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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 19 November 2018
Main ID:  EUCTR2017-004078-32-GR
Date of registration: 10/05/2018
Prospective Registration: Yes
Primary sponsor: Savara ApS
Public title: Clinical trial in patients with the lung disease, autoimmune pulmonary alveolar proteinosis, to assess continued treatment with molgramostim by inhalation
Scientific title: AN OPEN-LABEL, NON-CONTROLLED, MULTICENTRE CLINICAL TRIAL OF INHALED MOLGRAMOSTIM IN AUTOIMMUNE PULMONARY ALVEOLAR PROTEINOSIS PATIENTS - IMPALA-EX
Date of first enrolment: 05/06/2018
Target sample size: 90
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004078-32
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
Number of treatment arms in the trial: 1
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Denmark France Germany Greece Israel Italy Netherlands Russian Federation
United Kingdom
Contacts
Name: Trial Coordinator   
Address:  Slotsmarken 17, 2.tv 2970 Hørsholm Denmark
Telephone: +4524885979
Email: mette.vinge@savarapharma.com
Affiliation:  Savara ApS
Name: Trial Coordinator   
Address:  Slotsmarken 17, 2.tv 2970 Hørsholm Denmark
Telephone: +4524885979
Email: mette.vinge@savarapharma.com
Affiliation:  Savara ApS
Key inclusion & exclusion criteria
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, 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.
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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Autoimmune Pulmonary Alveolar Proteinosis (aPAP)
MedDRA version: 20.0 Level: LLT Classification code 10037316 Term: Pulmonary alveolar proteinosis System Organ Class: 100000004855
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
Intervention(s)

Product Name: Molgramostim Nebuliser solution 300 mcg
Pharmaceutical Form: Nebuliser solution
INN or Proposed INN: MOLGRAMOSTIM
CAS Number: 99283-10-0
Concentration unit: µg/ml microgram(s)/millilitre
Concentration type: equal
Concentration number: 250-

Primary Outcome(s)
Main Objective: To investigate safety of long term use of inhaled molgramostim.
Primary end point(s): Number of AEs, SAEs, adverse drug reactions (ADRs), and AEs leading to treatment discontinuation.
Secondary Objective: • To investigate effects of long term use of inhaled molgramostim on oxygenation.
• To investigate effects of long term use of inhaled molgramostim on exercise capacity.
• To investigate effects of long term use of inhaled molgramostim on respiratory quality of life.
• To investigate frequency of need for WLL during long term use of inhaled molgramostim.
• To investigate effects of long term use of inhaled molgramostim on lung function.
• To investigate maintenance of effect after discontinuation of inhaled molgramostim.
Timepoint(s) of evaluation of this end point: End of trial
Secondary Outcome(s)
Secondary end point(s): • (A-a)DO2 during the trial
• 6MWD during the trial
• SGRQ total score during the trial
• Frequency of WLL during the trial
• DLCO (% predicted), FEV1 (% predicted), and FVC (% predicted) during the trial
• PaO2 and disease severity score (DSS) during the trial
• Need for oxygen supplement therapy during the trial
• 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
Secondary ID(s)
2017-004078-32-DK
SAV006-03
Source(s) of Monetary Support
Savara ApS
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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