Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"
- Conditions
- Alpha 1-Antitrypsin Deficiency
- Interventions
- Drug: Placebo
- Registration Number
- NCT04204252
- Lead Sponsor
- Kamada, Ltd.
- Brief Summary
The goal of this clinical trial is to learn if AAT for inhalation, at a dose of 80 mg/day can slow the progression of lung disease in people who have lung disease caused by severe genetic deficiency in Alpha 1 Antitrypsin (AATD).
The main question it aims to answer is:
• Can daily treatment with Kamada AAT for inhalation at a dose of 80 mg/day prevent or slow lung function worsening ? Lung function will be measured by spirometry.
Other questions it aims to answer are:
* Can daily treatment with Kamada AAT for inhalation at a dose of 80 mg/day prevent or slow lung density loss ? Lung density will be measured by a CT scan.
* Can daily treatment with Kamada AAT for inhalation at a dose of 80 mg/day prevent or slow lung disease from worsening ? Lung disease will be measured using spirometry, lung volume, gas diffusion, six minute walk test, quality of life questionaires and biomarkers.
* What medical problems do participants have when taking AAT for inhalation 80 mg/day daily ? Researchers will compare AAT for inhalation to a placebo (a look-alike substance that contains no drug) to see if AAT for inhalation works to treat AAT-deficiency related lung disease. Study participants will receive either AAT for inhalation or placebo for the first two years of the study. During the third and fourth years of the study all participants will receive AAT for inhalation regardless of which drug they received during the first two years.
Participants will:
* Inhale the study drug every day
* Clean and disinfect the nebulizer every day
* Document daily symptoms and study drug use in an electronic diary
* Visit the clinic for tests and assessments. There are 11 clinic visits during the first two years of the study and 5-6 clinic visits during the third and fourth year, combined. After treatment ends, participants will visit the clinic 3 times in half a year.
- Detailed Description
Individuals with a genetic deficiency of alpha-1-antitrypsin (AATD) are at a significantly increased risk (80-100%) of developing lung disease (emphysema or COPD). This study is designed to administer a solution of AAT by nebulizer so that patients can inhale the drug instead of requiring infusions as in current treatment. A significant advantage of inhalation is that the AAT is directly transferred to the lungs, which is the site most in need of the protein. Previous results show that in addition to the added convenience, treatment restores normal levels of active AAT to the lung. To date, more than 220 participants have completed clinical trials with AAT for inhalation for several diseases.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 220
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Daily inhalation of a solution of NaCl in phosphate buffer solution with 0.01% TWEEN-80 Inhaled AAT Alpha 1-Antitrypsin Daily inhalation of 80 mg/day "Kamada-AAT for Inhalation" for 104 weeks
- Primary Outcome Measures
Name Time Method FEV1 post bronchodilator 104 weeks Change from baseline in post bronchodilator FEV1 at 104 weeks
- Secondary Outcome Measures
Name Time Method CT densitometry whole-lung 15th percentile lung density (PD15) at total lung capacity (TLC). 104 weeks Change from baseline over 104 weeks of treatment in CT densitometry whole-lung 15th percentile lung density (PD15) at total lung capacity (TLC).
FEV1 % of predicted 104 weeks Change from baseline over 104 weeks of treatment in FEV1 % of predicted
FEV1/FVC % 104 weeks Change from baseline over 104 weeks of treatment in FEV1/FVC %
Exacerbations 104 weeks Annual rate of exacerbations by severity and duration
6 minute walk test 104 weeks Change from Baseline in the distance walked in six minutes
Trial Locations
- Locations (9)
Royal Infirmary of Edinburgh
🇬🇧Edinburgh, United Kingdom
University Hospital Southampton NHS Foundation Trust
🇬🇧Southampton, United Kingdom
University Hospital (UZ) Leuven
🇧🇪Leuven, Belgium
Tays Central Hospital
🇫🇮Tampere, Finland
Beaumont Hospital
🇮🇪Dublin, Ireland
Leiden University Medical Center (LUMC)
🇳🇱Leiden, ZA, Netherlands
Canisius Wilhelmina Hospital (CWZ)
🇳🇱Nijmegen, Netherlands
Skåne University Hospital
🇸🇪Malmö, Sweden
University Hospitals Birmingham NHS Foundation Trust
🇬🇧Birmingham, United Kingdom