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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04138485




Registration number
NCT04138485
Ethics application status
Date submitted
14/10/2019
Date registered
24/10/2019
Date last updated
17/11/2020

Titles & IDs
Public title
Efficacy and Safety of IgPro10 in Adults With Systemic Sclerosis (SSc)
Scientific title
A Randomized, Multicenter, Double-Blind, Placebo Controlled, Phase 2 Study to Evaluate the Efficacy and Safety of IgPro10 (Intravenous Immunoglobulin, Privigen®) for the Treatment of Adults With Systemic Sclerosis
Secondary ID [1] 0 0
2019-000906-31
Secondary ID [2] 0 0
IgPro10_2001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Diffuse Cutaneous Systemic Sclerosis 0 0
Condition category
Condition code
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases
Skin 0 0 0 0
Dermatological conditions
Skin 0 0 0 0
Other skin conditions

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - IgPro10
Treatment: Other - Placebo

Experimental: IgPro10 - 10% liquid formulation of human immunoglobulin for intravenous use

Placebo comparator: Placebo - 0.5% human albumin solution stabilized with 250 mmol/L L-proline


Treatment: Other: IgPro10
10% liquid formulation of human immunoglobulin for IVIG

Treatment: Other: Placebo
0.5% human albumin solution stabilized with 250 mmol/L L-proline
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Response on American College of Rheumatology Combined Response Index in Diffuse Systemic Sclerosis (ACR CRISS) score in IgPro10 vs Placebo
Timepoint [1] 0 0
Over 48 weeks
Secondary outcome [1] 0 0
Proportion of subjects meeting cardiopulmonary or renal failure criteria in ACR CRISS Step 1 events
Timepoint [1] 0 0
Over 48 weeks
Secondary outcome [2] 0 0
Proportion of responders (ACR CRISS > 0.6)
Timepoint [2] 0 0
Over 48 weeks
Secondary outcome [3] 0 0
Mean change from Baseline in Modified Rodnan Skin Score (mRSS)
Timepoint [3] 0 0
Baseline and over48 weeks
Secondary outcome [4] 0 0
Mean change from Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)
Timepoint [4] 0 0
Baseline and over 48 weeks
Secondary outcome [5] 0 0
Mean change from Baseline in Forced Vital Capacity (FVC)% predicted
Timepoint [5] 0 0
Baseline and over 48 weeks
Secondary outcome [6] 0 0
Mean change from Baseline in diffusing capacity of lung for carbon monoxide (DLCO)% predicted
Timepoint [6] 0 0
Baseline and over 48 weeks
Secondary outcome [7] 0 0
Mean change from Baseline in Physician Global Assessment (MDGA)
Timepoint [7] 0 0
Baseline and over 48 weeks
Secondary outcome [8] 0 0
Mean change from Baseline in Patient Global Assessment (PGA)
Timepoint [8] 0 0
Baseline and over 48 weeks
Secondary outcome [9] 0 0
Mean change from Baseline in UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA SCTC GIT 2.0) total score and subscale
Timepoint [9] 0 0
Baseline and over 48 weeks
Secondary outcome [10] 0 0
Mean change from Baseline in Scleroderma Skin Patient Reported Outcome (SSPRO) score in IgPro10 vs Placebo
Timepoint [10] 0 0
Baseline and up to 48 weeks
Secondary outcome [11] 0 0
Proportion of responders in mRSS
Timepoint [11] 0 0
Up to 48 weeks
Secondary outcome [12] 0 0
Time to treatment failure (time from first infusion to time of first event) in IgPro10 vs Placebo
Timepoint [12] 0 0
Over 48 weeks
Secondary outcome [13] 0 0
Proportion of subjects with events at Week 48 in IgPro10 vs Placebo
Timepoint [13] 0 0
Over 48 weeks
Secondary outcome [14] 0 0
Mean change from Baseline in Cochin Hand Function Scale in IgPro10 vs Placebo
Timepoint [14] 0 0
Baseline and over 48 weeks
Secondary outcome [15] 0 0
Mean change from Baseline in Scleroderma Health Assessment Questionnaire (SHAQ) score in IgPro10 vs Placebo
Timepoint [15] 0 0
Baseline and over 48 weeks
Secondary outcome [16] 0 0
Mean change from baseline in muscle strength as measured by Manual Muscle Testing 8 (MMT) in IgPro10 vs Placebo
Timepoint [16] 0 0
Baseline and over 48 weeks
Secondary outcome [17] 0 0
Number of subjects with adverse events (AEs) including any AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)
Timepoint [17] 0 0
Over 48 weeks
Secondary outcome [18] 0 0
Percentage of subjects with AEs, TEAEs, SAEs, AESIs
Timepoint [18] 0 0
Over 48 weeks
Secondary outcome [19] 0 0
Concentration of serum trough IgG levels at Baseline and prior to first infusion
Timepoint [19] 0 0
Baseline and up to 72 weeks
Secondary outcome [20] 0 0
Mean change from Baseline in Modified Rodnan skin score (mRSS)
Timepoint [20] 0 0
Baseline and over 72 weeks
Secondary outcome [21] 0 0
Mean change from Baseline in Patient global assessment (PGA)
Timepoint [21] 0 0
Baseline and over 72 weeks
Secondary outcome [22] 0 0
Proportion of responders (ACR CRISS > 0.6)
Timepoint [22] 0 0
Over 72 weeks
Secondary outcome [23] 0 0
Mean change from Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)
Timepoint [23] 0 0
Baseline and over 72 weeks
Secondary outcome [24] 0 0
Mean change from Baseline in Forced Vital Capacity (FVC)% predicted
Timepoint [24] 0 0
Baseline and over 72 weeks
Secondary outcome [25] 0 0
Mean change from Baseline in diffusing capacity of lung for carbon monoxide (DLCO)% predicted
Timepoint [25] 0 0
Baseline and over 72 weeks
Secondary outcome [26] 0 0
Mean change from Baseline in Physician Global Assessment (MDGA)
Timepoint [26] 0 0
Baseline and over 72 weeks
Secondary outcome [27] 0 0
Number of subjects with adverse events (AEs) including any AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)
Timepoint [27] 0 0
Over 72 weeks
Secondary outcome [28] 0 0
Percentage of subjects with AEs, TEAEs, SAEs, AESIs
Timepoint [28] 0 0
Over 72 weeks

