Safety Extension Trial of Inhaled Molgramostim in Autoimmune Pulmonary Alveolar Proteinosis

Phase 3TerminatedNCT03482752

Savara Inc.60 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Autoimmune Pulmonary Alveolar Proteinosis

Interventions

MolgramostimDRUG

300 µg inhaled molgramostim in cycles of once daily administration for 7 days, then 7 days off treatment.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (13)

Dept. Of Respiratory Diseases & Allergy

Aarhus, Denmark

CHU Rennes Hospital Pontchaillou, Service de Pneumologie

Rennes, France

Outcomes

Primary Outcomes

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.

Time frame: 139 weeks

Number of Serious TEAEs

Serious TEAEs were defined as any untoward medicinal occurrence or effect that at any dose: * Results in death * Is life-threatening * Requires hospitalisation or prolongation of existing hospitalisation * Results in persistent or significant disability or incapacity * Is a congenital abnormality or birth defect * May jeopardise the participant or may require medical intervention to prevent one or more of the outcomes listed above (Important Medical Events).

Time frame: 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.

Time frame: 139 weeks

Number of TEAEs Leading to Treatment Discontinuation

Time frame: 139 weeks

Data from ClinicalTrials.gov

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