A Randomised, Double-Blind, Placebo-Controlled Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary AlveoLAr Proteinosis Patients
- Conditions
- Auto-immune PAPPulmonary alveolar proteinosis1000381610024970
- Registration Number
- NL-OMON46238
- Lead Sponsor
- Savara ApS
- Brief Summary
Trial is onging in other countries
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 9
- aPAP diagnosed by CT, or by biopsy, or by Broncho Alveolar Lavage (BAL), and by increased GM-CSF autoantibodies in serum.;- Stable or progressive aPAP (i.e. absolute VC not improved by more than 5% and/or DLCO not improved by more than 10% - assessed from medical records) during a minimum period of two months prior to the Baseline visit. ;- PaO2 <75 mmHg/<10 kPa at rest, OR desaturation of >4 percentage points on the 6 Minute Walk Test (6MWT) ;- An (A-a)DO2 of minimum 25 mmHg/3.33 kPa ;- Female or male >=18 years of age;- Females who have been post-menopausal for > 1 year or females of childbearing potential after a confirmed menstrual period using a highly efficient method of contraception (i.e. a method with < 1% failure rate such as combined hormonal contraception, progesterone-only hormonal contraception, intrauterine device, intrauterine hormone- releasing system, bilateral tubal occlusion, vasectomised partner, sexual abstinence ), during and until 30 days after last dose of double-blind trial treatment. Females of childbearing potential must have a negative serum pregnancy test at Screening (Visit 1) and a negative urine pregnancy test at dosing at Baseline (Visit 2) and must not be lactating;- Males agreeing to use condoms during and until 30 days after last dose of double-blind medication, or males having a female partner who is using adequate contraception as described above;- Willing and able to provide signed informed consent;- Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other trial procedures specified in the protocol as judged by the investigator
- Diagnosis of hereditary or secondary pulmonary alveolar proteinosis (PAP);- WLL within two months of Baseline ;- Treatment with GM-CSF within three months of Baseline;- Treatment with rituximab within six months of Baseline;- Treatment with plasmapheresis within three months of Baseline;- Treatment with any investigational medicinal product within four weeks of Screening;- Concomitant use of sputum modifying drugs such as carbocystein or ambroxol;- History of allergic reactions to GM-CSF;- Connective tissue disease, inflammatory bowel disease or other autoimmune disorder requiring treatment associated with significant immunosuppression, e.g. more than 10 mg/kg systemic prednisolone.;- Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product;- History of, or present, myeloproliferative disease or leukaemia;- Known active infection (viral, bacterial, fungal or mycobacterial);- Apparent pre-existing concurrent pulmonary fibrosis;- Any other serious medical condition which in the opinion of the investigator would make the subject unsuitable for the trial
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method