Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03482752




Registration number
NCT03482752
Ethics application status
Date submitted
21/03/2018
Date registered
29/03/2018
Date last updated
3/07/2024

Titles & IDs
Public title
Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
Scientific title
An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
Secondary ID [1] 0 0
2017-004078-32
Secondary ID [2] 0 0
SAV006-03
Universal Trial Number (UTN)
Trial acronym
IMPALA-X
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autoimmune Pulmonary Alveolar Proteinosis 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Molgramostim

Experimental: Molgramostim nebulizer solution (300 µg) - Open-label treatment with molgramostim nebulizer solution (300 µg) administered intermittently (repetitive cycles of 7 days of treatment followed by 7 days off-treatment).


Treatment: Drugs: Molgramostim
300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Treatment-emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
139 weeks
Primary outcome [2] 0 0
Number of Serious TEAEs
Timepoint [2] 0 0
139 weeks
Primary outcome [3] 0 0
Number of Treatment-emergent Adverse Drug Reactions (ADRs)
Timepoint [3] 0 0
139 weeks
Primary outcome [4] 0 0
Number of TEAEs Leading to Treatment Discontinuation
Timepoint [4] 0 0
139 weeks

Eligibility
Key inclusion criteria
* Completer of the IMPALA trial.
* Females who have been post menopausal for >1 year, or females of child-bearing potential who are not pregnant or lactating and are using acceptable contraceptive methods.
* Males agreeing to use using acceptable contraceptive methods.
* Willing and able to provide signed informed consent.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Treatment with GM-CSF products other than molgramostim nebuliser solution within three months of Baseline.
* Treatment with any investigational medicinal product other than inhaled molgramostim within four weeks of Baseline.
* 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/day 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.
* 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
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
16/04/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
14/01/2021
Sample size
Target
Accrual to date
Final
60
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Denmark
State/province [1] 0 0
Århus
Country [2] 0 0
France
State/province [2] 0 0
Rennes
Country [3] 0 0
Germany
State/province [3] 0 0
Essen
Country [4] 0 0
Germany
State/province [4] 0 0
Gauting
Country [5] 0 0
Germany
State/province [5] 0 0
Heidelberg
Country [6] 0 0
Germany
State/province [6] 0 0
Lübeck
Country [7] 0 0
Greece
State/province [7] 0 0
Athens
Country [8] 0 0
Israel
State/province [8] 0 0
Tel Aviv
Country [9] 0 0
Italy
State/province [9] 0 0
Pavia
Country [10] 0 0
Netherlands
State/province [10] 0 0
Nieuwegein
Country [11] 0 0
Russian Federation
State/province [11] 0 0
Saint Petersburg
Country [12] 0 0
Turkey
State/province [12] 0 0
Istanbul
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Savara Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
SAV006-03 is an open-label extension study for participants who had completed the IMPALA study.

At the baseline visit, eligible participants may continue or re-start treatment with 300 µg inhaled molgramostim (recombinant human Granulocyte-Macrophage Colony Stimulating Factor; GM-CSF) administered intermittently in cycles of seven days molgramostim, administered once daily, and seven days off treatment.

Participants will be treated with inhaled molgramostim for up to 36 months.

During the trial, whole lung lavage will be applied as rescue therapy.
Trial website
https://clinicaltrials.gov/study/NCT03482752
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Francesco Bonella, Prof.
Address 0 0
Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03482752