A Safety Study of KHK4951 in Healthy Volunteers and Patients With Wet Age-Related Macular Degeneration

A Phase I Study of KHK4951 in Healthy Volunteers and Patients With Wet Age-Related Macular Degeneration

The purpose of this study is to evaluate safety and tolerability after administration of KHK4951 in healthy volunteers and patients with wet age-related macular degeneration.

Study Overview

• Status: Terminated
• Conditions: Healthy Volunteers; Wet Age-related Macular Degeneration
• Intervention / Treatment: Drug: Placebo; Drug: KHK4951

• Study Type: Interventional
• Enrollment (Actual): 116
• Phase: Phase 1

Contacts and Locations

Study Locations

• Japan
  • Fukuoka, Japan
    • Hakata Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion Criteria: Healthy Volunteers

• Voluntary written informed consent to participate in the study
• Japanese or white men 20 to < 50 years at the time of informed consent
• BMI 18.5 to < 30.0 at screening
• Intraocular pressure (study eye) 10.0 mm Hg to 21.0 mm Hg
• Monocular or binocular visual acuity (corrected visual acuity if correction is required) ≥ 1.0 at screening

Inclusion Criteria: Patients

• Voluntary written informed consent to participate in the study
• Age ≥ 50 years at the time of informed consent
• AMD-associated by active subfoveal CNV lesions in the study eye or juxtafoveal CNV lesions with CNV-associated findings in the fovea in the study eye
• CST ≥ 300 μm in the study eye at screening, as measured by OCT
• BCVA score ≥ 23 letters in the study eye at screening and enrollment, as measured by the ETDRS visual acuity chart
• BCVA score ≥ 58 letters in the non-study eye at screening, as measured by the ETDRS visual acuity chart
• In subjects with prior treatment of the study eye with anti-vascular endothelial growth factor (anti-VEGF) drugs such as Beovu, Eylea, Lucentis, Avastin, or Macugen, presence of response to the anti-VEGF drug(s) as judged by the investigator or subinvestigator based OCT, FA, visual acuity, or other assessment results

Exclusion Criteria: Healthy Volunteers

• Current illness requiring treatment
• History of ophthalmologic laser surgery, ophthalmologic surgery, nasolacrimal duct surgery, orany eyelid surgery affecting flow in the nasolacrimal duct
• History of or current circulatory disease (e.g., cerebrovascular or cardiovascular disease)
• History of or current dry eye
• Abnormal findings on OCT at screening or enrollment examination

Exclusion Criteria: Patients

• Clinical findings or a history of conditions other than wAMD affecting the retina and choroid (e.g., diabetic retinopathy, diabetic macular edema, myopic CNV, retinal vein occlusion, or premacular membrane) in either of the eyes

• Any of the following diseases in the study eye:

  • Glaucoma
  • Ischemic optic neuropathy
  • Retinitis pigmentosa
• Current or history of vitreous hemorrhage or macular hole in the study eye
• Any abnormality in the anterior segment of the eye or vitreous body that may affect fundus observation by OCT, color fundus photography, or fluorescein angiography

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo is administered once daily or three times daily.
Experimental: KHK4951	Drug: KHK4951; KHK4951 is administered once daily or three times daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with adverse events	For adverse events that occurred after administration of the investigational drug, number of subjects with AEs and occurrence frequency are evaluated.	Day 1 to Day 43

Secondary Outcome Measures

Outcome Measure	Time Frame
Serum KHK4951 concentration	Healthy Volunteers: Day 1, Day2, Day4, Day8, Day12, Day16, Day21, Day23, Day 25, Day 29, Day 36, Day 43; Patients: Day 1, 8, 22, 43
Time to the maximum concentration	Day 1 to Day 43
The maximum concentration	Day 1 to Day 43
Area under the concentration-time curve	Day 1 to Day 43
Apparent clearance	Day 1 to Day 43
Elimination half-life	Day 1 to Day 43

Collaborators and Investigators

• Sponsor: Kyowa Kirin Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): September 16, 2020
• Primary Completion (Actual): August 26, 2022
• Study Completion (Actual): August 26, 2022

Study Registration Dates

• First Submitted: September 22, 2020
• First Submitted That Met QC Criteria: October 14, 2020
• First Posted (Actual): October 20, 2020

Study Record Updates

• Last Update Posted (Actual): May 18, 2023
• Last Update Submitted That Met QC Criteria: May 16, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration
• Other Study ID Numbers: 4951-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No