BridgeMD
Tests treatment safety and results for Alpha 1-Antitrypsin Deficiency
Official title Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"
ClinicalTrials.gov ID: NCT04204252
What this study is testing
What is Alpha 1-Antitrypsin?
Alpha 1-Antitrypsin is an investigational medicine, given as an once-daily infusion into a vein, being studied as a potential treatment for alpha 1-antitrypsin deficiency.
Also referred to as Kamada alpha 1-antitrypsin for inhalation.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 ?
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z/Null), or Pi(Null/Null) genotypes.
- Serum AAT levels ≤ 11 µM at screening.
- Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1/SVC≤70%) at screening.
- 40% ≤ FEV1 ≤ 80% of predicted post-bronchodilator at screening.
- Patients who are either naïve or washed out of any AAT treatment for at least 8 weeks prior to randomization.
You likely can't join if
- Immunoglobulin A (IgA) absolute deficiency defined as serum IgA levels \< 0.05 g/L.
- History of life-threatening transfusion reaction(s), allergy, anaphylactic reaction, or systemic response to human plasma-derived products.
- Two or more moderate or any severe exacerbation(s) within the year prior to baseline.
- A moderate exacerbation within 6 weeks prior to baseline.
- Use of oral or parenteral glucocorticoids in doses above 10 mg of prednisone daily or equivalent generics (substance and dose).
- Clinically significant inter-current illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic...
See the full eligibility criteria
- Diagnosis of severe AAT deficiency, i.e. patients with either Pi(ZZ), Pi(Z/Null), or Pi(Null/Null) genotypes.
- Serum AAT levels ≤ 11 µM at screening.
- Lung disease with clinical evidence of airflow limitation (post bronchodilator FEV1/SVC≤70%) at screening.
- 40% ≤ FEV1 ≤ 80% of predicted post-bronchodilator at screening.
- Patients who are either naïve or washed out of any AAT treatment for at least 8 weeks prior to randomization.
- Age between 18 to 65 years inclusive at screening.
- Able to read and sign informed consent and willing to participate in the study.
- Males or non-pregnant, non-lactating females whose screening pregnancy test is negative, who are willing to use contraceptive methods for the duration of the study, or who are postmenopausal, or surgically sterilized.
- Study medication use for at least 20 out of the 28 days of run-in, as recorded in the study nebulization PARI Track data.
- Demonstrated ability to complete eDiary for at least 20 out of the first 28 days of run-in.
- Immunoglobulin A (IgA) absolute deficiency defined as serum IgA levels \< 0.05 g/L.
- History of life-threatening transfusion reaction(s), allergy, anaphylactic reaction, or systemic response to human plasma-derived products.
- Two or more moderate or any severe exacerbation(s) within the year prior to baseline.
- A moderate exacerbation within 6 weeks prior to baseline.
- Use of oral or parenteral glucocorticoids in doses above 10 mg of prednisone daily or equivalent generics (substance and dose).
- Clinically significant inter-current illnesses (except for respiratory or liver disease secondary to AAT deficiency), including: cardiac, hepatic, renal, endocrine, neurological, hematological, neoplastic...
- Hospitalization for any cause during the 6 weeks prior to screening.
- History of lung or liver transplant.
- On any thoracic or hepatic surgery waiting list.
- Any lung surgery within the past two years (including bronchoscopic lung volume reduction).
- Any smoking within the year prior to screening.
- Evidence of alcohol abuse or history of alcohol abuse, or use of illegal drugs and/or abuse of legally prescribed drugs in the last 5 years prior to screening.
- Acute or chronic hepatitis (hepatitis A, hepatitis B, hepatitis C), or positive human immunodeficiency virus (HIV) serology.
- Signs of significant abnormalities in serum hematology, serum chemistry, serum inflammatory / immunogenic markers and urinalysis per investigator judgment, taking into considerations the potential effects of the AAT...
- Signs of significant abnormalities in ECG per investigator judgment at screening.
- Presence of psychiatric/ mental disorder or any other medical disorder that might impair the patient's ability to give informed consent or to comply with the requirements of the study protocol. If, in the opinion of the...
- Participation in another clinical trial involving investigational medication or treatment treatment within 30 days and/or last dose 5 half-lives prior to screening visit.
- Inability to attend scheduled clinic visits and/or comply with study protocol.
- Any other factor that, in the opinion of the investigator, would prevent the patient from complying with the requirements of the protocol. Additional eligibility criteria apply for the open label extension
The study team makes the final eligibility decision.
Where it's taking place
- Leuven, Belgium
- Tampere, Finland
- Dublin, Ireland
- Leiden, ZA, Netherlands
- Nijmegen, Netherlands
- Malmö, Sweden
- Birmingham, United Kingdom
- Edinburgh, United Kingdom
- Southampton, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Leuven, Belgium; Tampere, Finland; Dublin, Ireland; Leiden, ZA, Netherlands; Nijmegen, Netherlands; Malmö, Sweden and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.