MD study chair
University of Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Roflumilast in people with Sarcoidosis, run by University of Cincinnati at 1 site 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 9 inclusion and 7 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: status not confirmed, as of the sponsor's last update on 8 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Reduction in number of episodes of acute exacerbation (time frame: one year). 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 NCT01830959, which also lists 1 linked publication.
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 8 Dec 2015.