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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00670007




Registration number
NCT00670007
Ethics application status
Date submitted
29/04/2008
Date registered
1/05/2008
Date last updated
15/08/2016

Titles & IDs
Public title
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
Scientific 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
Secondary ID [1] 0 0
1466
Secondary ID [2] 0 0
CE1226_3001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Emphysema 0 0
Alpha 1-proteinase Inhibitor Deficiency 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Respiratory 0 0 0 0
Other respiratory disorders / diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Alpha1- proteinase inhibitor [human]

Experimental: Zemaira® -


Treatment: Other: Alpha1- proteinase inhibitor [human]
Lyophilized preparation of 60 mg/kg body weight intravenously once per week
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Rate of Change of Adjusted Lung Density
Timepoint [1] 0 0
Up to 2 years
Secondary outcome [1] 0 0
Absolute Change in Adjusted Lung Density
Timepoint [1] 0 0
From baseline to 2 years
Secondary outcome [2] 0 0
Percent Change in Adjusted Lung Density
Timepoint [2] 0 0
From baseline to 2 years
Secondary outcome [3] 0 0
Change in Subject-reported Symptoms
Timepoint [3] 0 0
From baseline to 2 years
Secondary outcome [4] 0 0
Percent Change in Lung Function as Measured by Forced Expiratory Volume in 1 Second (FEV1)
Timepoint [4] 0 0
From baseline up to 2 years
Secondary outcome [5] 0 0
Percent Change in Lung Function as Measured by Ratio of FEV1/FVC (Forced Vital Capacity)
Timepoint [5] 0 0
From baseline up to 2 years
Secondary outcome [6] 0 0
Percent Change in Lung Function as Measured by Percent Predicted FEV1
Timepoint [6] 0 0
From baseline up to 2 years
Secondary outcome [7] 0 0
Number of Subjects With Pulmonary Exacerbations
Timepoint [7] 0 0
Up to 2 years
Secondary outcome [8] 0 0
Annual Rate in Subject Years of Pulmonary Exacerbations
Timepoint [8] 0 0
Up to 2 years
Secondary outcome [9] 0 0
Time to First Pulmonary Exacerbation
Timepoint [9] 0 0
Up to 2 years
Secondary outcome [10] 0 0
Percentage of Subjects With Treatment Emergent Adverse Events
Timepoint [10] 0 0
From baseline up to 2.5 years

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 4
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/04/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/09/2014
Sample size
Target
Accrual to date
Final
140
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
Study Site - Adelaide
Recruitment hospital [2] 0 0
Study Site - Fitzroy
Recruitment hospital [3] 0 0
Study Site - Darlinghurst
Recruitment hospital [4] 0 0
Study Site - Nedlands
Recruitment hospital [5] 0 0
Study Site - New Lambton
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3065 - Fitzroy
Recruitment postcode(s) [3] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [4] 0 0
6009 - Nedlands
Recruitment postcode(s) [5] 0 0
2305 - New Lambton
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
British Columbia
Country [2] 0 0
Canada
State/province [2] 0 0
Nova Scotia
Country [3] 0 0
Canada
State/province [3] 0 0
Ontario
Country [4] 0 0
Czech Republic
State/province [4] 0 0
Praha 4 - Krc
Country [5] 0 0
Denmark
State/province [5] 0 0
Arhus C
Country [6] 0 0
Denmark
State/province [6] 0 0
Hellerup
Country [7] 0 0
Estonia
State/province [7] 0 0
Tartu
Country [8] 0 0
Finland
State/province [8] 0 0
Oulu
Country [9] 0 0
Germany
State/province [9] 0 0
Essen
Country [10] 0 0
Germany
State/province [10] 0 0
Heidelberg
Country [11] 0 0
Germany
State/province [11] 0 0
Nürnberg
Country [12] 0 0
Ireland
State/province [12] 0 0
Dublin
Country [13] 0 0
Poland
State/province [13] 0 0
Krakow
Country [14] 0 0
Poland
State/province [14] 0 0
Warsaw
Country [15] 0 0
Romania
State/province [15] 0 0
Bucuresti
Country [16] 0 0
Sweden
State/province [16] 0 0
Malmo

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
CSL Behring
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/study/NCT00670007
Trial related presentations / publications
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.
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. Erratum In: Lancet Respir Med. 2017 Feb;5(2):e13. doi: 10.1016/S2213-2600(17)30004-8.
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.
Public notes

Contacts
Principal investigator
Name 0 0
Program Director, Clinical R&D
Address 0 0
CSL Behring
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT00670007