BATMAN - Bronchiectasis alpha-1 augmentation trial
Research type
Research Study
Full title
A Proof of Concept Trial of Alpha-1 Antitrypsin Augmentation therapy in patients with Bronchiectasis
IRAS ID
1004039
Contact name
James Chalmers
Contact email
Sponsor organisation
University of Dundee and Tayside Health Board
Eudract number
2021-004250-29
Research summary
Research Summary
Patients with bronchiectasis frequently get chest infections which are difficult to treat and can cause coughing, sputum (phlegm) production, breathlessness and tiredness. These symptoms can be caused by or made worse by inflammation of the airways. Alpha1 antitrypsin is a naturally occurring protein in the body that reduces inflammation and is necessary for healthy lung function.
The purpose of this trial is to test whether an intravenous infusion (drip) containing Prolastin-C (Alpha-1 Antitrypsin Augmentation Therapy) can reduce lung inflammation in people with bronchiectasis by increasing their levels of alpha1 antitrypsin. We will also assess whether people with bronchiectasis can take this drug safely. This medication is currently used to treat people with alpha1 antitrypsin deficiency safely.
20 participants who have bronchiectasis will take part. The trial will be held in Dundee, Scotland.
Each participant will be given the Prolastin-C drip once per week for 4 weeks and will also be given a placebo (dummy) drip once per week for another 4 weeks. This cross-over trial means that all participants will receive the active medicine. We will assess participants' lung inflamation by looking at markers within blood and sputum samples. We will compare the lung inflammation of participants when they have received the Prolastin-C drip for 4 weeks with when they have received the placebo drip for 4 weeks.Summary of Results
Bronchiectasis is a chronic lung condition where the airway tubes are wide and mucus cannot be effectively cleared from the lungs. This causes inflammation. Chemicals from the immune system called proteases damage the lung and make the disease worse over time. In a condition called alpha-1 antitrypsin deficiency, which is caused by a faulty gene, large amounts of proteases cause extensive lung damage. This can be prevented by infusions of the bodies natural "antidote" to proteases called alpha-1 antitrypsin. This is an approved treatment in many countries for alpha-1 antitrypsin.
The aim of the Batman trial was to find out if, in people without the genetic problem, increasing the amount of alpha-1 antitrypsin in the body through regular injections, would block the proteases in the lungs and make patients feel better.
20 patients were randomized and had 1 month with the treatment and 1 month without the treatment. Patients were blinded, meaning they and the investigators did not know during which 1 month period they got the treatment or the placebo.
We found that alpha-1 antitrypsin treatment did not reduce protease levels (neutrophil elastase) in the sputum tests after 1 month. The drug had the desired effect because the levels of neutrophil elastase were reduced in the blood but this did not translate into benefits in the lung. We did not see any changes in lung function or symptoms. We conclude that the amount of medication given via injections into the blood may not be high enough to improve things in the lungs. Future research could investigate giving the treatment directly into the lungs or using other methods to reduce the levels of these harmful proteases.REC name
Wales REC 5
REC reference
21/WA/0293
Date of REC Opinion
13 Oct 2021
REC opinion
Further Information Favourable Opinion