BridgeMD
A Controlled Study with an active treatment and a Placebo to Assess the Efficacy and Safety of Weekly Intravenous Product in Subjects with Pulmonary Emphysema due to Alpha-1 Antitrypsin Deficiency
EU CTIS ID: 2023-510030-83-00
What this study is testing
The primary objective of this study is to demonstrate, using whole lung CT densitometry (PD15), a slower progression of lung tissue loss in subjects with AATD on Alpha-1 MP treatment (either 60 mg/kg/week or 120 mg/kg/week) as compared to placebo.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- At screening is between 18 and 70 years of age, inclusive.
- Have a documented total alpha1-PI serum level < 11 μM. If the total alpha1-PI serum level has yet to be documented, a blood draw for total alpha1-PI serum level will be performed at the Screening (Week -3) Visit
- Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or “at-risk” alleles. If the genotype has yet to be documented, genotyping and phenotyping will be performed at the Screening (Week -3) Visit.
- At the Screening (Week -3) Visit, have a post-bronchodilator FEV1 ≥ 30% and < 80% of predicted and FEV1/FVC < 70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III).
- Have a DLCO ≤ 60% of predicted (corrected for hemoglobin [HgB]) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator’s judgment.
- Have a body mass index (BMI) < 40 kg/m2.
You likely can't join if
- Has received alpha1-PI augmentation therapy for more than 1 month within the 6 months prior to the Screening (Week -3) Visit.
- Severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
- Malignant disease (including malignant melanoma; however, other forms of skin cancer are allowed) within five years of the Screening (Week -3) Visit.
- Females who are pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence*) throughout the study. * True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.)
- Has a metal object that might interfere with chest CT quality and quantification. Metal objects include, but are not limited to, cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile, metal weapon fragments, or metal shoulder prosthesis.
- Currently infected with Hepatitis A virus (HAV) or Parvovirus B19 (B19V) or has laboratory results indicative of an acute or chronic infection with Hepatitis B virus (HBV), Hepatitis C virus (HCV), or Human Immunodeficiency Virus (HIV).
See the full eligibility criteria
Who can join
- At screening is between 18 and 70 years of age, inclusive.
- Have a documented total alpha1-PI serum level < 11 μM. If the total alpha1-PI serum level has yet to be documented, a blood draw for total alpha1-PI serum level will be performed at the Screening (Week -3) Visit
- Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or “at-risk” alleles. If the genotype has yet to be documented, genotyping and phenotyping will be performed at the Screening (Week -3) Visit.
- At the Screening (Week -3) Visit, have a post-bronchodilator FEV1 ≥ 30% and < 80% of predicted and FEV1/FVC < 70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III).
- Have a DLCO ≤ 60% of predicted (corrected for hemoglobin [HgB]) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the past 2 years per the Investigator’s judgment.
- Have a body mass index (BMI) < 40 kg/m2.
- Willing and able to provide informed consent.
What rules you out
- Has received alpha1-PI augmentation therapy for more than 1 month within the 6 months prior to the Screening (Week -3) Visit.
- Severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
- Malignant disease (including malignant melanoma; however, other forms of skin cancer are allowed) within five years of the Screening (Week -3) Visit.
- Females who are pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence*) throughout the study. * True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.)
- Has a metal object that might interfere with chest CT quality and quantification. Metal objects include, but are not limited to, cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile, metal weapon fragments, or metal shoulder prosthesis.
- Currently infected with Hepatitis A virus (HAV) or Parvovirus B19 (B19V) or has laboratory results indicative of an acute or chronic infection with Hepatitis B virus (HBV), Hepatitis C virus (HCV), or Human Immunodeficiency Virus (HIV).
- Smoking, which includes electronic/vapor cigarettes, during the past 12 months or a positive urine cotinine test at screening that is due to smoking (Note: subjects who have a positive urine cotinine test and are also receiving nicotine replacement therapy (e.g., nicotine patches or chewing gum), or using snuff or snus (smokeless tobacco) may be eligible to participate in the study upon completion of an exhaled carbon monoxide [eCO] breath test; subjects with an eCO ≥ 10 ppm will be ineligible for study participation).
- History of chronic alcoholism or illicit drug abuse (addiction) in the 12 months prior to the Screening (Week -3) Visit.
- Participation in another investigational product study within one month or five half-lives prior to the Screening (Week -3) Visit.
- History of anaphylaxis or severe systemic response to any plasma-derived alpha1-PI preparation or other blood product(s).
- Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 5 weeks prior to the Screening (Week -3) Visit (inhaled steroids are not considered systemic steroids) or during the Screening Phase. It is recommended to maintain the same dose throughout the study.
- Has received alpha1-PI augmentation therapy within 1 month of the Screening (Week -3) Visit.
- Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks prior to the Screening (Week -3) Visit or during the Screening Phase.
- Known selective or severe Immunoglobulin A (IgA) deficiency.
- Mentally challenged, adult subject who cannot give independent informed consent.
- In the opinion of the Investigator the subject may have compliance problems with the protocol and the procedures of the protocol.
- Any medical condition which the Investigator feels might confound the results of the study or pose an additional risk to the subject during study participation.
- Has had a COPD exacerbation within the 5 weeks prior to the Screening (Week -3) Visit or during the Screening Phase. Investigator discretion should be used to determine if a subject is appropriate for study participation if the subject has had a COPD exacerbation which occurred more than 5 weeks prior to the Screening (Week -3) Visit.
- Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g., claustrophobic) undergo a CT scan.
- History of lung or liver transplant.
- Any lung surgery during the past 2 years (excluding lung biopsy).
- On the waiting list for lung surgery, including lung transplant.
- On the waiting list for liver transplant.
- Elevated liver enzymes (aspartate transaminase [AST], alanine aminotransferase [ALT], and alkaline phosphatase) equal to or greater than 2.5 times the upper limit of normal (ULN).
The study team makes the final eligibility decision.
Where it's taking place
- Moldova, Republic of
- United Kingdom
- United States
- Brazil
- Argentina
- New Zealand
- Canada
- Russian Federation
- Australia
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, 18-64 years, 65+ years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Moldova, Republic of; United Kingdom; United States; Brazil; Argentina; New Zealand 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 EU CTIS; the study team decides eligibility.