Eligibility
Key inclusion criteria
* 1. Age =18 years (male or female) at time of providing written informed consent
* Documented diagnosis of SSc according to ACR / EULAR criteria 2013
* mRSS = 15 and = 45
* Disease duration = 5 years defined as the time from the first non-Raynaud's phenomenon manifestation
* Subjects within first 18 months of disease duration from first non-Raynaud's phenomenon manifestation.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Primary rheumatic autoimmune disease other than dcSSc, including but not limited to rheumatoid arthritis, systemic lupus erythematosus, mixed connective tissue disorder, polymyositis, and dermatomyositis, as determined by the investigator Note: Subjects with fibromyalgia, secondary Sjogren's syndrome, and scleroderma-associated myopathy or myositis at Screening are not excluded
* Positive anti-centromere autoantibodies at Screening
* Evidence of severe chronic kidney disease with estimated glomerular filtration rate < 45 mL/min/1.73 m2 (as calculated by the Chronic Kidney Disease Epidemiology Collaboration equation) or receiving dialysis. Additionally, subjects with current confirmed diagnosis of diabetes mellitus and requiring medication, with eGFR < 90 mL/min/1.73m2 will be excluded from the study.
* History of documented thrombotic episode eg, PE, DVT, myocardial infarction, thromboembolic stroke at any time Note: past superficial thrombophlebitis more than two years from Screening is not exclusionary
* Documented thrombophilic abnormalities including blood hyperviscosity, protein S or protein C deficiency, anti-thrombin-3 deficiency, plasminogen deficiency, antiphospholipid syndrome, Factor V Leiden mutation, dysfibrinogenemia, or prothrombin G20210A mutation
* Greater than 3 specified current risk factors for TEEs (documented and currents conditions): atrial fibrillation, coronary disease, diabetes mellitus, dyslipidemia, hypertension, obesity (Body Mass Index = 30 kg/m2), recent significant trauma, and immobility (wheelchair-bound or bedridden)
* Ongoing active serious infection at Screening (including, but not limited to, pneumonia, bacteremia/septicemia, osteomyelitis/septic arthritis, bacterial meningitis, or visceral abscess)
* Malignancy in the past 2 years, except for non-melanoma skin cancer, cervical carcinoma in situ, or other in situ cancer if it has been excised and treated within in the past year
* Known hypoalbuminemia, protein-losing enteropathies, and any proteinuria (defined by total urine protein concentration > 0.2 g/L)
* Known IgA deficiency or serum IgA level < 5% lower limit of normal

