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NCT Number: NCT03349801

Development of Novel Clinical Endpoints in Intermediate AMD

Development of novel clinical endpoints for interventional clinical trials with a regulatory and patient access intention in patients with intermediate age-related macular degeneration (AMD) - MACUSTAR

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Age Related Macular Degeneration (AMD) Eye Diseases Macular Degeneration Retinal Degeneration Retinal Diseases

Age range

55 year–85 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Righospitalet Copenhagen, Copenhagen, Denmark

Loading trial locations.

About this study

The purpose of the MACUSTAR clinical study is to develop novel clinical endpoints for clinical trials with a regulatory and patient access intention in patients with intermediate age-related macular degeneration (iAMD). Additional objectives are to characterize the visual impairment in iAMD and its progression, as well as identify risk factors for progression to late stage AMD.

Moreover, MACUSTAR aims to optimize and standardize most relevant existing and/or rapidly available clinical endpoints in:

  • visual functional outcomes measures
  • structural outcomes measures
  • patient reported outcomes measures (PROMs)

The study will be composed by two parts:

  • a cross-sectional part to technically evaluate the functional and structural outcome measures to support a biomarker qualification by regulatory authorities and payers; and
  • a longitudinal part to assess the prognostic power of changes in retinal sensitivity (as measured by microperimetry) for progression from iAMD to late AMD (nAMD and GA).

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

General Inclusion criteria (applicable to all groups)

  • Male and female subjects.
  • Aged 55 - 85 years at baseline.
  • Able and willing to provide written informed consent and to comply with the study protocol visits and assessments.

Intermediate AMD

  • Study eye must have iAMD and,
  • The fellow eye must have iAMD and/or, in addition, extrafoveal GA (no atrophy within the central ETDRS subfield), maximum total GA size is 1.25 mm2.
  • ETDRS letter chart BCVA in the study eye not worse than 72 letters (approximately 20/40 Snellen VA equivalent).
  • All general inclusion criteria.

Late AMD

  • Subjects with bilateral GA, bilateral nAMD or nAMD in one eye and GA in the other.
  • BCVA between 20/80 and 20/200 in study eye.
  • All general inclusion criteria.

Early AMD

  • Subjects with medium drusen > 63μm and ≤ 125μm and no AMD pigmentary abnormalities in both eyes and not signs of intermediate or late AMD.
  • All general inclusion criteria.

No AMD

  • No signs of early, intermediate or late AMD in both eyes.
  • All general inclusion criteria only.

Exclusion criteria

General Exclusion criteria (applicable to all groups)

  • Media opacity or eye movement disorder (nystagmus) that interferes with retinal imaging data quality in the opinion of the investigator.
  • Severe ptosis, extraocular motility restriction or head tremor preventing adequate fundus visualization in the opinion of the investigator.
  • Any signs of nAMD or GA (does not apply to the late AMD group).
  • Any concurrent intraocular condition in the study eye (e. g. glaucoma or cataract) that, in the opinion of the investigator would either require surgical intervention during the study to prevent or treat visual loss that might result from that condition or affect interpretation of study results.
  • Severe non-proliferative diabetic retinopathy, or proliferative diabetic retinopathy.
  • Any diabetic macular edema or macular disease
  • Ocular disorders in the study eye (i. e., pre-retinal membrane) at the time of enrolment that may confound interpretation of study results and compromise visual acuity.
  • Diagnosis of uncontrolled glaucoma with intraocular pressure of >30 mmHg (despite current pharmacological or non-pharmacological treatment).
  • Known systemic illness which in the opinion of the investigator will prevent from actively participating in the study.
  • Concomitant treatment for AMD in either eye (concomitant use of vitamins/supplements is not excluded; does not apply to the late AMD group).
  • Any periocular or intravitreal injections (IVT) in either eye (does not apply to the late AMD group).
  • Participation in any other interventional trial.
  • Obvious retinal changes due to causes other than AMD (e.g. evidenced by an existing diagnosis of monogenetic macular dystrophies, Stargardt disease, cone rod dystrophy, or toxic maculopathies).
  • Any history of allergies to fluorescein.

Intermediate AMD

  • Any GA in the study eye
  • Any extrafoveal GA larger than 1.25 mm2 in the fellow eye.
  • All general exclusion criteria.

Late AMD

  • All general exclusion criteria only.

Early AMD

  • Intermediate or late AMD (following Beckman classification) in any eye.
  • All general exclusion criteria.

No AMD

  • Early to late AMD (following Beckman classification) in any eye.
  • All general exclusion criteria.

Treatment and study plan

No intervention

Other

According to clinical practice.

