A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of IVT MHU650 in Macular Edema Patients

Novartis

Status and phase: Completed; Phase 1

Conditions: Diabetic Macular Edema; Macular Edema; Neovascular Age-related Macular Degeneration; Retinal Vein Occlusion

Treatments: Drug: MHU650

Study type: Interventional

Funder types: Industry

Identifiers

NCT04635800

CMHU650A12101

Details and patient eligibility

About

This was an open-label, multi-center, FIH study with a single ascending dose (SAD) design that assessed the safety, tolerability and pharmacokinetics (PK) of a single IVT dose of MHU650 in up to 24 participants with macular edema.

Full description

A total of up to 4 cohorts were planned to be enrolled, with an additional lower or intermediate cohort of participants. First to third generation Japanese participants could be enrolled in all cohorts but were not required to be enrolled. The screening period for this study will be up to 60 days. A single dose of MHU650 intravitreal injection will be administered at Baseline / Day 1 in the study eye. The follow up period will last until Day 60 / end of study (EOS). A post study safety phone contact call will occur 30 days after the EOS visit.

Enrollment: 21 patients

Sex: All

Ages: 18 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Patients with macular edema in at least one eye, including those with focal or diffuse diabetic macular edema (DME), neovascular age-related macular degeneration (nAMD), or retinal vein occlusion (RVO). In the opinion of the investigator, the decrease in vision in the study eye must be due to macular edema.
• Early Treatment Diabetic Retinopathy (ETDRS) letter score in the study eye must equal to or worse than 60 letters (approximately Snellen equivalent of 20/63) but better than 14 letters (20/500) at screening and baseline. The ETDRS score in the non-study eye should be ≥ 60 letters at screening and baseline.
• Sufficiently clear ocular media and adequate pupil dilation to permit fundus photographs of adequate clarity to measure diameters of retinal arteries and veins
• Vital signs as specified in the protocol

Key Exclusion Criteria:

• Concomitant conditions or ocular disorders in the study eye which may, in the opinion of the investigator, confound the interpretation of study results, compromise visual acuity or require medical or surgical intervention during the study period
• High risk and/or/ active proliferative diabetic retinopathy in the study eye, as per investigator assessment at both screening and baseline.
• Participants with the following conditions in the study eye at screening or baseline must be excluded: structural damage of the fovea, vitreous hemorrhage, retinal detachment, vitreomacular traction, macular hole, retinal arterial occlusion, neovascularization of iris of any cause.
• Laser photocoagulation (macular or panretinal) in the study eye during the 6-month period prior to baseline.
• Patients with type 1or type 2 diabetes who have hemoglobin A1C of ≥ 12 at screening
• Other ocular conditions as specified in the protocol
• Systemic conditions as specified in the protocol

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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21 participants in 4 patient groups

Cohort 1

Experimental group

Description:

Cohort 1; open-label, non-randomized, single administration

Treatment:

Drug: MHU650

Cohort 2

Experimental group

Description:

Cohort 2; open-label, non-randomized, single administration

Treatment:

Drug: MHU650

Cohort 3

Experimental group

Description:

Cohort 3, open-label; non-randomized, single administration

Treatment:

Drug: MHU650

Cohort 4

Experimental group

Description:

Cohort 4, open-label, non-randomized, single administration

Treatment:

Drug: MHU650