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Report for Clinical Trial NCT04138485

Developed by Shray Alag, 2020.
SNP Clinical Trial Gene

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

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.

NCT04138485 Diffuse Cutaneous Systemic Sclerosis
MeSH:Scleroderma, Systemic Scleroderma, Diffuse Sclerosis

2 Interventions

Name: IgPro10

Description: 10% liquid formulation of human immunoglobulin for IVIG
Type: Biological
Group Labels: 1

IgPro10

Name: Placebo

Description: 0.5% human albumin solution stabilized with 250 mmol/L L-proline
Type: Biological
Group Labels: 1

Placebo


Primary Outcomes

Measure: Response on American College of Rheumatology Combined Response Index in Diffuse Systemic Sclerosis (ACR CRISS) score in IgPro10 vs Placebo

Time: Over 48 weeks

Secondary Outcomes

Measure: Proportion of subjects meeting cardiopulmonary or renal failure criteria in ACR CRISS Step 1 events

Time: Over 48 weeks

Measure: Proportion of responders (ACR CRISS > 0.6)

Time: Over 48 weeks

Measure: Mean change from Baseline in Modified Rodnan Skin Score (mRSS)

Time: Baseline and over48 weeks

Measure: Mean change from Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)

Time: Baseline and over 48 weeks

Measure: Mean change from Baseline in Forced Vital Capacity (FVC)% predicted

Time: Baseline and over 48 weeks

Measure: Mean change from Baseline in diffusing capacity of lung for carbon monoxide (DLCO)% predicted

Time: Baseline and over 48 weeks

Description: 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)

Measure: Mean change from Baseline in Physician Global Assessment (MDGA)

Time: Baseline and over 48 weeks

Description: 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)

Measure: Mean change from Baseline in Patient Global Assessment (PGA)

Time: Baseline and over 48 weeks

Description: 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.

Measure: Mean change from Baseline in UCLA Scleroderma Clinical Trial Consortium Gastrointestinal Tract 2.0 (UCLA SCTC GIT 2.0) total score and subscale

Time: Baseline and over 48 weeks

Measure: Mean change from Baseline in Scleroderma Skin Patient Reported Outcome (SSPRO) score in IgPro10 vs Placebo

Time: Baseline and up to 48 weeks

Description: Response is decrease of mRSS ≥ 5 points and change of ≥ 25% from Baseline in IgPro10 vs Placebo

Measure: Proportion of responders in mRSS

Time: Up to 48 weeks

Description: 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

Measure: Time to treatment failure (time from first infusion to time of first event) in IgPro10 vs Placebo

Time: Over 48 weeks

Description: 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

Measure: Proportion of subjects with events at Week 48 in IgPro10 vs Placebo

Time: Over 48 weeks

Measure: Mean change from Baseline in Cochin Hand Function Scale in IgPro10 vs Placebo

Time: Baseline and over 48 weeks

Measure: Mean change from Baseline in Scleroderma Health Assessment Questionnaire (SHAQ) score in IgPro10 vs Placebo

Time: Baseline and over 48 weeks

Measure: Mean change from baseline in muscle strength as measured by Manual Muscle Testing 8 (MMT) in IgPro10 vs Placebo

Time: Baseline and over 48 weeks

Measure: Number of subjects with adverse events (AEs) including any AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)

Time: Over 48 weeks

Measure: Percentage of subjects with AEs, TEAEs, SAEs, AESIs

Time: Over 48 weeks

Measure: Concentration of serum trough IgG levels at Baseline and prior to first infusion

Time: Baseline and up to 72 weeks

Measure: Mean change from Baseline in Modified Rodnan skin score (mRSS)

Time: Baseline and over 72 weeks

Measure: Mean change from Baseline in Patient global assessment (PGA)

Time: Baseline and over 72 weeks

Measure: Proportion of responders (ACR CRISS > 0.6)

Time: Over 72 weeks

Measure: Mean change from Baseline in Health Assessment Questionnaire - Disability Index (HAQ-DI)

Time: Baseline and over 72 weeks

Measure: Mean change from Baseline in Forced Vital Capacity (FVC)% predicted

Time: Baseline and over 72 weeks

Measure: Mean change from Baseline in diffusing capacity of lung for carbon monoxide (DLCO)% predicted

Time: Baseline and over 72 weeks

Measure: Mean change from Baseline in Physician Global Assessment (MDGA)

Time: Baseline and over 72 weeks

Measure: Number of subjects with adverse events (AEs) including any AEs, treatment-emergent AEs (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)

Time: Over 72 weeks

Measure: Percentage of subjects with AEs, TEAEs, SAEs, AESIs

Time: Over 72 weeks

Purpose: Treatment

Allocation: Randomized

Parallel Assignment


There is one SNP

SNPs


1 G20210A

- 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 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. --- G20210A ---

- 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 Diffuse Cutaneous Systemic Sclerosis Scleroderma, Systemic Scleroderma, Diffuse Sclerosis null --- G20210A ---



HPO Nodes