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Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis (IMPALA-X)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03482752
Recruitment Status : Completed
First Posted : March 29, 2018
Last Update Posted : February 3, 2021
Information provided by (Responsible Party):
Savara Inc.

Tracking Information
First Submitted Date  ICMJE March 21, 2018
First Posted Date  ICMJE March 29, 2018
Last Update Posted Date February 3, 2021
Actual Study Start Date  ICMJE April 16, 2018
Actual Primary Completion Date January 14, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 28, 2018)
  • Number of adverse events [ Time Frame: 36 months ]
  • Number of serious adverse events [ Time Frame: 36 months ]
  • Number of adverse drug reactions [ Time Frame: 36 months ]
  • Number of adverse events leading to treatment discontinuation [ Time Frame: 36 months ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 28, 2018)
  • Alveolar-arterial oxygen gradient [ Time Frame: 36 months ]
    Difference in oxygen tension between lungs and blood
  • 6-minute walk distance [ Time Frame: 36 months ]
  • St Georges Respiratory Questionnaire total score [ Time Frame: 36 months ]
    Respiratory-specific questionnaire measuring impact on overall health, daily life, and perceived well-being. Scores range from 0 to 100, with higher scores indicating more limitations.
  • Frequency of whole lung lavages during the trial [ Time Frame: 36 months ]
  • Diffusion capacity of the lung for carbon monoxide [ Time Frame: 36 months ]
  • Forced expiratory volume in one second [ Time Frame: 36 months ]
  • Forced vital capacity [ Time Frame: 36 months ]
  • Arterial oxygen tension [ Time Frame: 36 months ]
  • Pulmonary alveolar proteinosis Disease Severity Score [ Time Frame: 36 months ]
    Disease-specific score graded based on symptoms of PAP and oxygenation of blood. Scores range from 1 to 5, where 5 indicates most severe disease.
  • Need for oxygen supplement therapy [ Time Frame: 36 months ]
  • Number of subjects not requiring treatment for pulmonary alveolar proteinosis [ Time Frame: 36 months ]
  • Time off treatment for pulmonary alveolar proteinosis [ Time Frame: 36 months ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis
Official Title  ICMJE An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
Brief Summary

Clinical trial for subjects with autoimmune pulmonary alveolar proteinosis (aPAP) who have completed the IMPALA trial (NCT02702180).

At the Baseline visit, eligible subjects 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.

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

During the trial, whole lung lavage will be applied as rescue therapy.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description:
Open Label, Non-controlled
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Autoimmune Pulmonary Alveolar Proteinosis
Intervention  ICMJE Drug: Molgramostim
300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.
Other Name: Recombinant human GM-CSF
Study Arms  ICMJE Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: February 2, 2021)
Original Estimated Enrollment  ICMJE
 (submitted: March 28, 2018)
Actual Study Completion Date  ICMJE January 14, 2021
Actual Primary Completion Date January 14, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Denmark,   France,   Germany,   Greece,   Israel,   Italy,   Netherlands,   Russian Federation,   Turkey,   United Kingdom
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT03482752
Other Study ID Numbers  ICMJE SAV006-03
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Current Responsible Party Savara Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Savara Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Francesco Bonella, Prof. Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany
PRS Account Savara Inc.
Verification Date February 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP