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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/NCT01983241


Additional trial details provided through ANZCTR are available at the end of this record.


Registration number
NCT01983241
Ethics application status
Date submitted
28/10/2013
Date registered
13/11/2013
Date last updated
7/03/2024

Titles & IDs
Public title
Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
Scientific title
A Randomized, Double-Blind, Placebo Controlled Study to Assess the Efficacy and Safety of Two Dose Regimens (60 mg/kg and 120 mg/kg) of Weekly Intravenous Alpha1 Proteinase Inhibitor (Human) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency
Secondary ID [1] 0 0
GTi1201
Universal Trial Number (UTN)
Trial acronym
SPARTA
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pulmonary Emphysema in Alpha-1 PI 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
Other interventions - 0.9% Sodium Chloride for Injection, USP

Experimental: Alpha-1 MP 60 mg/kg - Alpha-1 MP 60 mg/kg administered weekly by IV infusion for 156 weeks

Experimental: Alpha-1 MP 120 mg/kg - Alpha-1 MP 120 mg/kg administered weekly by IV infusion for 156 weeks

Placebo Comparator: Placebo - 0.9% Sodium Chloride for Injection, USP, administered weekly by IV infusion for 156 weeks


Other interventions: Alpha-1 MP


Other interventions: 0.9% Sodium Chloride for Injection, USP
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline in Whole lung PD15 (15th percentile point)
Timepoint [1] 0 0
Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Secondary outcome [1] 0 0
Adverse Events (AEs)
Timepoint [1] 0 0
Week -3 through Week 160
Secondary outcome [2] 0 0
Serious Adverse Events (SAEs)
Timepoint [2] 0 0
Week -3 through Week 160
Secondary outcome [3] 0 0
Discontinuations from the study due to AEs
Timepoint [3] 0 0
Week -3 through Week 160
Secondary outcome [4] 0 0
Severe COPD Exacerbations
Timepoint [4] 0 0
Week -3 through Week 160
Secondary outcome [5] 0 0
Change from Baseline in PD15 of the basal lung region
Timepoint [5] 0 0
Week -3 (baseline measure), Week 52, Week 104, Week 130, Week 156
Secondary outcome [6] 0 0
Change from baseline in carbon monoxide diffusing capacity (DLco)
Timepoint [6] 0 0
Weeks 26, 52, 78, 104, 130 and 156
Secondary outcome [7] 0 0
Changes from baseline in forced expiratory volume in 1 second (FEV1)
Timepoint [7] 0 0
Weeks 26, 52, 78, 104, 130 and 156
Secondary outcome [8] 0 0
Change from baseline in Saint George's Respiratory Questionnaire: Minimum value = 0, maximum value = 100, higher scores indicate worse condition
Timepoint [8] 0 0
Weeks 26, 52, 78, 104, 130 and 156
Secondary outcome [9] 0 0
Change from baseline in the EuroQoL (Quality of Life)- 5 Dimension- 5 Level (EQ-5D-5L): Minimum value (for each one of the 5 levels) = 1, maximum value (for each one of the 5 levels) = 5, higher scores indicate worse condition
Timepoint [9] 0 0
Weeks 26, 52, 78, 104, 130 and 156

Eligibility
Key inclusion criteria
- Have a documented total alpha1-PI serum level < 11 µM.

- Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null),
(null)(null), S(null), or "at-risk" alleles.

- At the Screening (Week -3) Visit, have a post-bronchodilator forced expiratory volume
in 1 second (FEV1) = 30% and < 80% of predicted and FEV1/forced vital capacity (FVC) <
70% (Global Initiative for Chronic Obstructive Lung Disease [GOLD] stage II or III).

- Have a carbon monoxide diffusing capacity (DLCO) = 60% of predicted (corrected for
HgB) within the past 2 years OR evidence of pulmonary emphysema on CT scan within the
past 2 years per the Investigator's judgment.

- Have clinical evidence of pulmonary emphysema per the Investigator's judgment.
Minimum age
18 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Has received alpha1-PI augmentation therapy for more than 1 month within the six
months prior to the Screening Visit.

- Has received alpha1-PI augmentation therapy within one month of the Screening Visit.

- Has had a chronic obstructive pulmonary disease (COPD) exacerbation within the 5 weeks
prior to the Screening Visit or during the Screening Phase.

- Unable to physically (e.g., unable to fit inside the CT scanner) or mentally (e.g.,
claustrophobic) undergo a CT scan.

- History of lung or liver transplant.

- Any lung surgery during the past 2 years (excluding lung biopsy).

