A Phase 3 Study of KHK4827 in Patients With Systemic Sclerosis

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2023
• Main ID: NCT03957681
• Date of registration: 16/05/2019
• Prospective Registration: Yes
• Primary sponsor: Kyowa Kirin Co., Ltd.
• Public title: A Phase 3 Study of KHK4827 in Patients With Systemic Sclerosis
• Scientific 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
• Date of first enrolment: May 23, 2019
• Target sample size: 100
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03957681
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 3

Countries of recruitment

Japan

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Moderate to Severe Systemic Sclerosis

Intervention(s)

Drug: Placebo

Drug: KHK4827

Primary Outcome(s)

Change in modified Rodnan skin score (mRSS) from baseline at Week 24 [Time Frame: at least 2 weeks up to 24 weeks]

Secondary Outcome(s)

Change in modified Rodnan skin score (mRSS) from baseline at Week 52 [Time Frame: at least 2 weeks up to 52 weeks]

Secondary ID(s)

4827-008

Source(s) of Monetary Support

Please refer to primary and secondary sponsors