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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
20/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/09/2020
Sample size
Target
Accrual to date
Final
0
Recruitment in Australia
Recruitment state(s)
NSW,SA
Recruitment hospital [1] 0 0
John Hunter Hospital / Autoimmune Resource and Research Centre - New Lambton Heights
Recruitment hospital [2] 0 0
PARC Clinical Research - Adelaide
Recruitment postcode(s) [1] 0 0
2305 - New Lambton Heights
Recruitment postcode(s) [2] 0 0
5005 - Adelaide
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
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Colorado
Country [4] 0 0
United States of America
State/province [4] 0 0
District of Columbia
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Louisiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Maryland
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
New Jersey
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
South Carolina
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
Argentina
State/province [16] 0 0
Buenos Aires
Country [17] 0 0
Argentina
State/province [17] 0 0
Ciudad Autonoma de Buenos Aires
Country [18] 0 0
Argentina
State/province [18] 0 0
Rosario
Country [19] 0 0
Belgium
State/province [19] 0 0
Gent
Country [20] 0 0
Belgium
State/province [20] 0 0
Leuven
Country [21] 0 0
Canada
State/province [21] 0 0
Toronto
Country [22] 0 0
France
State/province [22] 0 0
Caen
Country [23] 0 0
France
State/province [23] 0 0
Lille Cedex
Country [24] 0 0
France
State/province [24] 0 0
Nantes
Country [25] 0 0
France
State/province [25] 0 0
Paris
Country [26] 0 0
France
State/province [26] 0 0
Rennes
Country [27] 0 0
France
State/province [27] 0 0
Rouen cedex
Country [28] 0 0
France
State/province [28] 0 0
Strasbourg
Country [29] 0 0
Germany
State/province [29] 0 0
Bad Nauheim
Country [30] 0 0
Germany
State/province [30] 0 0
Berlin
Country [31] 0 0
Germany
State/province [31] 0 0
Freiburg
Country [32] 0 0
Germany
State/province [32] 0 0
Köln
Country [33] 0 0
Germany
State/province [33] 0 0
Mainz
Country [34] 0 0
Germany
State/province [34] 0 0
Tuebingen
Country [35] 0 0
Germany
State/province [35] 0 0
Ulm
Country [36] 0 0
Germany
State/province [36] 0 0
Wuppertal
Country [37] 0 0
Italy
State/province [37] 0 0
Ancona
Country [38] 0 0
Italy
State/province [38] 0 0
Bari
Country [39] 0 0
Italy
State/province [39] 0 0
Brescia
Country [40] 0 0
Italy
State/province [40] 0 0
Firenze
Country [41] 0 0
Italy
State/province [41] 0 0
L'Aquila
Country [42] 0 0
Italy
State/province [42] 0 0
Milano
Country [43] 0 0
Italy
State/province [43] 0 0
Modena
Country [44] 0 0
Italy
State/province [44] 0 0
Napoli
Country [45] 0 0
Italy
State/province [45] 0 0
Pavia
Country [46] 0 0
Italy
State/province [46] 0 0
Rome
Country [47] 0 0
Mexico
State/province [47] 0 0
Ciudad de México
Country [48] 0 0
Mexico
State/province [48] 0 0
Guadalajara
Country [49] 0 0
Mexico
State/province [49] 0 0
Jalisco
Country [50] 0 0
Mexico
State/province [50] 0 0
Mexico City
Country [51] 0 0
Mexico
State/province [51] 0 0
Mexico
Country [52] 0 0
Mexico
State/province [52] 0 0
San Luis Potosi
Country [53] 0 0
Poland
State/province [53] 0 0
Bialystok
Country [54] 0 0
Poland
State/province [54] 0 0
Gdansk
Country [55] 0 0
Poland
State/province [55] 0 0
Katowice
Country [56] 0 0
Poland
State/province [56] 0 0
Warszawa
Country [57] 0 0
Spain
State/province [57] 0 0
A Coruna
Country [58] 0 0
Spain
State/province [58] 0 0
Barcelona
Country [59] 0 0
Spain
State/province [59] 0 0
Madrid
Country [60] 0 0
Spain
State/province [60] 0 0
Sevilla
Country [61] 0 0
Spain
State/province [61] 0 0
Valencia
Country [62] 0 0
Switzerland
State/province [62] 0 0
Saint Gallen
Country [63] 0 0
United Kingdom
State/province [63] 0 0
Chester
Country [64] 0 0
United Kingdom
State/province [64] 0 0
Leeds
Country [65] 0 0
United Kingdom
State/province [65] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
This randomized, multicenter, double-blind (DB), placebo controlled, phase 2 study will evaluate the efficacy and safety of IgPro10. The DB Treatment Period will be followed by a 24-week Open-label (OL) Treatment Period.

Eligible subjects will be randomized at Baseline in a 2:1 ratio of treatment IgPro10 or placebo in the DB Treatment Period. All subjects who enter OL Treatment Period will receive IgPro10.
Trial website
https://clinicaltrials.gov/study/NCT04138485
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
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/NCT04138485