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Trial record 2 of 8 for:    savara

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

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ClinicalTrials.gov Identifier: NCT03482752
Recruitment Status : Enrolling by invitation
First Posted : March 29, 2018
Last Update Posted : September 19, 2019
Sponsor:
Information provided by (Responsible Party):
Savara Inc.

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.


Condition or disease Intervention/treatment Phase
Autoimmune Pulmonary Alveolar Proteinosis Drug: Molgramostim Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: Open Label, Non-controlled
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Non-controlled, Multicentre Clinical Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis Patients
Actual Study Start Date : April 16, 2018
Estimated Primary Completion Date : December 31, 2021
Estimated Study Completion Date : December 31, 2021

Resource links provided by the National Library of Medicine



Intervention Details:
  • 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


Primary Outcome Measures :
  1. Number of adverse events [ Time Frame: 36 months ]
  2. Number of serious adverse events [ Time Frame: 36 months ]
  3. Number of adverse drug reactions [ Time Frame: 36 months ]
  4. Number of adverse events leading to treatment discontinuation [ Time Frame: 36 months ]

Secondary Outcome Measures :
  1. Alveolar-arterial oxygen gradient [ Time Frame: 36 months ]
    Difference in oxygen tension between lungs and blood

  2. 6-minute walk distance [ Time Frame: 36 months ]
  3. 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.

  4. Frequency of whole lung lavages during the trial [ Time Frame: 36 months ]
  5. Diffusion capacity of the lung for carbon monoxide [ Time Frame: 36 months ]
  6. Forced expiratory volume in one second [ Time Frame: 36 months ]
  7. Forced vital capacity [ Time Frame: 36 months ]
  8. Arterial oxygen tension [ Time Frame: 36 months ]
  9. 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.

  10. Need for oxygen supplement therapy [ Time Frame: 36 months ]
  11. Number of subjects not requiring treatment for pulmonary alveolar proteinosis [ Time Frame: 36 months ]
  12. Time off treatment for pulmonary alveolar proteinosis [ Time Frame: 36 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03482752


Locations
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Denmark
Dept. Of Respiratory Diseases & Allergy
Århus, Denmark
France
CHU Rennes Hospital Pontchaillou, Service de Pneumologie
Rennes, France
Germany
Ruhrlandklinik Essen Westdeutsches Lungenzentrum am Universitätsklinikum Essen GmbH
Essen, Germany
Asklepios Fachkliniken München - Gauting Klinik für Pneumologie
Gauting, Germany
Thoraxklinik am Universitätsklinikum Heidelberg Abteilung für Pneumologie und Beatmungsmedizin
Heidelberg, Germany
Universitätsklinikum Schleswig-Holstein Zentralklinikum Lübeck Medizinische Klinik III - Pneumologie
Lübeck, Germany
Greece
Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens
Athens, Greece
Israel
Rabin Medical Center Institute of Pulomonary Medicine
Tel Aviv, Israel
Italy
S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Netherlands
St. Antonius Hospital
Nieuwegein, Netherlands
Russian Federation
Pavlov first Saint Petersburg State Medical Univerisity
Saint Petersburg, Russian Federation
Turkey
Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital
Istanbul, Turkey, 34020
United Kingdom
Dept. Of Intensive Care Unit Royal Brompton Hospital London
London, United Kingdom
Sponsors and Collaborators
Savara Inc.
Investigators
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Principal Investigator: Francesco Bonella, Prof. Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany

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Responsible Party: Savara Inc.
ClinicalTrials.gov Identifier: NCT03482752     History of Changes
Other Study ID Numbers: SAV006-03
First Posted: March 29, 2018    Key Record Dates
Last Update Posted: September 19, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pulmonary Alveolar Proteinosis
Autoimmune Diseases
Lung Diseases
Respiratory Tract Diseases
Immune System Diseases
Molgramostim
Antineoplastic Agents