A Study of KHK4827 in Patients With Systemic Sclerosis

A Phase 1, Open-label, Multiple-dose Study of KHK4827 in Subjects With Systemic Sclerosis

Exploratory evaluation of pharmacokinetics and safety of KHK4827 in subjects with systemic sclerosis

Study Overview

• Status: Completed
• Conditions: Systemic Sclerosis
• Intervention / Treatment: Drug: KHK4827

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 1

Contacts and Locations

Study Locations

• Japan
  • Tokyo
    • Bunkyo-ku, Tokyo, Japan, 113-8655
      • The University of Tokyo Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 70 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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)
   • Myocardial 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 (%Forced Vital Capacity (FVC) < 60% and %Diffusing capacity of lung for carbon monoxide (DLco) < 40%, calculated according to the Reference Values for Spirometry, Including Viral Capacity, in Japanese Adults Calculated 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: KHK4827	Drug: KHK4827; 210 mg every 2 weeks (Q2W), subcutaneous (SC) injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
serum KHK4827 concentration	Pre-dose, Week 1, 2, 4, 8, 12, 16, 22, 24, 36, 48, 52 and every 24 weeks through study completion until 2025.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in modified Rodnan skin score (mRSS) from baseline	mRSS: scored 0(normal), 1(mild), 2(moderate), or 3(severe) per each site, assessed in 17 different body sites, total score=51	Pre-dose, Week 1, 2, 4, 8, 12, 16, 20, 24, 28, 32, 36, 40, 44, 48, 52 and every 8 weeks through study completion until 2025.

Collaborators and Investigators

• Sponsor: Kyowa Kirin Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): October 6, 2017
• Primary Completion (Actual): January 31, 2019
• Study Completion (Actual): March 31, 2025

Study Registration Dates

• First Submitted: April 13, 2020
• First Submitted That Met QC Criteria: April 28, 2020
• First Posted (Actual): April 29, 2020

Study Record Updates

• Last Update Posted (Actual): July 11, 2025
• Last Update Submitted That Met QC Criteria: July 9, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Keywords: systemic sclerosis; brodalumab; KHK4827
• Additional Relevant MeSH Terms: Pathologic Processes; Connective Tissue Diseases; Skin Diseases; Sclerosis; Scleroderma, Systemic; Scleroderma, Diffuse; Dermatologic Agents; Brodalumab
• Other Study ID Numbers: 4827-007

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: There is a plan to make IPD and related data dictionaries available. The datasets generated and/or analyzed during the study sponsored by Kyowa Kirin will be available in the Vivli repository, https://vivli.org/ourmember/kyowa-kirin/ as long as conditions of data disclosure specified in the policy section of the Vivli website are satisfied.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No