SarcoidosisPhase 2BOPSACNCT00926627
Safety and Efficacy Study of Bosentan in Progressive Pulmonary Sarcoidosis
A Phase 2 study testing bosentan in people with Sarcoidosis and Pulmonary Hypertension, run by Daniel Doberer at 2 sites in Austria.
Trial summary
Progressive pulmonary sarcoidosis occurs in up to twenty percent of patients who require persistent treatment, but available treatment options have shown considerable long-term toxicity and uncertain or unproven efficacy. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Sarcoidosis, Pulmonary Hypertension
- What is being tested?
- bosentan
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 32 participants
- Where
- 2 sitesAustria
- Study dates
- Apr 2009to Mar 2010
- Phase
- Phase 2
- 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
- Signed informed consent prior to any study-mandated procedure.
- Male and female patients aged > 18 and < 70 years.
- Histologically proven sarcoidosis diagnosed at least one year before screening.
- Diagnosis of sarcoidosis and with evidence of pulmonary parenchymal disease on chest X-ray or CT (radiological stage II, III) with or without pulmonary hypertension. Subjects with concurrent extrapulmonary sarcoidosis are encouraged to be enrolled.
- Progressive disease, defined as follows:
- Deterioration in the 3-12 month period prior to screening in at least two of the following criteria:
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- increase in clinical symptoms (cough, shortness of breath, chest pain, fatigue or hemoptysis).
- lung function: decrease of 10% in TLC, FVC or DLCO.
- worsening of radiographic opacities.
- Have been receiving pre-study treatment with prednisolone (or equivalent dose of corticosteroid) as a single agent (≥ 10 mg/day) or other immunosuppressants (methotrexate, azathioprine, cyclophosphamide, TNF inhibitors, etc.) within the 3-month period immediately prior to screening. Patients must be on a stable dose of these medications for > 4 weeks before starting the study medication.
- AST and ALT values within three times upper limit of normal.
- Ability to communicate well with the investigator, in the local language, and to understand and comply with the requirements of the study.
- Negative pregnancy test in female patients.
- Adequate contraception in female patients of childbearing age.
Who cannot join
- Known hypersensitivity to any excipients of the drug formulation or to bosentan.
- Treatment with another investigational drug within 3 months prior to screening.
- Pulmonary sarcoidosis:
- without disease progression as defined above
- with radiological stage I
- with radiological stage IV (pulmonary fibrosis with evidence of honey-combing, hilar retraction, bullae and cysts)
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- Other cause of pulmonary disease:
- Active tuberculosis (or positive Quantiferon test), fungi infection, lymphoma.
- Chronic obstructive pulmonary disease, asthma, interstitial lung disease other than sarcoid-related
- Anamnesis of beryllium or asbestos exposition
- Previous smoking (> 10 PY), or active smoker
- Previous administration of bosentan
- Positive results from the hepatitis serology, except for vaccinated subjects, at screening.
- Positive results from the HIV serology at screening.
- Malignancy requiring chemotherapy or radiation
- Uncontrolled other disease like
- Chronic heart failure (NYHA III, IV)
- Diabetes mellitus (blood glucose 2x per day > 250 mg/dl , HbA1c > 10 %)
- Arterial hypertension (SBP > 180 mmHg)
- Concomitant treatment with cyclosporine A
- Concomitant treatment with tacrolimus or sirolimus
- Concomitant treatment with glibenclamide
- Are pregnant, nursing, or planning pregnancy during the trial or within six month period thereafter.
- Have a known substance dependency (drug or alcohol within 3 years of screening).
- Presumed non-compliance.
- Legal incapacity or limited legal capacity at screening.
Trial locations and contacts
2 sites in 1 country.
Austria2 sites
General Hospital Vienna
Vienna, State of Vienna, 1090
Wilhelminenspital Wien
Vienna, State of Vienna, 1180
About this study
Official title: A Prospective Randomized, Double Blind, Placebo-controlled, Safety and Efficacy Study of Bosentan as add-on Therapy in Progressive Pulmonary Sarcoidosis
Progressive pulmonary sarcoidosis occurs in up to twenty percent of patients who require persistent treatment, but available treatment options have shown considerable long-term toxicity and uncertain or unproven efficacy. In these patients, pulmonary fibrosis and pulmonary hypertension are common complications which have major prognostic impact. Endothelin-1 (ET-1) has been demonstrated to play a key role in pulmonary fibrosis and pulmonary hypertension, and a potential role in pulmonary sarcoidosis. ET-1 is a potent vasoconstrictor and can promote fibrosis, cell proliferation, and remodeling, and is pro-inflammatory. Preliminary data have shown the therapeutic potential of the endothelin receptor antagonist (ERA) bosentan in sarcoidosis associated pulmonary hypertension.
In this light, the therapeutic potential of bosentan as an add-on treatment in progressive pulmonary sarcoidosis needs to be evaluated.
