Phase 4
Completed N=140
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
emphysema · Alpha 1-proteinase Inhibitor Deficiency
Source: ClinicalTrials.gov NCT00670007 ↗
Enrolled (actual)
140
Serious AEs
36.4%
Results posted
Jul 2016
Primary outcomePrimary: Rate of Change of Adjusted Lung Density — -1.632; -1.352; -1.627; -1.256 g/L per year — p== 0.752
◆ Published Evidence
Highly cited
505citations · ~46 / year
Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial.
Summary
This study is a continuation of the placebo-controlled study CE1226\_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v). administration in subjects with emphysema due to alpha1-proteinase inhibitor deficiency. The long-term verification of a disease-modifying benefit of Zemaira® on the progression of emphysema will be assessed by volume-adjusted lung density, measured yearly by computed tomography (CT).
Linked Publications (3)
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Intravenous augmentation treatment and lung density in severe α1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial.
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Long-term efficacy and safety of α1 proteinase inhibitor treatment for emphysema caused by severe α1 antitrypsin deficiency: an open-label extension trial (RAPID-OLE).
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Safety of biweekly α<sub>1</sub>-antitrypsin treatment in the RAPID programme.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Rate of Change of Adjusted Lung Density |
-1.632; -1.352; -1.627; -1.256; -1.658; -1.482 | = 0.752 |
| SECONDARY Absolute Change in Adjusted Lung Density |
-3.031; -2.502; -2.971; -2.485; -2.934; -2.953 | 0.526 |
| SECONDARY Percent Change in Adjusted Lung Density |
-6.741; -7.035; -6.825; -6.674; -6.494; -8.281 | 0.883 |
| SECONDARY Change in Subject-reported Symptoms |
1.185; 1.499; 6.601; 0.728; 0.55; 2.831 | — |
| SECONDARY Percent Change in Lung Function as Measured by Forced Expiratory Volume in 1 Second (FEV1) |
-8.610; -8.666 | — |
| SECONDARY Percent Change in Lung Function as Measured by Ratio of FEV1/FVC (Forced Vital Capacity) |
0.560; -5.441 | — |
| SECONDARY Percent Change in Lung Function as Measured by Percent Predicted FEV1 |
-7.165; -6.958 | — |
| SECONDARY Number of Subjects With Pulmonary Exacerbations |
13; 16; 63; 48; 60; 46 | — |
| SECONDARY Annual Rate in Subject Years of Pulmonary Exacerbations |
1.71; 1.39 | — |
| SECONDARY Time to First Pulmonary Exacerbation |
0.405; 0.602 | — |
| SECONDARY Percentage of Subjects With Treatment Emergent Adverse Events |
100; 96.9; 19.7; 15.6; 50.0; 51.6 | — |
Eligibility Criteria
Inclusion Criteria
- Subjects who have completed the 2-year treatment and observation period in the Phase 3/4 Zemaira® CE1226\_4001 study (NCT00261833) and are willing to sign informed consent
- Males, and non-pregnant, non-lactating females, whose screening pregnancy test is negative and who are using contraceptive methods deemed reliable by the investigator
Exclusion Criteria
- Individuals residing in the US
- Current evidence of alcohol abuse or abuse of drugs such as barbiturates, benzodiazepines, amphetamines, cocaine, opioids, and cannabinoids
- History of allergy, anaphylactic reaction, or severe systemic response to human plasma derived products, or known mannitol hypersensitivity, or history of prior adverse reaction to mannitol
- Current tobacco smoker (smoking must be discontinued for at least 6 months prior to study participation)
- Conditions or behaviors that interfere with attending scheduled study visits in the opinion of the investigator
- History of non-compliance
- Administration of any other experimental new drug or participation in an investigation of a marketed product
- Inability to perform necessary study procedures
Data sourced from ClinicalTrials.gov (NCT00670007) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.