Pulmonary SarcoidosisPhase 3NCT00262132
Mycophenolate for Pulmonary Sarcoidosis
A Phase 3 study testing Mycophenolate in people with Pulmonary Sarcoidosis, run by Medical University of South Carolina at 1 site in United States.
Trial summary
Corticosteroids are presently the drug of choice for the treatment of pulmonary sarcoidosis. However, corticosteroids are associated with many significant side effects. For this reason, it would be beneficial to find an alternative agent to corticosteroids for the treatment of pulmonary sarcoidosis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pulmonary Sarcoidosis
- What is being tested?
- Mycophenolate
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2003to Aug 2006
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- New acute pulmonary sarcoidosis defined as a FVC or FEV1 less than 80 percent of predicted plus symptoms of worsening dyspnea, cough, chest pain, or wheezing in patients without a prior history of pulmonary sarcoidosis. All such patients will also require bronchoscopy with transbronchial biopsy that demonstrates noncaseating granulomas of unknown cause. Such patients are usually treated for acute pulmonary sarcoidosis. The transbronchial biopsy specimens must have negative stains for mycobacteria and fungi.
- > 18 years of age
- Signing a written informed consent form.
Who cannot join
- Previous history of an adverse reaction to mycophenolate.
- Current use of another immunosuppressive medication.
- History of active hepatitis or another significant liver disease with the exception of proven or presumed sarcoidosis of the liver.
- Previous history of skin cancer
- Active peptic ulcer disease
- Pregnant and/ or lactating female
Show all 19Show fewer
- Serum creatinine > 2.0 mg/dl
- Serum bilirubin > 3.0 mg/dl
- WBC < 4,000/cu mm3
- Has demonstrated non-compliance in the past
- Current alcohol or drug abuse
- Evidence of significant sarcoidosis in extrapulmonary organs that requires therapy such that the mycophenolate doses could not be tapered if the pulmonary sarcoidosis was stable or improved.
- History of previous severe digestive system disease
- Patients taking azathioprine
- Patients taking cholestyramine or other drugs that affect enterohepatic recirculation
- Patients with Lesch-Nyhan or Kelley-Seegmiller syndrome
- Patients be willing not to receive live vaccines during the study
- Patients with phenylketonuria
- Patients with elevated serum transaminases or total bilirubin at baseline
Trial locations and contacts
1 site in 1 country.
United States1 site
Medical University of South Carolina
Charleston, South Carolina, 29425
About this study
Corticosteroids are presently the drug of choice for the treatment of pulmonary sarcoidosis. However, corticosteroids are associated with many significant side effects. For this reason, it would be beneficial to find an alternative agent to corticosteroids for the treatment of pulmonary sarcoidosis. This study is an open label trial of mycophenolate for new onset pulmonary sarcoidosis. Patients are candidates for this study if they have biopsy proven pulmonary sarcoidosis and a vital capacity or FEV1 less than 80% of predicted. Patients must undergo bronchoscopy where not only is the diagnosis of pulmonary sarcoidosis required, but in addition, cells are obtained from bronchoalveolar lavage. If the patients are diagnosed with pulmonary sarcoidosis, they are placed on an initial dose of 500mg BID of mycophenolate for 1 week. If their blood counts are not affected on this dose and they have no significant symptoms that are thought to be drug related, then their dose is escalated to 1g BID for the remaining 9 weeks of the study (the total study drug therapy time is 10 weeks). The patients are followed with multiple study visits. At these visits blood tests are drawn to make sure that there are no significant side effects from mycophenolate. In addition, the patients have a history and physical performed to evaluate the clinical state of their sarcoidosis and to detect mycophenolate side effects. On completion of 10 weeks of mycophenolate therapy, the patients undergo a second bronchoscopy with bronchoalveolar lavage to obtain cells for analysis. The patients are evaluated with spirometry, measurements of shortness of breath (dyspnea), and a quality of life scale (SF36) at serial visits during their study. The primary endpoint of the study is improved, unchanged or worse FVC. It is hoped that this pilot study will suggest that mycophenolate is a reasonable treatment option for new onset pulmonary sarcoidosis.
