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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004078-32 | EudraCT Number |
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SAV006-03 was initiated before the evaluation of the IMPALA study (NCT02702180) results. Considering these results and authority advice there would not be adequate efficacy and safety data from SAV006-03 and the study was terminated.
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Molgramostim nebulizer solution (300 μg) | Experimental | Open-label treatment with molgramostim nebulizer solution (300 μg) administered intermittently (repetitive cycles of 7 days of treatment followed by 7 days off-treatment). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Molgramostim | Drug | 300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Treatment-emergent Adverse Events (TEAEs) | The primary objective of this trial was safety assessed by adverse event (AE) reporting. Definitions and reporting procedures for AEs were done according to current regulatory standards. AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns. Treatment-emergent was defined as events occurring on study drug and up to 7 days after last dose of study drug. | 139 weeks |
| Number of Serious TEAEs | Serious TEAEs were defined as any untoward medicinal occurrence or effect that at any dose:
| 139 weeks |
| Number of Treatment-emergent Adverse Drug Reactions (ADRs) | All AEs were assessed by the investigator for causality (unlikely, possible, probable, not applicable) according to current regulatory standards. AEs which had a 'possible' or 'probable' causality were classified as ADRs. | 139 weeks |
| Number of TEAEs Leading to Treatment Discontinuation | 139 weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Francesco Bonella, Prof. | Interstitial and Rare Lung Disease Unit, Ruhrlandklinik University Hospital, Essen, Germany | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Dept. Of Respiratory Diseases & Allergy | Aarhus | Denmark | ||||
| CHU Rennes Hospital Pontchaillou, Service de Pneumologie |
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SAV006-03 was an open-label extension trial for participants who had completed the IMPALA study. Among the 30 clinical sites enrolling participants in IMPALA, 13 sites participated in SAV006-03. First participant was enrolled on 16 April 2018 and last participant completed the study on 14 January 2021.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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). Molgramostim: 300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Aug 24, 2018 | Dec 22, 2022 |
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Open label, non-controlled.
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| Rennes |
| France |
| 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 |
| Attikon University Hospital 2nd Pulmonary Department Athens Medical School National and Kapodistrian University of Athens | Athens | Greece |
| Rabin Medical Center Institute of Pulomonary Medicine | Tel Aviv | Israel |
| S.C. Pneumologia Fondazione IRCCS Policlinico San Matteo | Pavia | Italy |
| St. Antonius Hospital | Nieuwegein | Netherlands |
| Pavlov first Saint Petersburg State Medical Univerisity | Saint Petersburg | Russia |
| Yedikule Pulmonary Diseases and Pulmonary Surgery Training and Research Hospital | Istanbul | 34020 | Turkey (Türkiye) |
| Dept. Of Intensive Care Unit Royal Brompton Hospital London | London | United Kingdom |
| Safety Analysis Set | The safety analysis set included all enrolled participants who were exposed to molgramostim nebulizer solution in SAV006-03. |
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| COMPLETED | The participants who remained in the study when the study was terminated were classified as early terminators. |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | 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). Molgramostim: 300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants | No |
| ||||||||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants | No |
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| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Treatment-emergent Adverse Events (TEAEs) | The primary objective of this trial was safety assessed by adverse event (AE) reporting. Definitions and reporting procedures for AEs were done according to current regulatory standards. AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns. Treatment-emergent was defined as events occurring on study drug and up to 7 days after last dose of study drug. | Safety analysis set. | Posted | Number | events | 139 weeks |
|
|
| ||||||||||||||||||||||||||
| Primary | Number of Serious TEAEs | Serious TEAEs were defined as any untoward medicinal occurrence or effect that at any dose:
| Safety analysis set. | Posted | Number | events | 139 weeks |
|
| |||||||||||||||||||||||||||
| Primary | Number of Treatment-emergent Adverse Drug Reactions (ADRs) | All AEs were assessed by the investigator for causality (unlikely, possible, probable, not applicable) according to current regulatory standards. AEs which had a 'possible' or 'probable' causality were classified as ADRs. | Safety analysis set. | Posted | Number | events | 139 weeks |
|
| |||||||||||||||||||||||||||
| Primary | Number of TEAEs Leading to Treatment Discontinuation | Safety analysis set. | Posted | Number | events | 139 weeks |
|
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Adverse events (AEs) were collected from the baseline visit up to a follow-up telephone call, which occurred 2 weeks after study completion (maximum duration 139 weeks).
AEs were collected by the investigator by a non-leading question and by reporting events directly observed or spontaneously volunteered by participants. Participants were also encouraged to contact the clinic in between visits if they experienced AEs or had any concerns.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 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). Molgramostim: 300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment. | 1 | 59 | 8 | 59 | 38 | 59 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Alveolar proteinosis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
| |
| HIV infection CDC Group IV subgroup A | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Laryngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Cellulitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
| |
| Alveolar proteinosis | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 21.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 21.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 21.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Respiratory tract infection | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Bronchitis | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 21.0 | Systematic Assessment |
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No hypothesis/statistical testing was planned for any of the endpoints of this study but only descriptive statistics were to be used. Efficacy endpoints were secondary endpoints. Due to early study termination, only limited efficacy data were available for evaluation and in general, no firm conclusion based on efficacy data are applicable. No deaths occurred during the study but 1 participant died due to COVID-19 24 days after last dose, i.e. outside the definition of 'treatment-emergent'.
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Raymond D Pratt, Chief Medical Officer | Savara Inc | +1 512 784 8757 | ray.pratt@savarapharma.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 21, 2021 | Dec 21, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| C567049 | Pulmonary Alveolar Proteinosis, Acquired |
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| ID | Term |
|---|---|
| C082430 | molgramostim |
| D016178 | Granulocyte-Macrophage Colony-Stimulating Factor |
| ID | Term |
|---|---|
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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| Turkey |
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| Denmark |
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| United Kingdom |
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| Italy |
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| Israel |
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| France |
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| Germany |
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| Russia |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
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