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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-522792-29-00 | EU Trial (CTIS) Number |
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This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses of the same medicine given through a vein.
Adults with Alpha-1 Antitrypsin Deficiency will take part. Participants will be randomly assigned to one of the treatment groups, and both the study doctors and participants will know which treatment is being given.
The main goals of the study are to understand how the body processes the medicine (pharmacokinetics) and to assess how safe and well tolerated the different weekly doses are.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment Arm 1 (60 mg/kg/week of Liquid Alpha1-PI followed by 90 mg/kg/week of Alpha-1 15%) | Experimental | 8 weeks of IV treatment with 60 mg/kg/week Liquid Alpha1-PI followed by 8 weeks of SC treatment with 90 mg/kg/week Alpha-1 15% |
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| Treatment Arm 2 (120 mg/kg/week of Liquid Alpha1-PI followed by 180 mg/kg/week of Alpha-1 15%) | Experimental | 8 weeks of IV treatment with 120 mg/kg/week Liquid Alpha1-PI followed by 8 weeks of SC treatment with 180 mg/kg/week Alpha-1 15% |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Alpha-1 15% | Drug | Alpha1-Proteinase Inhibitor Subcutaneous (Human), 15% |
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| Measure | Description | Time Frame |
|---|---|---|
| Steady-state AUC of alpha1-PI over the weekly dosing interval (from 0 to 7 days) (AUC0-7 days) in the IV Treatment Period 1 and in the SC Treatment Period 2 for both dose levels | Week 1 to Week 16 |
| Measure | Description | Time Frame |
|---|---|---|
| The average value of the steady-state trough alpha1-PI measurements obtained at Weeks 6, 7, 8, and 9 (Period 1) | Weeks 6, 7, 8 and 9 | |
| The average value of the steady-state trough alpha1-PI measurements obtained at Weeks 14, 15, 16, and 17 (Period 2) | Weeks 14, 15, 16 and 17 |
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Inclusion Criteria:
Exclusion Criteria:
Have had a moderate or severe chronic obstructive pulmonary disease (COPD) exacerbation during the 4 weeks before the Week 1 (Baseline) Visit.
Have history of lung or liver transplant or on transplantation waiting list.
Have any lung surgery during the past 1 year (excluding lung biopsy).
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).
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).
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, post-ovulation methods], declaration of abstinence for the duration of a study, and withdrawal are not acceptable methods of contraception.)
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.
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.
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.
Have history of anaphylaxis or severe systemic response to any plasma-derived alpha1- PI preparation or other blood product(s).
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.
Use systemic or aerosolized antibiotics for a COPD exacerbation within the 4 weeks prior to the Week 1 (Baseline) Visit.
Have known selective or severe Immunoglobulin A (IgA) deficiency based on prior medical records.
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.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Beatriz Garcia Castro | Contact | +(34) 670923669 | beatriz.garcia@grifols.com | |
| Elsa Mondou | Contact | +1 919 316 2079 | elsa.mondou@grifols.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama at Birmingham | Not yet recruiting | Birmingham | Alabama | 35233 | United States |
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| Liquid Alpha1-PI | Other | Liquid Alpha1-Proteinase Inhibitor (Human) for Intravenous infusion |
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| Pulmonary Associates | Not yet recruiting | Phoenix | Arizona | 85032 | United States |
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| Mayo Clinic | Not yet recruiting | Scottsdale | Arizona | 85259 | United States |
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| UCLA | Not yet recruiting | Los Angeles | California | 90095 | United States |
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| University of Florida | Not yet recruiting | Gainesville | Florida | 32610 | United States |
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| University of Miami | Not yet recruiting | Miami | Florida | 33136 | United States |
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| Mayo Clinic | Not yet recruiting | Rochester | Minnesota | 55905 | United States |
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| Cleveland Clinic | Not yet recruiting | Cleveland | Ohio | 44195 | United States |
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| Medical University of South Carolina | Not yet recruiting | Charleston | South Carolina | 29425 | United States |
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| UT Southwestern Medical Center | Not yet recruiting | Dallas | Texas | 75390 | United States |
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| Vijle Sygeheus | Recruiting | Vejle | Denmark |
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| Beaumont Hospital | Not yet recruiting | Dublin | D09YD60 | Ireland |
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| Leiden University Medical Center | Not yet recruiting | Leiden | Netherlands |
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| Instytut Gruzlicy I Charób Pluc | Not yet recruiting | Warsaw | Poland |
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| Unidade Local de Saúde do Alto Ave | Recruiting | Guimarães | Portugal |
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| Unidade Local de Saúde Loures-Odivelas | Recruiting | Loures | Portugal |
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| Hospital Clinico San Carlos | Not yet recruiting | Madrid | Spain |
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| Hospital Álvaro Cunqueiro | Recruiting | Vigo | Spain |
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| Skane University Hospital | Recruiting | Malmö | Sweden |
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| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| D011656 | Pulmonary Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D029424 | Pulmonary Disease, Chronic Obstructive |
| D008173 | Lung Diseases, Obstructive |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D013352 | Subcutaneous Emphysema |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
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| ID | Term |
|---|---|
| C088172 | 15-keto-17-phenyl-18,19,20-trinorprostaglandin F2 alpha-1-isopropyl ester |
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