BridgeMD
A study to compare the safety and how the body processes two different doses of an under-the-skin Alpha1-Proteinase inhibitor treatment in people with Alpha1-Antitrypsin Deficiency
EU CTIS ID: 2025-522792-29-00
What this study is testing
The primary pharmacokinetic (PK) objective is to determine if ( commercially confidential information) repeat doses over ( commercially confidential information) of subcutaneously administered alpha1-proteinase inhibitor subcutaneous (SC) (human) 15% (Alpha-1 15%) produce a steady-state area under the concentration versus time curve (AUC) of alpha1-PI, measured using an antigenic content assay, that is non-inferior to that of the 60 mg/kg/week and 120 mg/kg/week, respectively, administered intravenous (IV) doses of Liquid Alpha1 Proteinase Inhibitor (Human) (Liquid Alpha1-PI) over ( commercially confidential information) , in participants with Alpha1-Antitrypsin Deficiency (AATD).
- 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
- 1. At Screening Visit, is between 18 and 80 years of age, inclusive.
- 2. Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or “at-risk” alleles (patients with “at-risk” alleles listed in Appendix 2 must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a blood draw for genotyping (i.e., allelic discrimination) and phenotyping will be obtained at the Screening Visit.
- 3. Participants may be naïve to Alpha1-PI augmentation therapy or may be currently receiving Alpha1-PI augmentation therapy or received Alpha1-PI augmentation therapy in the past. If the total alpha1-PI serum (alpha-1 antitrypsin [AAT]) level has yet to be documented as in a treatment-naïve patient, a blood draw for total alpha1-PI serum level will be obtained at the Screening Visit. For participants currently receiving Alpha1-PI augmentation, a pre-Alpha1-PI augmentation AAT level must be documented in the participant’s medical history/records.
- 4. All participants must have a documented total alpha1-PI serum level <11 μM (80 mg/dL if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry) which is documented pre-Alpha1-PI augmentation for participants receiving AAT augmentation.
- 5. At the Screening Visit, have a post-bronchodilator Forced Expiratory Volume in 1 second (FEV1) ≥25% and <80% of predicted and FEV1/Forced Vital Capacity (FVC) <70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III and some individuals who are GOLD stage IV).
- 6. If the participant has received alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment at the Week 1 (Baseline) Visit and remain off any kind of alpha1-PI treatment, other than the IPs of this study, while participating in the study.
You likely can't join if
- 1. Have had a moderate or severe chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.
- 10. Have history of anaphylaxis or severe systemic response to any plasma-derived alpha1- PI preparation or other blood product(s).
- 11. Use systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 4 weeks prior to the Week 1 (Baseline) Visit (Note: inhaled steroids are not considered systemic steroids). It is recommended to maintain the same dose throughout the study.
- 12. Use systemic or aerosolized antibiotics for a COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.
- 13. Have known selective or severe Immunoglobulin A (IgA) deficiency based on prior medical records.
- 14. In the opinion of the Investigator, the participant may have compliance problems or any medical condition that may place them at safety risk with the protocol and the procedures of the protocol, or because of unstable health be unable to come to the study site for in-person clinic visits required by the protocol.
See the full eligibility criteria
Who can join
- 1. At Screening Visit, is between 18 and 80 years of age, inclusive.
- 2. Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or “at-risk” alleles (patients with “at-risk” alleles listed in Appendix 2 must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a blood draw for genotyping (i.e., allelic discrimination) and phenotyping will be obtained at the Screening Visit.
- 3. Participants may be naïve to Alpha1-PI augmentation therapy or may be currently receiving Alpha1-PI augmentation therapy or received Alpha1-PI augmentation therapy in the past. If the total alpha1-PI serum (alpha-1 antitrypsin [AAT]) level has yet to be documented as in a treatment-naïve patient, a blood draw for total alpha1-PI serum level will be obtained at the Screening Visit. For participants currently receiving Alpha1-PI augmentation, a pre-Alpha1-PI augmentation AAT level must be documented in the participant’s medical history/records.
- 4. All participants must have a documented total alpha1-PI serum level <11 μM (80 mg/dL if measured by radial immunodiffusion or 50 mg/dL if measured by nephelometry) which is documented pre-Alpha1-PI augmentation for participants receiving AAT augmentation.
- 5. At the Screening Visit, have a post-bronchodilator Forced Expiratory Volume in 1 second (FEV1) ≥25% and <80% of predicted and FEV1/Forced Vital Capacity (FVC) <70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III and some individuals who are GOLD stage IV).
- 6. If the participant has received alpha1-PI augmentation therapy of any kind, he/she must be willing to discontinue that treatment at the Week 1 (Baseline) Visit and remain off any kind of alpha1-PI treatment, other than the IPs of this study, while participating in the study.
- 7. Willing and able to provide written informed consent indicating that they understand the purpose of, and procedures required for the study and are willing to participate in it.
What rules you out
- 1. Have had a moderate or severe chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.
- 10. Have history of anaphylaxis or severe systemic response to any plasma-derived alpha1- PI preparation or other blood product(s).
- 11. Use systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e., 10 mg every 2 days) within the 4 weeks prior to the Week 1 (Baseline) Visit (Note: inhaled steroids are not considered systemic steroids). It is recommended to maintain the same dose throughout the study.
- 12. Use systemic or aerosolized antibiotics for a COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.
- 13. Have known selective or severe Immunoglobulin A (IgA) deficiency based on prior medical records.
- 14. In the opinion of the Investigator, the participant may have compliance problems or any medical condition that may place them at safety risk with the protocol and the procedures of the protocol, or because of unstable health be unable to come to the study site for in-person clinic visits required by the protocol.
- 2. Have history of lung or liver transplant or on transplantation waiting list.
- 3. Have any lung surgery during the past 1 year (excluding lung biopsy).
- 4. At screening, have elevated liver enzymes (aspartate aminotransferase [AST], alanine aminotransferase [ALT], and alkaline phosphatase [ALP]) ≥ 2.5 times the upper limit of normal (ULN).
- 5. Have severe concomitant disease (e.g., congestive heart failure, clinically significant pulmonary fibrosis, malignant disease [except for skin cancers other than melanoma], history of acute hypersensitivity pneumonitis reaction, or current chronic hypersensitivity pneumonitis).
- 6. 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. †Women of childbearing potential are defined as premenopausal and not surgically sterile, post tubal ligation, nor documented as infertile due to a concurrent medical condition. *True abstinence: When this is in line with the preferred and usual lifestyle of the participant. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, postovulation methods], declaration of abstinence for the duration of a study, and withdrawal are not acceptable methods of contraception.)
- 7. Have known previous infection with or clinical signs and symptoms consistent with current Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), or Human Immunodeficiency Virus (HIV) infection.
- 8. Have smoked during the past 6 months (this includes electronic/vapor cigarettes) or a positive urine cotinine test at the Screening Visit that is due to smoking.
- 9. Received IP in another study within 30 days prior to the Week 1 (Baseline) Visit or received any recombinant human AAT-Fc fusion protein (e.g., INBRX-101) or other extended half-life AAT products within 5 half-lives of the product relative to the Screening Visit date.
The study team makes the final eligibility decision.
Where it's taking place
- United States
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, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. 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.