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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-522964-33-00 | EU Trial (CTIS) Number |
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This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density decline in alpha-1 antitrypsin (AAT) deficient participants over 3 years as compared with the marketed dose 60 milligrams per kilogram (mg/kg).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CE1226 low dose | Active Comparator | CE1226 at low dose. |
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| CE1226 medium dose | Experimental | CE1226 at medium dose. |
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| CE1226 high dose | Experimental | CE1226 at high dose. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CE1226 | Biological | CE1226 will be administered via intravenous (IV) infusion weekly over 3 years. |
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| Measure | Description | Time Frame |
|---|---|---|
| Annual rate of change in adjusted lung density | The annual rate of change (expressed as gram per liter per year [g/L/year]) in adjusted lung density (15th percentile lung density [PD15]) will be calculated on CT scan assessments of whole lung at total lung capacity (TLC). | From Baseline to Month 36 |
| Measure | Description | Time Frame |
|---|---|---|
| Annual rate of change in forced expiratory volume in 1 second percent predicted (FEV1%) | The annual rate of change in FEV1% will be assessed by spirometry. | From Baseline to Month 36 |
| Annual rate of change in diffusion capacity of carbon monoxide (DLco) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Trial Registration Coordinator | Contact | +1 610-878-4697 | clinicaltrials@cslbehring.com |
| Name | Affiliation | Role |
|---|---|---|
| Study Director | CSL Behring | Study Director |
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CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Requests for IPD will generally be considered once review by major regulatory authorities (i.e. FDA, EMA) is complete and the primary publication is available.
Proposed research should seek to answer a previously unanswered important medical or scientific question.
Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.
If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
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This is a double blind, parallel group assignment study.
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Sponsor will also be blinded to treatment allocation.
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The annual rate of change in DLco will be assessed by gas transfer using site machinery. |
| From Baseline to Month 36 |
| Number of severe pulmonary exacerbations | Pulmonary excerbations will be assessed by the investigator using Rodriguez-Roisin criteria, where an exacerbation is defined as: a sustained worsening of the patient's condition, from a stable state and beyond normal day-to-day variations, necessitating a change in regular medication in a patient with underlying chronic obstructive pulmonary disease (COPD). A severe exacerbation is defined as an event requiring the introduction of corticosteroids and / or antibiotics which results in hospitalization or death. | From screening up to Month 36 |
| Duration of severe pulmonary exacerbations | Pulmonary excerbations will be assessed by the investigator using Rodriguez-Roisin criteria, where an exacerbation is defined as: a sustained worsening of the patient's condition, from a stable state and beyond normal day-to-day variations, necessitating a change in regular medication in a patient with underlying COPD. A severe exacerbation is defined as an event requiring the introduction of corticosteroids and / or antibiotics which results in hospitalization or death. | From screening up to Month 36 |
| Number of participants experiencing treatment emergent adverse events (TEAEs) | From screening up to Month 36 |
| Percentage of participants experiencing TEAEs | From screening up to Month 36 |
| ID | Term |
|---|---|
| D019896 | alpha 1-Antitrypsin Deficiency |
| D004646 | Emphysema |
| ID | Term |
|---|---|
| D008107 | Liver Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D013352 | Subcutaneous Emphysema |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D000515 | alpha 1-Antitrypsin |
| C000654178 | Respreeza |
| ID | Term |
|---|---|
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D015843 | Serpins |
| 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 |
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