This is a Phase 2b study, or clinical trial, for people living with PPF. The goal of the MIST Study is to learn more about the investigational study medicine: an inhaled version of pirfenidone called AP01.
Progressive Pulmonary Fibrosis (PPF) is a rare lung disease that causes lung scarring. This makes it hard to breathe.
52 weeks long (or more for participants who choose to continue on an extension study)
The study medicine or a placebo will be inhaled by nebulizer twice a day
Medical exams and tests at most appointments
Study exams, tests and treatments are provided at no charge to participants
Frequent appointments with study doctors
Many study sites across the U.S.
Some study participants will be randomly selected to take a placebo. A placebo is a substance that looks like the study medicine but has no active ingredients. Study participants and staff will not know who is taking the placebo. This is done to prevent bias and to understand if the investigative medicine is working.
Joining a study is an important, personal decision. There are many reasons to consider joining:
All study participants who complete this study will have the chance to join an ongoing extension study to take the investigative medicine (not the placebo) at no charge.
A Randomized, Double-Blind, Placebo-Controlled, Phase 2b Study Evaluating the Safety and Efficacy of Pirfenidone Solution for Inhalation (AP01) in People with Progressive Pulmonary Fibrosis (PPF)
Randomized: to ensure there is no bias or unfairness in the study results, study participants are assigned by chance to receive the investigative medicine or placebo
Double-Blind: to prevent bias, no one in the study (patients) and no one running the study (doctors or nurses) will know if the study participant is taking the investigative medicine or placebo until after the study is finished
Placebo: A placebo is made to look like the investigative medicine, but it is not meant to affect health
Placebo-controlled: a type of study that has one group of study participants who take the investigative medicine with active ingredients, and another group that gets the placebo, which has no active ingredients
Phase 2b: a study that is researching the safety, effectiveness, and dosage of the investigative medicine
Efficacy: researchers want to know if an investigative medicine works the way it was intended
Safety: this study will be monitored by the study doctor, study sponsor, and independent experts to monitor the safety of the investigative medicine
Pirfenidone Solution for Inhalation (AP01): the investigative medicine in this study
Talk to your doctor to find out if the MIST Study is an option for you.
Visit ClinicalTrials.gov and search for NCT06329401
Avalyn Pharma is a biopharma company focused on developing medicines for rare lung diseases.
AP01 is an investigational drug device combination and is not approved by any health authority