Status and phase
Conditions
Treatments
Study type
Funder types
Identifiers
About
160 subjects with autoimmune pulmonary alveolar proteinosis (aPAP) will be randomized to receive once daily treatment with inhaled molgramostim or placebo for 48 weeks. Subjects completing the 48 week placebo-controlled period will receive open-label treatment with once daily inhaled molgramostim for 96 weeks.
Full description
This is an interventional, randomized, double-blind, 2-arm, parallel groups, placebo-controlled, multi-center, phase 3 trial in adult subjects who are diagnosed with aPAP.
An aPAP diagnosis should be confirmed by an anti-GM-CSF auto-antibody test result, and history of PAP based on either high resolution computed tomography, lung biopsy, or bronchoalveolar lavage cytology, should be available.
The trial consists of a 6-week screening period, a 48-week randomized, double-blind treatment period, a 96-week open-label treatment period, and a conditional 4-week safety follow-up period. The maximum treatment duration will be 145 weeks and the maximum trial duration will be 156 weeks. During the trial, whole lung lavage will be allowed as rescue treatment in case of worsening of aPAP.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Subject must be ≥18 years of age, at the time of signing the informed consent (≥20 in Japan).
A serum anti-GM-CSF autoantibody test result confirming autoimmune PAP.
History of PAP, based on examination of a lung biopsy, bronchoalveolar lavage (BAL) cytology, or a high-resolution computed tomogram (HRCT) of the chest.
DLCO 70% predicted or lower at the screening and baseline visits.
Change in % predicted DLCO of <15% points during the screening period.
Demonstrated functional impairment in the treadmill exercise test (defined as a peak MET ≤8).
Willing and able to come off supplemental oxygen use prior to and during the treadmill exercise test, the DLCO assessment, and the arterial blood gas sampling.
Resting SpO2 >85% during 15 minutes without use of supplemental oxygen at the screening visits.
Male or female
Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other trial procedures specified in the protocol as judged by the Investigator.
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
160 participants in 2 patient groups, including a placebo group
Loading...
Central trial contact
Brian T Maurer; Raymond D Pratt, MD
Data sourced from clinicaltrials.gov
Clinical trials
Research sites
Resources
Legal