NCT04635800

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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Dec 2020

Geographic Reach
2 countries

6 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 13, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 19, 2020

Completed
21 days until next milestone

Study Start

First participant enrolled

December 10, 2020

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 24, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 24, 2022

Completed
Last Updated

May 18, 2025

Status Verified

May 1, 2025

Enrollment Period

1.5 years

First QC Date

November 13, 2020

Last Update Submit

May 14, 2025

Conditions

Macular EdemaDiabetic Macular EdemaNeovascular Age-related Macular DegenerationRetinal Vein Occlusion

Keywords

Macular degenerationage-related macular degenerationdry macular degenerationwet macular degenerationDiabetic Macular EdemaDMENeovascular age-related macular edemanAMDRetinal Vein OcclusionRVOMHU650macular edema

Outcome Measures

Primary Outcomes (4)

  • Number of participants with ocular and nonocular adverse events

    An adverse event (AE) is any untoward medical occurrence (e.g. any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study.

    Day 1 to Day 60

  • Change from baseline in Best Correct Visual Acuity (BCVA ) by dose level and timepoint - Study Eye

    Best Correct Visual Acuity was assessed using Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity testing charts.

    Day 1 to Day 60

  • Change from baseline in Central subfield thickness (CSFT) (µm) in the study eye by dose level and timepoint

    Central subfield thickness was measured by spectral domain optical coherence tomography (SD-OCT).

    Day 1 to Day 60

  • Change from baseline in Intraocular pressure (IOP) (mmHg) in the study eye by dose level and timepoint

    Intraocular pressure was measured per the sites' local standard operating procedures, via tonometry.

    Day 1 to Day 60

Secondary Outcomes (5)

  • Pharmacokinetics of single dose of MHU650 - Cmax

    Days 1, 2, 5, 15, 29, 43 and 60

  • Pharmacokinetics of single dose of MHU650 - Tmax

    Days 1, 2, 5, 15, 29, 43 and 60

  • Pharmacokinetics of single dose of MHU650 - T1/2

    Days 1, 2, 5, 15, 29, 43 and 60

  • Pharmacokinetics of single dose of MHU650 - AUClast

    Days 1, 2, 5, 15, 29, 43 and 60

  • Pharmacokinetics of single dose of MHU650 - AUCinf

    Days 1, 2, 5, 15, 29, 43 and 60

Study Arms (4)

Cohort 1

EXPERIMENTAL

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

Drug: MHU650

Cohort 2

EXPERIMENTAL

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

Drug: MHU650

Cohort 3

EXPERIMENTAL

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

Drug: MHU650

Cohort 4

EXPERIMENTAL

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

Drug: MHU650

Interventions

MHU650DRUG

MHU650 powder for solution for injection

Cohort 1Cohort 2Cohort 3Cohort 4

Eligibility Criteria

Age18 Years - 90 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (6)

Novartis Investigative Site

Huntington Beach, California, 92647, United States

Location

Novartis Investigative Site

Honolulu, Hawaii, 96813, United States

Location

Novartis Investigative Site

Hagerstown, Maryland, 21740, United States

Location

Novartis Investigative Site

Austin, Texas, 78793, United States

Location

Novartis Investigative Site

Houston, Texas, 77030, United States

Location

Novartis Investigative Site

Arecibo, 00612, Puerto Rico

Location

Related Links

MeSH Terms

Conditions

Macular EdemaRetinal Vein OcclusionMacular DegenerationGeographic AtrophyWet Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal DegenerationRetinal DiseasesEye DiseasesVenous ThrombosisThrombosisEmbolism and ThrombosisVascular DiseasesCardiovascular Diseases

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Masking Details
Open-label; no masking
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Model Details: The primary objective of this open-label study is to evaluate the initial safety, tolerability, and pharmacokinetics (PK) of single IVT doses of MHU650 in up to 24 patients. The screening period will be up to 60 days. A single dose of MHU650 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. A total of up to 4 cohorts may be enrolled, with an additional optional lower or intermediate cohort of 6 participants.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 13, 2020

First Posted

November 19, 2020

Study Start

December 10, 2020

Primary Completion

May 24, 2022

Study Completion

May 24, 2022

Last Updated

May 18, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations

US(5)PR(1)