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

NCT04594681 | Terminated Phase 1

• 1 other identifier: 4951-001
• Study Type: interventional
• Target: 116
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Healthy Volunteers; Wet Age-related Macular Degeneration

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (6)

• 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

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Drug: Placebo

KHK4951

EXPERIMENTAL

Drug: KHK4951

Interventions

Placebo DRUG

Placebo is administered once daily or three times daily.

Placebo

KHK4951 DRUG

KHK4951 is administered once daily or three times daily.

KHK4951

Eligibility Criteria

• Age: 20 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Kyowa Kirin Co., Ltd.: lead

Study Sites (1)

Hakata clinic

Fukuoka, Japan

Location

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2020
• First Posted: October 20, 2020
• Study Start: September 16, 2020
• Primary Completion: August 26, 2022
• Study Completion: August 26, 2022
• Last Updated: May 18, 2023

Record last verified: 2023-05

Locations

JP (1)