MOL-PAP-002 Version 1.0
Research type
Research Study
Full title
A clinical trial where patients with the lung disease autoimmune Pulmonary Alveolar Proteinosis will be given the drug molgramostim by inhalation.
IRAS ID
190025
Contact name
Cliff Morgan
Contact email
Sponsor organisation
SAVARA
Eudract number
2015-003878-33
Duration of Study in the UK
2 years, 5 months, 27 days
Research summary
The IMPALA study is primarily designed to compare the efficacy of inhaled molgramostim on the Alveolar-arterial oxygen difference with placebo after 24-weeks treatment. Furthermore, this study also focuses on the efficacy of inhaled molgramostim on the requirement for, and time to Whole Lung Lavage (WLL), and Vital Capacity (VC)-'The maximum amount of air a person can expel from the lungs after a maximum inhalation' (% predicted) with placebo after 24-weeks treatment.
Subjects considered to be eligible comprises of Autoimmune Pulmonary Alveolar Proteinosis (aPAP), and by increased autoantibodies against Granulocyte Macrophage Colony Stimulating Factor (auto anti GM-CSF) in serum. Subjects have to provide written informed consent, passed screening assessments and comply with inclusion/exclusion criteria.
The trail design includes a Screening Visit prior to the Baseline Visit to determine the eligibility, and at the Baseline Visit, eligible subjects not requiring ″WILL’’ will be directly randomised to the treatment.
The trail will include two phases; a Double-blind period consisting of up to eight trail visits or nine trail visits, and a Follow-up period consisting of up to five trail visits.
During the Double-blind treatment period, ‘’WILL’’ will be applied as rescue therapy, and the criterion for performing ‘’WLL’’ is an increase in alveolar-arterial oxygen difference ((A-a)DO2) by more than 10 mm Hg/1.33 kPa compared to baseline.
During the Follow-up period, subjects experiencing an increase in (A-a)DO2 by more than 10 mm Hg/1.33 kPa compared to baseline (using the same oxygen amount as at baseline, if applicable) may be treated with molgramostim, ‘’WLL’’ or other treatments according to the investigator’s discretion.
After completion of both the Double-blind and Follow-up trial periods, subjects who require further treatment will be offered treatment with molgramostim in a Compassionate Use Programme, where national regulations so allow.
The primary endpoint for this study is defined as the absolute change from baseline of (A-a)DO2 after 24-weeks treatment.REC name
London - South East Research Ethics Committee
REC reference
15/LO/1971
Date of REC Opinion
28 Jan 2016
REC opinion
Further Information Favourable Opinion