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Safety and Efficacy Study of Bosentan in Progressive Pulmonary Sarcoidosis (BOPSAC)

D

Daniel Doberer

Status and phase

Terminated
Phase 2

Conditions

Sarcoidosis
Pulmonary Hypertension

Treatments

Drug: bosentan
Drug: placebo

Study type

Interventional

Funder types

Other

Identifiers

NCT00926627
EudraCT - 2007-005117-18

Details and patient eligibility

About

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.

Enrollment

32 patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • 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:

      • 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.

Exclusion criteria

  • 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)
  • 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 design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

32 participants in 2 patient groups, including a placebo group

Placebo
Placebo Comparator group
Description:
placebo b.i.d.
Treatment:
Drug: placebo
Bosentan
Experimental group
Description:
62.5 mg/125 mg bosentan b.i.d.
Treatment:
Drug: bosentan

Trial contacts and locations

2

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Data sourced from clinicaltrials.gov

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