- Conditions
- Pulmonary Sarcoidosis
- Being tested
- Mycophenolate
- Medical subject headings
- Sarcoidosis, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MycophenolateDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2005 (estimated)
- Study startSep 2003
- Primary completionAug 2006
- Study completionAug 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pulmonary SarcoidosisStudies of the same condition, open and under way.
- More trials run by Medical University of South CarolinaThe 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
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Collaborators
- Hoffmann-La Roche
- Investigators
- Marc A Judson, MD, Principal investigator, Medical University of South CarolinaDon C Rockey, MD, Study chair, Medical University of South Carolina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00262132
- Sponsor's protocol ID
- CEL401
- Other IDs
- HR 11031
- Status last verified
- Apr 2018
- First submitted
- 5 Dec 2005
- Last updated
- 18 Jun 2018
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mycophenolate for Pulmonary Sarcoidosis
- ClinicalTrials.gov ID
- NCT00262132
- Acronym / sponsor ID
- CEL401
- Conditions
- Pulmonary Sarcoidosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 20 participants
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- Hoffmann-La Roche
- Study start
- Sep 2003
- Primary completion
- Aug 2006 (actual)
- Study completion
- Aug 2006 (actual)
- First posted
- 6 Dec 2005 (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.
Corticosteroids are presently the drug of choice for the treatment of pulmonary sarcoidosis. However, corticosteroids are associated with many significant side effects. For this reason, it would be beneficial to find an alternative agent to corticosteroids for the treatment of pulmonary sarcoidosis. This study is an open label trial of mycophenolate for new onset pulmonary sarcoidosis. Patients are candidates for this study if they have biopsy proven pulmonary sarcoidosis and a vital capacity or FEV1 less than 80% of predicted. Patients must undergo bronchoscopy where not only is the diagnosis of pulmonary sarcoidosis required, but in addition, cells are obtained from bronchoalveolar lavage. If the patients are diagnosed with pulmonary sarcoidosis, they are placed on an initial dose of 500mg BID of mycophenolate for 1 week. If their blood counts are not affected on this dose and they have no significant symptoms that are thought to be drug related, then their dose is escalated to 1g BID for the remaining 9 weeks of the study (the total study drug therapy time is 10 weeks). The patients are followed with multiple study visits. At these visits blood tests are drawn to make sure that there are no significant side effects from mycophenolate. In addition, the patients have a history and physical performed to evaluate the clinical state of their sarcoidosis and to detect mycophenolate side effects. On completion of 10 weeks of mycophenolate therapy, the patients undergo a second bronchoscopy with bronchoalveolar lavage to obtain cells for analysis. The patients are evaluated with spirometry, measurements of shortness of breath (dyspnea), and a quality of life scale (SF36) at serial visits during their study. The primary endpoint of the study is improved, unchanged or worse FVC. It is hoped that this pilot study will suggest that mycophenolate is a reasonable treatment option for new onset pulmonary sarcoidosis.
Study design
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- New acute pulmonary sarcoidosis defined as a FVC or FEV1 less than 80 percent of predicted plus symptoms of worsening dyspnea, cough, chest pain, or wheezing in patients without a prior history of pulmonary sarcoidosis. All such patients will also require bronchoscopy with transbronchial biopsy that demonstrates noncaseating granulomas of unknown cause. Such patients are usually treated for acute pulmonary sarcoidosis. The transbronchial biopsy specimens must have negative stains for mycobacteria and fungi.
- > 18 years of age
- Signing a written informed consent form.
Key exclusion criteria (19 listed)
- Previous history of an adverse reaction to mycophenolate.
- Current use of another immunosuppressive medication.
- History of active hepatitis or another significant liver disease with the exception of proven or presumed sarcoidosis of the liver.
- Previous history of skin cancer
- Active peptic ulcer 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 |
|---|---|---|
| Mycophenolate | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2018.
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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.
Marc A Judson, MD principal investigator
Medical University of South Carolina
Don C Rockey, MD study chair
Medical University of South Carolina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Mycophenolate in people with Pulmonary Sarcoidosis, run by Medical University of South Carolina 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 3 inclusion and 19 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: stopped early, as of the sponsor's last update on 18 Jun 2018. 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. 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?
The study was stopped early. 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.
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 18 Jun 2018.