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Macular EdemaPhase 1NCT04635800

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

A Phase 1 study testing MHU650 in people with Macular Edema and Diabetic Macular Edema, run by Novartis Pharmaceuticals at 6 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 90 years
What is being tested?
MHU650
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
21 participants
Where
6 sitesin 2 countries
Study dates
10 Dec 2020to 24 May 2022
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 90 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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 locations and contacts

6 sites in 2 countries.

6 sites in 2 countries

United States5 sites

  • Novartis Investigative Site

    Austin, Texas, 78793

  • Novartis Investigative Site

    Hagerstown, Maryland, 21740

  • Novartis Investigative Site

    Honolulu, Hawaii, 96813

  • Novartis Investigative Site

    Houston, Texas, 77030

  • Novartis Investigative Site

    Huntington Beach, California, 92647

Other trials in United States

Puerto Rico1 site

  • Novartis Investigative Site

    Arecibo, 00612

Other trials in Puerto Rico

About this study

Official title: A First-in-human, Open-label, Single Ascending Dose Study to Assess Safety and Tolerability of Intravitreal MHU650 in Participants With Macular Edema From Diabetic Macular Edema (DME), Neovascular Age-related Macular Degeneration (nAMD), or Retinal Vein Occlusion (RVO)

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.

Read the 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.

Being tested
MHU650
Keywords
Macular degeneration, age-related macular degeneration, dry macular degeneration, wet macular degeneration, Diabetic Macular Edema, DME, Neovascular age-related macular edema, nAMD, Retinal Vein Occlusion, RVO, MHU650, macular edema
Medical subject headings
Macular Edema, Retinal Vein Occlusion, Macular Degeneration, Geographic Atrophy, Wet Macular Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MHU650Drug

    MHU650 powder for solution for injection

Study groups in NCT04635800
Study groupRoleWhat they receive
Cohort 1ExperimentalCohort 1; open-label, non-randomized, single administration
Receives: Drug: MHU650
Cohort 2ExperimentalCohort 2; open-label, non-randomized, single administration
Receives: Drug: MHU650
Cohort 3ExperimentalCohort 3, open-label; non-randomized, single administration
Receives: Drug: MHU650
Cohort 4ExperimentalCohort 4, open-label, non-randomized, single administration
Receives: Drug: MHU650

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Day 1 to Day 60

  2. 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.

    Measured: Day 1 to Day 60

  3. 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).

    Measured: Day 1 to Day 60

  4. 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.

    Measured: Day 1 to Day 60

Secondary outcomes (5)

  1. Pharmacokinetics of single dose of MHU650 - Cmax

    Assess serum PK profile of MHU650 by Cmax (if feasibile)

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

  2. Pharmacokinetics of single dose of MHU650 - Tmax

    Assess serum PK profile of MHU650 by Tmax (if feasibile)

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

  3. Pharmacokinetics of single dose of MHU650 - T1/2

    Assess serum PK profile of MHU650 by T1/2 (if feasibile)

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

  4. Pharmacokinetics of single dose of MHU650 - AUClast

    Assess serum PK profile of MHU650 by AUClast (if feasibile)

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

  5. Pharmacokinetics of single dose of MHU650 - AUCinf

    Assess serum PK profile of MHU650 by AUCinf (if feasibile)

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Nov 2020
  2. Study start10 Dec 2020
  3. Primary completion24 May 2022
  4. Study completion24 May 2022

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Model description
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.
Masking
None (open label)
Enrolment
21 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04635800
Sponsor's protocol ID
CMHU650A12101
Status last verified
May 2025
First submitted
13 Nov 2020
Last updated
18 May 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing MHU650 in people with Macular Edema and Diabetic Macular Edema, run by Novartis Pharmaceuticals at 6 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 18 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States, Puerto Rico. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with ocular and nonocular adverse events (time frame: Day 1 to Day 60). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04635800.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 May 2025.

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