Clinical trial in patients with the lung disease, autoimmune pulmonary alveolar proteinosis, to assess continued treatment with molgramostim by inhalatio

Phase 1

Conditions: Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
MedDRA version: 20.0Level: LLTClassification code 10037316Term: Pulmonary alveolar proteinosisSystem Organ Class: 100000004855
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: EUCTR2017-004078-32-IT

Lead Sponsor: Savara ApS

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 90

Inclusion Criteria

1.Completer of the IMPALA trial.
2.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 where inhibition of ovulation is the primary mode of action, intrauterine device, intrauterine hormone releasing system, bilateral tubal occlusion, vasectomised partner, sexual abstinence*), during and until 30 days after last dose of trial treatment. Females of childbearing potential must have a negative urine pregnancy test at Baseline, or at start of treatment if treatment is not dispensed at Baseline, and must not be lactating during and until 30 days after last dose of trial treatment*.
3.Males agreeing to use condoms during and until 30 days after last dose of trial treatment, or males having a female partner who is using adequate contraception as described above.
4.Willing and able to provide signed informed consent.
* Vasectomised partner is a highly effective birth control method provided that partner is the sole sexual partner of the female trial participant of child bearing potential and that the vasectomised partner has received medical assessment of the surgical success. Sexual abstinence is considered a highly effective method only if defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments. The reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical trial and the preferred and usual lifestyle of the subject
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 80
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

1. Treatment with GM-CSF products other than molgramostim nebuliser solution within three months of Baseline.
2. Treatment with any IMP other than inhaled molgramostim within four weeks of Baseline.
3. History of allergic reactions to GM-CSF.
4. Connective tissue disease, inflammatory bowel disease or other autoimmune disorder requiring treatment associated with significant immunosuppression, e.g. more than 10 mg/day systemic prednisolone.
5. Previous experience of severe and unexplained side-effects during aerosol delivery of any kind of medicinal product.
6. History of, or present, myeloproliferative disease or leukaemia.
7. Apparent pre-existing concurrent pulmonary fibrosis.
8. 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 clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To investigate safety of long term use of inhaled molgramostim.;Secondary Objective: • To investigate effects of long term use of inhaled molgramostim on oxygenation.<br>• To investigate effects of long term use of inhaled molgramostim on exercise capacity.<br>• To investigate effects of long term use of inhaled molgramostim on respiratory quality of life.<br>• To investigate frequency of need for WLL during long term use of inhaled molgramostim.<br>• To investigate effects of long term use of inhaled molgramostim on lung function.<br>• To investigate maintenance of effect after discontinuation of inhaled molgramostim.;Primary end point(s): Number of AEs, SAEs, adverse drug reactions (ADRs), and AEs leading to treatment discontinuation.;Timepoint(s) of evaluation of this end point: End of trial

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • (A-a)DO2 during the trial<br>• 6MWD during the trial<br>• SGRQ total score during the trial<br>• Frequency of WLL during the trial<br>• DLCO (% predicted), FEV1 (% predicted), and FVC (% predicted) during the trial<br>• PaO2 and disease severity score (DSS) during the trial<br>• Need for oxygen supplement therapy during the trial<br>• Number of subjects not requiring treatment for aPAP and time off treatment after discontinuation of inhaled molgramostim;Timepoint(s) of evaluation of this end point: End of trial

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