A Study of KHK4827 in Patients With Systemic Sclerosis

Phase 1

Active, not recruiting

• Conditions: Systemic Sclerosis
• Interventions: Drug: KHK4827

• Registration Number: NCT04368403
• Lead Sponsor: Kyowa Kirin Co., Ltd.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2017-10-06
• Primary Completion: 2019-01-31
• Study First Submitted: 2020-04-13
• Study First Submitted QC: 2020-04-28
• Study First Posted: 2020-04-29
• Status Verified: 2024-07
• Last Update Submitted: 2024-12-19
• Last Update Posted: 2024-12-24
• Study Completion: 2025-04

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 8

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
KHK4827	KHK4827	-

Primary Outcome Measures

Name	Time	Method
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

Name	Time	Method
Change in modified Rodnan skin score (mRSS) from baseline	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.	mRSS: scored 0(normal), 1(mild), 2(moderate), or 3(severe) per each site, assessed in 17 different body sites, total score=51

Trial Locations

Locations (1)

The University of Tokyo Hospital; 🇯🇵 Bunkyo-ku, Tokyo, Japan