Clinical trial NCT03957681: A Phase 3 Study of KHK4827 in Patients With Systemic Sclerosis
Official title: A Phase 3, Placebo-controlled, Double-blind Comparative Study of KHK4827 With an Open-label Extension Period in Subjects With Systemic Sclerosis Who Have Moderate to Severe Skin Thickening
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Moderate to Severe Systemic Sclerosis
Keywords: systemic sclerosis
brodalumab
KHK4827
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2019-05-21
Enrollment: 100
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: To evaluate the efficacy and safety of KHK4827 in patients with systemic sclerosis who have moderate to severe skin thickening


Eligibility criteria: Inclusion Criteria:

* Subject meets the criteria for diagnosis of the Diagnostic Criteria, Severity Classification, and Clinical Practice Guidelines for Systemic Sclerosis （Japanese Dermatological Association 2016） at the pre-examination
* Subject presented the first symptoms of sclerosis other than Raynaud's phenomenon within 60 months before enrollment
* Subject who has systemic sclerosis accompanied by moderate to severe skin thickening with an mRSS of 10 to \<30 at the pre-examination and who has progressing skin thickening

Exclusion Criteria:

1. Any of the following significant concomitant diseases:

   * Type 1 diabetes
   * Poorly controlled type 2 diabetes (HbA1c \> 8.5%)
   * Congestive heart failure (Class II to IV of the New York Heart Association Functional Classification)
   * Myocartial infarction, unstable angina, or stroke occurring within 12 months before the first dose of investigational product
   * Poorly controlled hypertension (systolic pressure \> 150 mm Hg or diastolic pressure \> 90 mg Hg at screening)
   * Severe chronic lung disease (%FVC \< 60% and %DLco \< 40%, calculated according to the Reference Values for Spirometry, Including Viral Capacity, in Japanese Adults Calcluated with the LMS Method and Compared with Previous Values \[Japanese Respiratory Society\])
   * Major chronic inflammatory diseases or connective tissue diseases other than scleroderma
2. Patient has a history or evidence of a psychiatric disorder, alcohol and/or substance abuse, or any other mental health disorder that, in the opinion of the investigators, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion
3. Patient has a history or evidence of suicidal ideation (severity of 4 or 5) or any suicidal behavior based on an assessment with the Columbia-Suicide Severity Rating Scale (C-SSRS) at enrollment
4. Patient has severe depression based on a total score of ≥ 15 on the Patient Health Questionnaire-8 (PHQ-8) at enrollment

Primary outcomes:
measure: Change in modified Rodnan skin score (mRSS) from baseline at Week 24
timeFrame: at least 2 weeks up to 24 weeks
Secondary outcomes:
measure: Change in modified Rodnan skin score (mRSS) from baseline at Week 52
timeFrame: at least 2 weeks up to 52 weeks
Other outcomes:
Locations:
city: Nagoya
country: Japan
facility: Chukyo Hospital
state: Aichi
city: Tokyo
country: Japan
facility: The University of Tokyo Hospital
state: Bunkyo-ku
city: Yoshida-gun
country: Japan
facility: The University of Fukui Hospital
state: Fukui
city: Kawasaki
country: Japan
facility: St. Marianna University School of Medecine Hospital
state: Kanagawa
city: Suita
country: Japan
facility: Osaka University Hospital
state: Osaka
city: Fukushima
country: Japan
facility: Fukushima Medical University Hospital
Study officials:
References: