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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/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
Other interventions - IgPro10
Other interventions - 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


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

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

Intervention code [1] 0 0
Other interventions
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) - MDGA evaluates the overall impact of SSc on the participant as assessed by the physician on a 11-point Numeric rating scale scale from 0 (excellent) to 10 (extremely poor)
Timepoint [7] 0 0
Baseline and over 48 weeks
Secondary outcome [8] 0 0
Mean change from Baseline in Patient Global Assessment (PGA) - PGA evaluates the overall impact of SSc on the participant as assessed by the physician on a 11-point Numeric rating scale scale from 0 (excellent) to 10 (extremely poor)
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 - This survey consists of 34 questions and items are scored on a scale of 0 (better health) to 3 (worse health). Scores are combined to form total score.
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 - Response is decrease of mRSS = 5 points and change of = 25% from Baseline in IgPro10 vs Placebo
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 - Treatment failure - defined as occurrence of SSc associated complications in ACR CRISS step 1 events, digital ischemia (requiring hospitalization for IV prostacyclin, surgical intervention or amputation), serious gastrointestinal events (events requiring parenteral nutrition due to SSc -such as total parenteral nutrition or enteral nutrition), all-cause mortality
Timepoint [12] 0 0
Over 48 weeks
Secondary outcome [13] 0 0
Proportion of subjects with events at Week 48 in IgPro10 vs Placebo - Events defined as occurrence of SSc associated complications in ACR CRISS step 1 events, digital ischemia (requiring hospitalization for IV prostacyclin, surgical intervention or amputation), serious gastrointestinal events (events requiring parenteral nutrition due to SSc -such as total parenteral nutrition or enteral nutrition), all -cause mortality
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
Gender
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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/show/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/show/NCT04138485