Clinical trial NCT00670007: Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
Official title: An Open-label, Non-controlled, Multicenter, Multinational Study to Evaluate the Efficacy and Safety of Zemaira® Administration in Chronic Augmentation and Maintenance Therapy in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency Who Completed Clinical Study CE1226_4001
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Emphysema
Alpha 1-proteinase Inhibitor Deficiency
Keywords: Alpha1-proteinase inhibitor deficiency
Chronic augmentation and maintenance therapy
Emphysema
Emphysema due to Alpha 1-proteinase inhibitor deficiency
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2008-05-01
Enrollment: 140
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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).


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

Primary outcomes:
measure: Rate of Change of Adjusted Lung Density
timeFrame: Up to 2 years
Secondary outcomes:
measure: Absolute Change in Adjusted Lung Density
timeFrame: From baseline to 2 years
measure: Percent Change in Adjusted Lung Density
timeFrame: From baseline to 2 years
measure: Change in Subject-reported Symptoms
timeFrame: From baseline to 2 years
measure: Percent Change in Lung Function as Measured by Forced Expiratory Volume in 1 Second (FEV1)
timeFrame: From baseline up to 2 years
measure: Percent Change in Lung Function as Measured by Ratio of FEV1/FVC (Forced Vital Capacity)
timeFrame: From baseline up to 2 years
measure: Percent Change in Lung Function as Measured by Percent Predicted FEV1
timeFrame: From baseline up to 2 years
measure: Number of Subjects With Pulmonary Exacerbations
timeFrame: Up to 2 years
measure: Annual Rate in Subject Years of Pulmonary Exacerbations
timeFrame: Up to 2 years
measure: Time to First Pulmonary Exacerbation
timeFrame: Up to 2 years
measure: Percentage of Subjects With Treatment Emergent Adverse Events
timeFrame: From baseline up to 2.5 years
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Study Site
state: South Australia
city: Fitzroy
country: Australia
facility: Study Site
state: Victoria
city: Darlinghurst
country: Australia
facility: Study Site
city: Nedlands
country: Australia
facility: Study Site
city: New Lambton
country: Australia
facility: Study Site
city: Vancouver
country: Canada
facility: Study Site
state: British Columbia
city: Halifax
country: Canada
facility: Study Site
state: Nova Scotia
city: Toronto
country: Canada
facility: Study Site
state: Ontario
city: Praha 4 - Krc
country: Czech Republic
facility: Study Site
city: Arhus C
country: Denmark
facility: Study Site
city: Hellerup
country: Denmark
facility: Study Site
city: Tartu
country: Estonia
facility: Study Site
city: Oulu
country: Finland
facility: Study Site
city: Essen
country: Germany
facility: Study Site
city: Heidelberg
country: Germany
facility: Study Site
city: Nürnberg
country: Germany
facility: Study Site
city: Dublin
country: Ireland
facility: Study Site
city: Krakow
country: Poland
facility: Study Site
city: Warsaw
country: Poland
facility: Study Site
city: Bucuresti
country: Romania
facility: Study Site
city: Malmo
country: Sweden
facility: Study Site
Study officials:
affiliation: CSL Behring
name: Program Director, Clinical R&D
role: STUDY_DIRECTOR
References:
citation: Greulich T, Chlumsky J, Wencker M, Vit O, Fries M, Chung T, Shebl A, Vogelmeier C, Chapman KR, McElvaney NG; RAPID Trial Group. Safety of biweekly alpha1-antitrypsin treatment in the RAPID programme. Eur Respir J. 2018 Nov 29;52(5):1800897. doi: 10.1183/13993003.00897-2018. Print 2018 Nov.
citation: McElvaney NG, Burdon J, Holmes M, Glanville A, Wark PA, Thompson PJ, Hernandez P, Chlumsky J, Teschler H, Ficker JH, Seersholm N, Altraja A, Makitaro R, Chorostowska-Wynimko J, Sanak M, Stoicescu PI, Piitulainen E, Vit O, Wencker M, Tortorici MA, Fries M, Edelman JM, Chapman KR; RAPID Extension Trial Group. Long-term efficacy and safety of alpha1 proteinase inhibitor treatment for emphysema caused by severe alpha1 antitrypsin deficiency: an open-label extension trial (RAPID-OLE). Lancet Respir Med. 2017 Jan;5(1):51-60. doi: 10.1016/S2213-2600(16)30430-1. Epub 2016 Dec 2.
citation: Chapman KR, Burdon JG, Piitulainen E, Sandhaus RA, Seersholm N, Stocks JM, Stoel BC, Huang L, Yao Z, Edelman JM, McElvaney NG; RAPID Trial Study Group. Intravenous augmentation treatment and lung density in severe alpha1 antitrypsin deficiency (RAPID): a randomised, double-blind, placebo-controlled trial. Lancet. 2015 Jul 25;386(9991):360-8. doi: 10.1016/S0140-6736(15)60860-1. Epub 2015 May 27.