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IND has been closed.
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This randomized trial is being conducted to show non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state. This will be conducted in individuals with alpha-1-antitrypsin deficiency and clinical evidence of emphysema.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| OctaAlpha1 | Experimental |
| |
| Glassia® | Active Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| OctaAlpha1 | Drug | For OctaAlpha1 the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions |
|
| Measure | Description | Time Frame |
|---|---|---|
| Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state | Non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state | 26 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (AUC) | Compare PK parameters following a single dose between the two treatment groups calculating area under the plasma concentration-time curve (AUC)-ratio: 90% confidence interval (CI) should lie within 80%-125%. | Time period including days 1 to 14 after first infusion in study |
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Inclusion Criteria:
Any subject who needs chronic IV augmentation and maintenance therapy with A1PI because of congenital alpha-1-proteinase inhibitor (A1PI) deficiency and clinically diagnosed emphysema
≥18 years of age
Individuals with A1PI serum concentration <11 µM at screening
Following bronchodilators:
Following bronchodilators: Initial forced expiratory volume/forced vital capacity (FEV1/FVC) ratio less than 70%
Non-smoking for at least 6 months before study treatment starts
Able to understand and provide written informed consent
Women of reproductive age: negative result of pregnancy test (human chorionic gonadotropin [HCG]-based assay) and agreement to use adequate contraception for the duration of the trial
Exclusion Criteria:
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| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
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| Glassia | Drug | For Glassia the standard weekly dose of 60mg/kg will be given for 24 consecutive infusions |
|
| Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (Cmax) | Compare PK parameters following a single dose between the two treatment groups calculating maximum plasma concentration (Cmax)-ratio: 90% CI should lie within 80%-125% | Time period including days 1 to 14 after first infusion in study |
| Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (tmax) | Compare PK parameters following a single dose between the two treatment groups calculating tmax (time to reach maximum serum concentration) | Time period including days 1 to 14 after first infusion in study |
| Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (t1/2) | Compare PK parameters following a single dose between the two treatment groups calculating t1/2 (apparent terminal half-life) | Time period including days 1 to 14 after first infusion in study |
| Compare PK parameters following a single dose between the two treatment groups following principles of bio-equivalence testing (λZ) | Compare PK parameters following a single dose between the two treatment groups calculating λZ (apparent terminal elimination rate constant determined by log-linear regression analysis) | Time period including days 1 to 14 after first infusion in study |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on occurrence of adverse events | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on blood pressure | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on pulse | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on body temperature | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on respiratory rate | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hematocrit via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter hemoglobin via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter white blood cells via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hematological parameter platelet count via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter alanine aminotransferase via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring hepatic parameter aspartate aminotransferase via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter creatinine via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN) | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on monitoring renal parameter Blood Urea Nitrogen (BUN) via lab test | 26 weeks |
| Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19) | Evaluate descriptively the safety and tolerability of OctaAlpha1 based on viral safety (HAV, HBV, HCV, HIV-1/2, HN, and parvovirus B19) | 26 weeks |
| Trough Levels of A1PI | Investigate descriptively the trough levels of A1PI and anti-NE capacity of OctaAlpha1 compared to Glassia® | 26 weeks |
| Pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests | Investigate the pharmacodynamics of OctaAlpha1 measuring Pulmonary function tests (done according to the American Thoracic Society/European Respiratory Society Taskforce Standardisation of Lung Function Testing guideline) | 26 weeks |
| Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total A1PI in the airway lining fluid of the lung. | Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total A1PI in the airway lining fluid of the lung. | 26 weeks |
| Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total LTB4 in the airway lining fluid of the lung. | Investigate the pharmacodynamics of OctaAlpha1 measuring Induced sputum test measuring the amount of functional and total LTB4 in the airway lining fluid of the lung. | 26 weeks |
| Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory | Investigate the pharmacodynamics of OctaAlpha1 measuring antibodies to A1PI using normal ranges of the central laboratory | 26 weeks |
| Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1 | Determine PK parameters of the A1PI serum concentration versus time curve following a single dose of OctaAlpha1 | 26 weeks |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D004646 | Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D010455 |
| Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D000209 | Acute-Phase Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000510 | Alpha-Globulins |
| D012712 | Serum Globulins |
| D005916 | Globulins |