To study the efficacy of danazol in patients with pulmonary fibrosis, and to investigate what factors are associated with effective treatment.
ID
Source
Brief title
Condition
- Lower respiratory tract disorders (excl obstruction and infection)
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The main study parameter is difference in yearly decline in lung function
between the period prior to danazol treatment and the first year after start of
danazol treatment.
Secondary outcome
Secondary outcome parameters are telomere length at baseline compared to that
during danazol treatment, haematological parameters at baseline compared to
during danazol treatment, survival, radiological qualification of pulmonary
fibrosis at baseline compared to during danazol treatment, quality of life at
baseline compared to that during danazol treatment, and adverse events during
danazol treatment.
Background summary
Idiopathic pulmonary fibrosis is a severe lung disease, for which there are
limited treatment options. Telomere shortening plays an important role in the
pathogenesis of idiopathic pulmonary fibrosis. Danazol is a synthetic hormone
with light androgenic effects that has been shown to lengthen telomeres in
patients with telomere disease. At present, idiopathic pulmonary fibrosis
patients at St. Antonius Hospital are offered off-label treatment with danazol
when they have progressive disease despite other treatments. However, the use
of danazol has not been studied extensively in pulmonary fibrosis patients.
Furthermore, it is not known if danazol would be an effective therapy for all
pulmonary fibrosis patients, or just for a subset of patients.
Study objective
To study the efficacy of danazol in patients with pulmonary fibrosis, and to
investigate what factors are associated with effective treatment.
Study design
Observational drug study
Study burden and risks
Patients will receive danazol in the course of regular clinical care.
Participating in this study is optional. Participation in the study will
involve the drawing of extra blood samples at five regular control visits (at
these visits the study requires the drawing of additional blood, next to blood
required for regular tests; no extra line is required). Furthermore, study
participants will have to fill in three questionnaires prior to starting
treatment with danazol, as well as three-monthly during the first twelve months
of danazol treatment. After the first year of danazol treatment, the study
requires 6-monthly drawing of extra blood samples (in addition to regular
samples; no extra line required), when patients are still taking danazol.
Koekoekslaan 1
Nieuwegein 3435CM
NL
Koekoekslaan 1
Nieuwegein 3435CM
NL
Listed location countries
Age
Inclusion criteria
- Consensus (working) diagnosis of IPF
- Age >= 18
- Starting treatment with danazol at St. Antonius Hospital (danazol treatment is offered to patients in the course of regular clinical care when: [1] treatment with both pirfenidone and nintedanib has been given or is not possible; and [2] there is proven clinical (decline in forced vital capacity (FVC) of >5% of predicted) and radiological (worsening of high-resolution computed tomography (HRCT) picture) progression of the disease)
Exclusion criteria
- Concomitant (severe) disease that precludes the ability to participate in the study protocol, or is likely to result in death within 30 days:
- Inability to give informed consent
- Not eligible for danazol treatment (patients will not be eligible to receive danazol treatment in the course of regular clinical care in case of: [1] severe cardiac, hepatic or renal disease; [2] use of statins metabolized by CYP3A4 (simvastatin, atorvastatin, lovastatin); [3] androgen-dependent tumor (e.g. prostate carcinoma); [4] increased thromboembolic risk, and no adequate prophylactic measures taken; [5] genital neoplasia; [6] porphyria; [7] pregnancy, or of childbearing potential and unwilling to take adequate contraceptive measures during danazol treatment)
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2017-004906-16-NL |
| CCMO | NL62034.100.17 |