Clinical trial NCT04683029: A Study of Guselkumab in Participants With Systemic Sclerosis
Official title: A Multicenter, Randomized, Placebo-controlled, Double-blind, Proof-of-concept Study of Guselkumab in Participants With Systemic Sclerosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Scleroderma, Systemic
Lead sponsor: Janssen Pharmaceutical K.K.
Sponsor class: INDUSTRY
First posted: 2020-12-24
Enrollment: 56
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate the efficacy of guselkumab in participants with systemic sclerosis (SSc).


Eligibility criteria: Inclusion criteria:

* Diagnosis of systemic sclerosis (SSc) according to American College of Rheumatology (ACR) and European League Against Rheumatism (EULAR) 2013 criteria
* Diffuse cutaneous SSc according to the LeRoy criteria that is, skin fibrosis proximal to the elbows and knees in addition to acral fibrosis
* Disease duration of ≤36 months (defined as time from first non-Raynaud phenomenon manifestation).
* Greater than or equal to (\>=) 10 and less than or equal to (\<=) 22 modified Rodnan skin score (mRSS) units at screening and Week 0
* Forced vital capacity (FVC) \>= 60 percent (%) of predicted at screening
* Diffusing capacity of the lung for carbon monoxide (DLCO) \>= 40% of predicted (hemoglobin-corrected) at screening.
* Participants who meet 1 of the following criteria at screening: increase of \>=3 mRSS units, compared with an assessment performed within the previous 2 to 6 months; Involvement of 1 new body area with an increase of \>=2 mRSS units compared with an assessment performed within the previous 2 to 6 months; and Involvement of 2 new body areas with increase of \>=1 mRSS units compared with the assessment within the previous 2 to 6 months

Exclusion Criteria:

* History of liver or renal insufficiency (estimated creatinine clearance below 60 milliliter per minute \[mL/min\]); significant cardiac, vascular, pulmonary, gastrointestinal, endocrine, neurologic, hematologic, rheumatologic, psychiatric, or metabolic disturbances
* Has any known severe or uncontrolled SSc complications including hemoptysis, pulmonary hemorrhage, renal crisis
* Has an interstitial lung disease requiring oxygen therapy
* Has any rheumatic disease other than SSc such as rheumatoid arthritis (RA), polymyalgia rheumatica (PMR), systemic lupus erythematosus, polymyositis/dermatomyositis that could interfere with assessment of SSc
* Has a current diagnosis or signs or symptoms of severe, progressive, or uncontrolled renal, cardiac, vascular, pulmonary, gastrointestinal, endocrine, neurologic, hematologic, rheumatologic, psychiatric, or metabolic disturbances. (or, in the investigator's opinion, any other concomitant medical condition that places the participant at risk by participating in this study)

Primary outcomes:
measure: Change From Baseline in Modified Rodnan Skin Score (mRSS) at Week 24
timeFrame: Baseline and Week 24
Secondary outcomes:
measure: Change From Baseline in Modified Rodnan Skin Score at Week 52
timeFrame: Baseline and Week 52
measure: Percentage of Participants Who Experienced Worsening of Modified Rodnan Skin Score at Week 24 and Week 52
timeFrame: Week 24 and Week 52
measure: Percentage of Participants Who Achieved a Score of 0.6 in American College of Rheumatology Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (ACR CRISS) at Week 24 and Week 52
timeFrame: Week 24 and Week 52
measure: Change From Baseline in Forced Vital Capacity (FVC) at Week 24 and Week 52
timeFrame: Baseline, Week 24 and Week 52
measure: Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 24 and Week 52
timeFrame: Baseline, Week 24 and Week 52
measure: Change From Baseline in the Measured Absolute Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) at Week 24 and Week 52
timeFrame: Baseline, Week 24 and Week 52
measure: Change From Baseline in the Percent Predicted Diffusing Capacity of the Lung for Carbon Monoxide (DLCO) at Week 24 and Week 52
timeFrame: Baseline, Week 24 and Week 52
measure: Change From Baseline in Digital Ulcer Counts at Week 24 and Week 52
timeFrame: Baseline, Week 24 and Week 52
measure: Change From Baseline in Health Assessment Questionnaire-Disability Index (HAQ-DI) Score at Week 24 and Week 52
timeFrame: Baseline, Week 24 and Week 52
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Through Week 24 and Week 52
timeFrame: From Baseline (Week 0) up to Week 24 and Week 52
measure: Number of Participants With Treatment-Emergent Serious Adverse Events (TESAEs) Through Week 24 and Week 52
timeFrame: From Baseline (Week 0) up to Week 24 and Week 52
measure: Number of Participants With Adverse Events of Special Interest (AESI) Through Week 24 and Week 52
timeFrame: From Baseline (Week 0) up to Week 24 and Week 52
measure: Serum Concentration of Guselkumab
timeFrame: Pre-dose at Weeks 0, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 and Post- dose at Weeks 0, 4, 8
measure: Number of Participants With Anti-Guselkumab Antibody
timeFrame: From Baseline (Week 0) up to Week 52
Other outcomes:
Locations:
city: Aichi
country: Japan
facility: Chukyo Hospital
city: Tokyo
country: Japan
facility: The University of Tokyo Hospital
city: Wakayama
country: Japan
facility: Wakayama Medical University Hospital
city: Yoshida
country: Japan
facility: University of Fukui Hospital
Study officials:
affiliation: Janssen Pharmaceutical K.K.
name: Janssen Pharmaceutical K.K., Japan Clinical Trial
role: STUDY_DIRECTOR
References: