PULMATRIX 601-0018
Research type
Research Study
Full title
A Randomized, Double-Blind, Multicenter, Placebo-Controlled Study to Evaluate the Effect of Dose and Duration of Treatment of Itraconazole Administered as a Dry Powder for Inhalation (PUR1900) on Safety, Tolerability, and Potential Outcomes in Adult Patients with Asthma and Allergic Bronchopulmonary Aspergillosis
IRAS ID
1006803
Contact name
Margaret Wasilewski
Contact email
Sponsor organisation
PULMATRIX Inc.
Eudract number
2022-002289-33
Clinicaltrials.gov Identifier
Research summary
Summary of Research:
This study will investigate how safe, effective, and tolerable PUR1900 is compared to placebo in the treatment of ABPA (Allergic bronchopulmonary aspergillosis). ABPA is an allergic reaction to a fungus called Aspergillus fumigatus (Af), found naturally in the soil. In some people with asthma, the immune system overreacts to Af, resulting in damage to the airways, lungs and development of ABPA. To date, there are no approved therapies to treat ABPA. PUR1900 is an inhaled form of an oral antifungal medicine called itraconazole, used under the trade name Sporanox®. This study is sponsored by Pulmatrix Inc. PUR1900 is an investigational medication as it has not yet been approved by government agencies in any country to treat any condition. Its safety and how well it works when administered by inhalation are still being studied. About 30 participants aged 18 - 80 years, with asthma and ABPA, are expected to take part in this randomized, double-blind, multicenter, placebo-controlled study. The study will consist of a 28-day screening period, a 16-week treatment period, and an 8-week observation period. Following screening, patients will be randomly assigned into 1 of 3 treatment groups: PUR1900 20 mg (12 patients), PUR1900 40 mg (12 patients) and Matching Placebo (6 participants) with investigators, subjects, and the Sponsor not aware of treatment assignment. The study will monitor Treatment Emergent Adverse Events, frequency of exacerbations, Changes in sputum A. fumigatus antifungal susceptibility, among other end-points.Research Summary:
1.OBJECTIVE Allergic Bronchopulmonary Aspergillosis (ABPA) is an allergic reaction to a fungus called Aspergillus fumigatus,which is found naturally in soil.In certain people,especially people who have asthma,the immune system overreacts to the fungus.This may damage the airways and the lungs.The study drug is called PUR1900,an inhaled version of an oral(taken by mouth)antifungal medicine called itraconazole or Sporanox®.This study was a Phase 2 study;a Phase 2 study is a clinical trial that tests a new treatment in a small number of patients for a specific disease or condition to see whether the treatment works to help patients and whether it is safe for them.The study was planned to enroll 30 patients with both asthma and ABPA to compare two dose levels of PUR1900 to placebo(“dummy” medicine which does not contain any active ingredients).The main objectives of the Phase 2 study were•To test the safety of PUR1900 compared to placebo•To check the effect of PUR1900 on breathing and asthma symptoms.Participants were put into 3 groups by chance (randomized) to reduce differences between the groups.Putting participants into groups by chance helps to reduce differences between the groups,which makes the comparison between the groups fairer.This trial was also “double-blinded.”This means that neither patients nor doctors knew who was given which treatment/drug.This was done to make sure that the trial results were not influenced in any way2.WHEN AND WHERE?The trial started in February 2023,and it ended in February 2024.The study was stopped after 8 participants were enrolled because it took much longer than planned to identify patients eligible to participate in the study.Where did the clinical trial take place?The study included 8 patients in the following countries:6 patients in the US,1 patient in France and 1 patient in Australia.3.WHO PARTICIPATED?Patients in the clinical trial had to meet the following criteria:•18 years or older•Diagnosed with asthma and ABPA•On a stable asthma medication regimen•Had at least one exacerbation(worsening breathing problems)in the last 12 months.How many men and women were in the clinical trial?5 Women(62.5%)and 3Men(37.5%)How old were the patients?18 to 33 years-2 patients (25%);34 to 64 years-3 patients(37.5%) and 65 to 82 years-3 patients(37.5%)4.STUDY TREATMENT? Study participants were put into 3 groups by chance to reduce differences between the groups:PUR1900 20 mg-3 patients (37.5%); PUR1900 40 mg-3 patients(37.5%);Placebo-2 patients(25%)5.SIDE EFFECTS:Side effects are unwanted medical events (such as headache) that happen during the study and are reported because the trial doctor believes the side effects were related to the treatments in the trial.Not all patients in this trial had side effects.The below summary shows the number of patients who experienced side effects.In this study,side effects were unwanted events thought to be related to the study drug-Related to PUR1900 20 mg:1 patient(33.3%)who had side effects.No patients who had seriousside effects and no patients who withdrew because of a side effect;-Related to PUR1900 40 mg:1 patient(33.3%) who had side effects.No patients who had seriousside effects and no patients who withdrew because of a side effect;-Related to placebo:1 patient (50%) who had side effects.No patients who had serious*side effects.1 patient (50%) who withdrew because of a side effect.*Defined as reactions that are life threatening or require the individual to have to go to hospital.•One patient in each treatment group experienced a side effect.oSide effects in the PUR1900 20 mg group included:itchy rash in 1 patient(33.3%)oSide effects in the PUR1900 40 mg group included:wheezing(33.3%)and shortness of breath(33.3%)in 1 patientoSide effects in the placebo group included:increased cough(50%)in 1 patient•No patients experienced serious side effects.•One patient in the placebo group experienced a side effect resulting in stopping treatment6.STUDY RESULTS?The study was stopped after 8 patients were enrolled because it took much longer than planned to identify patients eligible to participate in the study. Due to the small number of subjects in the dosing groups, no conclusions can be drawn from the study.These results are only for patients with asthma and ABPA.7HOW HAS THIS STUDY HELPED?This trial helped researchers in their search for new treatment options for patients with ABPA.Unfortunately,this trial was not completed and no conclusions can be drawn8.PLANED STUDIES?No future studies with PUR1900 in ABPA are planned.Exclusive rights to the development or commercialization of PUR1900 in all markets other than the USA have been granted to Cipla9.FURTHER INFO:Protocol No:601-0018,EudraCT2022-002289-33,NCT No:05667662.Sponsor name? Pulmatrix,Inc.Contact details? Contact us at (888)3554440 or https://www.pulmatrix.com/contact.htmlMore information about this study is available at: https://classic.clinicaltrials.gov/ct2/show/NCT05667662REC name
London - Fulham Research Ethics Committee
REC reference
22/LO/0840
Date of REC Opinion
16 May 2023
REC opinion
Further Information Favourable Opinion