- Conditions
- Sarcoidosis, Pulmonary Hypertension
- Being tested
- bosentan, placebo
- Keywords
- endothelin receptor antagonist, bosentan
- Medical subject headings
- Sarcoidosis, Hypertension, Pulmonary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
bosentanDrug
62.5 mg tablets b.i.d. administered orally for 4 weeks followed by the maintenance dose of 125 mg b.i.d. (62.5 mg b.i.d. if body weight < 40 kg/90 lb)
placeboDrug
identical preparation as the study drug, but without the active substance, administered b.i.d.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | placebo b.i.d. Receives: Drug: placebo |
| Bosentan | Experimental | 62.5 mg/125 mg bosentan b.i.d. Receives: Drug: bosentan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment efficacy is assessed by a composite clinical score, including six parameters: Pulmonary function test (FVC and DLCO), Blood gas analysis (AaDO2), HRCT (Oberstein score), 6 minute walk test (6-MWD), Dyspnoea (ATS dyspnea scale)
Measured: 6 months
Secondary outcomes (2)
Assess safety and tolerability of bosentan in progressive pulmonary sarcoidosis
Measured: 6 months
To evaluate the efficacy of bosentan treatment in the subgroups of patents with and without sarcoidosis-associated pulmonary hypertension.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2009 (estimated)
- Study startApr 2009
- Primary completionMar 2010
- Study completionMar 2010
Why it stopped: Not enough patients with the specified criteria could not
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SarcoidosisStudies of the same condition, open and under way.
- 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Daniel Doberer
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Daniel Doberer, Medical University of Vienna
- Investigators
- Daniel Doberer, MD, MSc, Study director, Medical University of Vienna
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00926627
- Sponsor's protocol ID
- EudraCT - 2007-005117-18
- Acronym
- BOPSAC
- Status last verified
- Sep 2016
- First submitted
- 22 Jun 2009
- Last updated
- 15 Sep 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Randomized, Double Blind, Placebo-controlled, Safety and Efficacy Study of Bosentan as add-on Therapy in Progressive Pulmonary Sarcoidosis
- ClinicalTrials.gov ID
- NCT00926627
- Acronym / sponsor ID
- BOPSAC / EudraCT - 2007-005117-18
- Conditions
- Sarcoidosis, Pulmonary Hypertension
- Keywords
- endothelin receptor antagonist, bosentan
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Not enough patients with the specified criteria could not
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Daniel DobererUniversity, hospital or other
- Study start
- Apr 2009
- Primary completion
- Mar 2010 (actual)
- Study completion
- Mar 2010 (actual)
- First posted
- 23 Jun 2009 (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.
Progressive pulmonary sarcoidosis occurs in up to twenty percent of patients who require persistent treatment, but available treatment options have shown considerable long-term toxicity and uncertain or unproven efficacy. In these patients, pulmonary fibrosis and pulmonary hypertension are common complications which have major prognostic impact. Endothelin-1 (ET-1) has been demonstrated to play a key role in pulmonary fibrosis and pulmonary hypertension, and a potential role in pulmonary sarcoidosis. ET-1 is a potent vasoconstrictor and can promote fibrosis, cell proliferation, and remodeling, and is pro-inflammatory. Preliminary data have shown the therapeutic potential of the endothelin receptor antagonist (ERA) bosentan in sarcoidosis associated pulmonary hypertension.
In this light, the therapeutic potential of bosentan as an add-on treatment in progressive pulmonary sarcoidosis needs to be evaluated.
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 |
|---|---|---|
| Placebo | Placebo comparator | Drug: placeboplacebo b.i.d. |
| Bosentan | Experimental | Drug: bosentan62.5 mg/125 mg bosentan b.i.d. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Signed informed consent prior to any study-mandated procedure.
- Male and female patients aged > 18 and < 70 years.
- Histologically proven sarcoidosis diagnosed at least one year before screening.
- Diagnosis of sarcoidosis and with evidence of pulmonary parenchymal disease on chest X-ray or CT (radiological stage II, III) with or without pulmonary hypertension. Subjects with concurrent extrapulmonary sarcoidosis are encouraged to be enrolled.
- Progressive disease, defined as follows:
Key exclusion criteria (26 listed)
- Known hypersensitivity to any excipients of the drug formulation or to bosentan.
- Treatment with another investigational drug within 3 months prior to screening.
- Pulmonary sarcoidosis:
- Other cause of pulmonary disease:
- Anamnesis of beryllium or asbestos exposition
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 |
|---|---|---|
| bosentan62.5 mg tablets b.i.d. administered orally for 4 weeks followed by the maintenance dose of 125 mg b.i.d. (62.5 mg b.i.d. if body weight < 40 kg/90 lb) | Drug | Bosentan |
| placeboidentical preparation as the study drug, but without the active substance, administered b.i.d. | Drug | Placebo |
Primary outcome
Treatment efficacy is assessed by a composite clinical score, including six parameters: Pulmonary function test (FVC and DLCO), Blood gas analysis (AaDO2), HRCT (Oberstein score), 6 minute walk test (6-MWD), Dyspnoea (ATS dyspnea scale)
Time frame: 6 months
2 secondary outcomes
Assess safety and tolerability of bosentan in progressive pulmonary sarcoidosis
Time frame: 6 months
To evaluate the efficacy of bosentan treatment in the subgroups of patents with and without sarcoidosis-associated pulmonary hypertension.
Time frame: 6 months
Results and safety
The study was stopped early (reason given: Not enough patients with the specified criteria could not). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Sep 2016.
- Department of Clinical Pharmacology of the Medical University of ViennaLink 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.
Daniel Doberer, MD, MSc study director
Medical University of Vienna
2 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 bosentan in people with Sarcoidosis and Pulmonary Hypertension, run by Daniel Doberer at 2 sites in Austria.
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 14 inclusion and 26 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 15 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Austria. 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?
The study was stopped early (reason given: Not enough patients with the specified criteria could not). 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?
Treatment efficacy is assessed by a composite clinical score, including six parameters: Pulmonary function test (FVC and DLCO), Blood gas analysis (AaDO2), HRCT (Oberstein score), 6 minute walk test (6-MWD), Dyspnoea (ATS dyspnea scale) (time frame: 6 months). 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 15 Sep 2016.