- On the waiting list for lung surgery, including lung transplant.

- Smoking during the past 12 months or a positive urine cotinine test at screening that
is due to smoking. Maybe on Nicotine replacement, including vapor cigarettes.

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

- Use of systemic steroids above a stable dose equivalent to 5 mg/day prednisone (i.e.,
10 mg every 2 days) within the 5 weeks prior to the Screening Visit (inhaled steroids
are not considered systemic steroids) or during the Screening Phase.

- Use of systemic or aerosolized antibiotics for a COPD exacerbation within the 5 weeks
prior to the Screening Visit or during the Screening Phase.

- Known selective or severe Immunoglobulin A (IgA) deficiency.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/11/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2027
Actual
Sample size
Target
Accrual to date
Final
345
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC,WA
Recruitment hospital [1] 0 0
St Vincent's Hospital Sydney - Sydney
Recruitment hospital [2] 0 0
The Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - Adelaide
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 - Sydney
Recruitment postcode(s) [2] 0 0
4032 - Chermside
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
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
Brazil
State/province [7] 0 0
Sao Paulo
Country [8] 0 0
Brazil
State/province [8] 0 0
São Paulo
Country [9] 0 0
Canada
State/province [9] 0 0
Newfoundland and Labrador
Country [10] 0 0
Canada
State/province [10] 0 0
Nova Scotia
Country [11] 0 0
Canada
State/province [11] 0 0
Ontario
Country [12] 0 0
Denmark
State/province [12] 0 0
Arhus C
Country [13] 0 0
Denmark
State/province [13] 0 0
Hellerup
Country [14] 0 0
Estonia
State/province [14] 0 0
Tallinn
Country [15] 0 0
Estonia
State/province [15] 0 0
Tartu
Country [16] 0 0
Finland
State/province [16] 0 0
Turku
Country [17] 0 0
France
State/province [17] 0 0
Rhone
Country [18] 0 0
New Zealand
State/province [18] 0 0
Auckland
Country [19] 0 0
New Zealand
State/province [19] 0 0
Christchurch
Country [20] 0 0
New Zealand
State/province [20] 0 0
Hamilton
Country [21] 0 0
Poland
State/province [21] 0 0
Krakow
Country [22] 0 0
Poland
State/province [22] 0 0
Warszawa
Country [23] 0 0
Russian Federation
State/province [23] 0 0
Barnaul
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Yaroslavl
Country [25] 0 0
Sweden
State/province [25] 0 0
Göteborg
Country [26] 0 0
Sweden
State/province [26] 0 0
Linköping
Country [27] 0 0
Sweden
State/province [27] 0 0
Malmö
Country [28] 0 0
Sweden
State/province [28] 0 0
Stockholm
Country [29] 0 0
Sweden
State/province [29] 0 0
Uppsala

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 multi-center, randomized, placebo-controlled, double blind clinical study to assess
the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156
weeks (i.e., 3 years) using computed tomography (CT) of the lungs as the main measure of
efficacy. The two Alpha-1 MP doses to be tested are 60 mg/kg and 120 mg/kg administered
weekly by IV infusion for 156 weeks. The study consists of an optional pre-screening phase,
Screening Phase, a 156-week Treatment Phase, and an End of Study Visit at Week 160.
Trial website
https://clinicaltrials.gov/ct2/show/NCT01983241
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/NCT01983241

Additional trial details provided through ANZCTR
Accrual to date
8
Recruiting in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 9
The Royal Adelaide Hospital
Recruitment postcode(s) [1] 9
5000
Funding & Sponsors
Primary sponsor
Primary sponsor name
Primary sponsor address
Primary sponsor country
Ethics approval
Ethics application status
Approved
 
Public notes

Contacts
Principal investigator
Title 13 0
Prof
Name 13 0
Mark Holmes
Address 13 0
Respiratory Clinical Trials Unit Thoracic Medicine, Royal Adelaide Hospital Adelaide, SA 5000
Country 13 0
Australia
Phone 13 0
+61 (0)8 7074 5244
Fax 13 0
Email 13 0
[email protected]
Contact person for public queries
Title 14 0
Ms
Name 14 0
Jenny McGrath
Address 14 0
Respiratory Clinical Trials Unit Thoracic Medicine, Royal Adelaide Hospital Adelaide, SA 5000
Country 14 0
Australia
Phone 14 0
+61 (0)8 7074 5244
Fax 14 0
Email 14 0
[email protected]
Contact person for scientific queries
Title 15 0
Name 15 0
Address 15 0
Country 15 0
Phone 15 0
Fax 15 0
Email 15 0