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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02796937




Registration number
NCT02796937
Ethics application status
Date submitted
3/06/2016
Date registered
13/06/2016
Date last updated
7/03/2024

Titles & IDs
Public title
Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency
Scientific title
An Open-Label, Multicenter Study to Evaluate the Long-term Safety of Weekly Intravenous Alpha1-Proteinase Inhibitor (Human), Modified Process 60 mg/kg in Subjects With Pulmonary Emphysema Due to Alpha1-Antitrypsin Deficiency
Secondary ID [1] 0 0
GTi1201-OLE
Universal Trial Number (UTN)
Trial acronym
SPARTA-OLE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Emphysema in Alpha-1 Antitrypsin 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
Other interventions - Alpha-1 MP

Experimental: Alpha-1 MP - Alpha-1 MP 60 mg/kg/week for up to 104 weeks


Other interventions: Alpha-1 MP
Alpha-1 MP 60 mg/kg/week for up to 104 weeks
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse events (AEs)
Timepoint [1] 0 0
Week 1 through Week 108
Primary outcome [2] 0 0
Serious AEs (SAEs)
Timepoint [2] 0 0
Week 1 through Week 108
Primary outcome [3] 0 0
Discontinuations from the study due to AEs
Timepoint [3] 0 0
Week 1 through Week 108
Secondary outcome [1] 0 0
Change from baseline in whole lung PD15 (15th percentile point)
Timepoint [1] 0 0
Week 1 through Week 104
Secondary outcome [2] 0 0
Change from baseline in carbon monoxide diffusing capacity (DLco)
Timepoint [2] 0 0
Week 52 and Week 104
Secondary outcome [3] 0 0
Changes from baseline in forced expiratory volume in 1 second (FEV1)
Timepoint [3] 0 0
Week 52 and Week 104
Secondary outcome [4] 0 0
Change from baseline in Saint George's Respiratory Questionnaire
Timepoint [4] 0 0
Week 52 and Week 104
Secondary outcome [5] 0 0
Incidence and severity of Chronic Obstructive Pulmonary Disease (COPD)exacerbations
Timepoint [5] 0 0
Week 2 through Week 108
Secondary outcome [6] 0 0
Change from baseline in the EQ-5D-5L Questionnaire
Timepoint [6] 0 0
Week 52 and Week 104

Eligibility
Key inclusion criteria
- Has completed participation in Study GTi1201 (ie, completed Week 156 and Week
160/End-of-Study Visit) OR has experienced a decline in FEV1 at the annualized rate of
=134.4 mL/year at or after the Week 104 Visit in GTi1201.

- Is willing and able to provide informed consent
Minimum age
20 Years
Maximum age
72 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Is unable to physically or mentally undergo a CT scan (eg, unable to fit inside the CT
scanner, claustrophobic).

- Has severe concomitant disease including, but not limited to, congestive heart failure
and liver cirrhosis.

- Has primary and/or secondary (metastatic disease) pulmonary malignancy or other
current malignancy with <1 year predicted overall survival.

- Has a metal object (newly received since starting GTi1201) that might interfere with
chest CT quality and quantification. Metal objects include, but are not limited to,
cardiac pacemaker, defibrillator, metal prosthetic heart valve, metal projectile,
metal weapon fragments, or metal shoulder prosthesis.

- Is a female who is pregnant, breastfeeding or, if of child-bearing potential,
unwilling to practice a highly effective method of contraception (oral, injectable, or
implanted hormonal methods of contraception; placement of an intrauterine device or
intrauterine system; condom or occlusive cap with spermicidal
foam/gel/film/cream/suppository; male sterilization; or abstinence) throughout the
study.

- Has clinical signs and symptoms of viral infection requiring virus safety testing at
the Week 160/End-of-Study Visit or Early Discontinuation Visit in Study GTi1201, and
the virus safety test results are indicative of acute or chronic infection with
hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), human
immunodeficiency virus (HIV), or parvovirus B19 (B19V). Note: If the virus safety test
results are indicative of acute or chronic infection with HAV, HBV, HCV, HIV, or B19V,
the subject will be considered a screen failure and must be withdrawn from the study.

- Has current evidence of smoking, which includes electronic/vapor cigarettes, or has a
positive urine cotinine test at the Week 160/End-of- Study Visit in Study GTi1201 that
is due to smoking.

- Has current evidence of chronic alcoholism or illicit drug abuse (addiction).

- Is currently participating in another investigational product (IP) study.

- Has a history of anaphylaxis or severe systemic response to any plasma- derived
alpha1-PI preparation or other blood product(s).

- In the opinion of the investigator, is likely to have compliance problems with the
protocol and the procedures of the protocol.

- Has any medical condition that the investigator feels might confound the results of
the study or pose an additional risk to the subject during study participation.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/07/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2029
Actual
Sample size
Target
290
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
St Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [3] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [4] 0 0
St Vincent's Hospital Melbourne - Fitzroy
Recruitment hospital [5] 0 0
Institute for Respiratory Health Inc - Nedlands
Recruitment postcode(s) [1] 0 0
2010 - Darlinghurst
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
4032 - Chermside
Recruitment postcode(s) [4] 0 0
3065 - Fitzroy
Recruitment postcode(s) [5] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Florida
Country [3] 0 0
United States of America
State/province [3] 0 0
North Carolina
Country [4] 0 0
United States of America
State/province [4] 0 0
Oregon
Country [5] 0 0
United States of America
State/province [5] 0 0
South Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
Canada
State/province [7] 0 0
Halifax
Country [8] 0 0
Canada
State/province [8] 0 0
Toronto
Country [9] 0 0
Denmark
State/province [9] 0 0
Arhus C
Country [10] 0 0
Denmark
State/province [10] 0 0
Hellerup
Country [11] 0 0
Estonia
State/province [11] 0 0
Tallinn
Country [12] 0 0
Finland
State/province [12] 0 0
Turku
Country [13] 0 0
France
State/province [13] 0 0
Rhone
Country [14] 0 0
New Zealand
State/province [14] 0 0
Auckland
Country [15] 0 0
New Zealand
State/province [15] 0 0
Christchurch
Country [16] 0 0
New Zealand
State/province [16] 0 0
Hamilton
Country [17] 0 0
Poland
State/province [17] 0 0
Krakow
Country [18] 0 0
Poland
State/province [18] 0 0
Warszawa
Country [19] 0 0
Russian Federation
State/province [19] 0 0
Barnaul
Country [20] 0 0
Sweden
State/province [20] 0 0
Göteborg
Country [21] 0 0
Sweden
State/province [21] 0 0
Malmö
Country [22] 0 0
Sweden
State/province [22] 0 0
Stockholm

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Grifols Therapeutics LLC
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled
GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data
for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin
deficiency (AATD).
Trial website
https://clinicaltrials.gov/ct2/show/NCT02796937
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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/ct2/show/NCT02796937