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

NCT ID: NCT04635800

Last Updated: 2025-05-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-10
• Study Completion Date: 2022-05-24

Brief Summary

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.

Detailed 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.

Conditions

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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Cohort 1

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

Group Type: EXPERIMENTAL

MHU650

Intervention Type: DRUG

MHU650 powder for solution for injection

Cohort 2

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

Group Type: EXPERIMENTAL

MHU650

Intervention Type: DRUG

MHU650 powder for solution for injection

Cohort 3

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

Group Type: EXPERIMENTAL

MHU650

Intervention Type: DRUG

MHU650 powder for solution for injection

Cohort 4

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

Group Type: EXPERIMENTAL

MHU650

Intervention Type: DRUG

MHU650 powder for solution for injection

Interventions

MHU650

MHU650 powder for solution for injection

Intervention Type: DRUG

Eligibility Criteria

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

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 90 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Huntington Beach, California, United States

Novartis Investigative Site

Honolulu, Hawaii, United States

Novartis Investigative Site

Hagerstown, Maryland, United States

Novartis Investigative Site

Austin, Texas, United States

Novartis Investigative Site

Houston, Texas, United States

Novartis Investigative Site

Arecibo, , Puerto Rico

Countries

United States; Puerto Rico

Related Links

https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=18049

Results for CMHU650A12101 from the Novartis Trials Website

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=1501

A Plain Language Trial Summary is available on www.novctrd.com

Other Identifiers

CMHU650A12101

Identifier Type: -

Identifier Source: org_study_id