Primary outcomes

  1. Mean change from baseline in best corrected visual acuity (BCVA) using an early treatment diabetic retinopathy study (ETDRS) chart

    Time frame: 3 years from baseline

    standard parameter, tested for comparison (reference variable)

  2. Mean change from baseline in scotopic and mesopic microperimetry sensitivity

    Time frame: 3 years from baseline

  3. Mean change from baseline in low luminance visual acuity (LLVA)

    Time frame: 3 years from baseline

  4. Mean change from baseline in vanishing optotypes visual acuity (VA)

    Time frame: 3 years from baseline

  5. Mean change from baseline in low luminance deficit (LLD)

    Time frame: 3 years from baseline

    LLD = BCVA-LLVA

  6. Mean change from baseline in absolute rod threshold of the dark adaptation test

    Time frame: 3 years from baseline

  7. Mean change from baseline in rod intercept time of the dark adaptation test

    Time frame: 3 years from baseline

  8. Proportion of subjects with progression in dark adaptation deficit beyond coefficient of repeatability structural

    Time frame: 3 years from baseline

  9. Mean change from baseline in the cube root of drusen volume by spectral-domain optical coherence tomography (SD-OCT)

    Time frame: 3 years from baseline

  10. Mean change from baseline in retinal thickness by spectral-domain optical coherence tomography (SD-OCT)

    Time frame: 3 years from baseline

  11. Focal pigmentary changes captured by colour fundus photography (CFP)

    Time frame: 3 years from baseline

  12. Presence of refractile deposits

    Time frame: 3 years from baseline

  13. Presence of intraretinal cystoid spaces

    Time frame: 3 years from baseline

  14. Presence of localized retinal pigment epithelium (RPE) hypertransmission

    Time frame: 3 years from baseline

  15. Presence of localized disruption of ellipsoid zone

    Time frame: 3 years from baseline

  16. Presence of localized subsidence of the outer plexiform layer and the inner nuclear layer

    Time frame: 3 years from baseline

  17. Presence of hyporeflective wedge-shaped bands

    Time frame: 3 years from baseline

  18. Presence of reticular drusen/subretinal drusenoid deposits and associated local changes as determined by multimodal imaging

    Time frame: 3 years from baseline

  19. Changes in localized fundus autofluorescence signal alterations

    Time frame: 3 years from baseline

  20. Proportion of subjects with reduction in drusen volume

    Time frame: 3 years from baseline

  21. Proportion of subjects with study eye that progressed to geogrphic atrophy (GA) and/or neovascular age-related macular degeneration (nAMD)

    Time frame: 3 years from baseline

  22. Proportion of subjects with conversions to late AMD detected with fluorescein angiography (FA) that could be detected with OCT-A (at equipped sites)

    Time frame: 3 years from baseline

  23. Presence of quiescent choroidal neovascularisation (CNV) as assessed by optical coherence tomography angiography (OCT-A) (at equipped sites)

    Time frame: 3 years from baseline

  24. OCT-A findings (at equipped sites)

    Time frame: 3 years from baseline

  25. Mean change from baseline in patient-reported low luminance visual functioning, as measured by the vision impairment in low luminance (VILL) questionnaire, including the domains of reading & accessing information

    Time frame: 3 years from baseline

  26. Mean change from baseline in patient-reported low luminance visual functioning, as measured by the VILL questionnaire, including the domains of Orientation & mobility (incl. driving)

    Time frame: 3 years from baseline

  27. Mean change from baseline in patient-reported low luminance visual functioning, as measured by the VILL questionnaire, including the domains of safety

    Time frame: 3 years from baseline

  28. Mean change from baseline in patient-reported low luminance visual functioning, as measured by the VILL questionnaire, including the domains of socio-emotional well-being

    Time frame: 3 years from baseline

  29. Change in utility index from baseline as measure by the patient-reported outcome measure (PROM) utility index

    Time frame: 3 years from baseline

  30. Mean change from baseline in patient-reported health status and utility using the EQ-5D-5L questionnaire (EuroQol Group)

    Time frame: 3 years from baseline

Sponsors and collaborators

Lead sponsor

Frank G. Holz

Other

Collaborators

  • Association for Innovation and Biomedical Research on Light and Image
  • Bayer
  • Carl Zeiss Meditec AG
  • City, University of London
  • European Clinical Research Infrastructure Network
  • Hoffmann-La Roche
  • Innovative Medicines Initiative
  • La Fondation Voir et Entendre
  • Moorfields Eye Hospital NHS Foundation Trust
  • Novartis Pharmaceuticals
  • Radboud University Medical Center
  • University College, London
  • University of Sheffield

Registry information

Official study title

Development of Novel Clinical Endpoints for Interventional Clinical Trials With a Regulatory and Patient Access Intention in Patients With Intermediate Age-related Macular Degeneration (AMD)

Acronym: MACUSTAR

Important dates

Study start
2018
Primary completion
2026
Study completion
2026
First posted
Nov 22, 2017
Registry last updated
Jan 16, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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