Clinical trial NCT04594681: A Safety Study of KHK4951 in Healthy Volunteers and Patients With Wet Age-Related Macular Degeneration
Official title: A Phase I Study of KHK4951 in Healthy Volunteers and Patients With Wet Age-Related Macular Degeneration
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
Conditions: Healthy Volunteers
Wet Age-related Macular Degeneration
Lead sponsor: Kyowa Kirin Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2020-10-20
Enrollment: 116
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with adverse events
timeFrame: Day 1 to Day 43
Secondary outcomes:
measure: Serum KHK4951 concentration
timeFrame: 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
measure: Time to the maximum concentration
timeFrame: Day 1 to Day 43
measure: The maximum concentration
timeFrame: Day 1 to Day 43
measure: Area under the concentration-time curve
timeFrame: Day 1 to Day 43
measure: Apparent clearance
timeFrame: Day 1 to Day 43
measure: Elimination half-life
timeFrame: Day 1 to Day 43
Other outcomes:
Locations:
city: Fukuoka
country: Japan
facility: Hakata clinic
Study officials:
References: