Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)

NCT ID: NCT03364153

Last Updated: 2025-05-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

121 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-01-12

Study Completion Date

2025-03-31

Brief Summary

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The purpose of this study is to evaluate the safety and efficacy of avacincaptad pegol intravitreal injection compared to Sham in participants with autosomal recessive Stargardt disease 1 (STGD1).

Detailed Description

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Conditions

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Stargardt's Macular Dystrophy

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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avacincaptad pegol

Participants will receive avacincaptad pegol monthly for up to 17 Months.

Group Type EXPERIMENTAL

avacincaptad pegol

Intervention Type DRUG

Intravitreal Injection

Sham

Participants will receive a matching sham monthly for up to 17 Months.

Group Type SHAM_COMPARATOR

Sham

Intervention Type DRUG

Intravitreal Injection

Interventions

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avacincaptad pegol

Intravitreal Injection

Intervention Type DRUG

Sham

Intravitreal Injection

Intervention Type DRUG

Other Intervention Names

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Zimura (previous name) ARC1905 Izervay

Eligibility Criteria

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Inclusion Criteria

* At least two pathogenic mutations of ATP-Binding Cassette (ABC)A4 gene confirmed by a Clinical Laboratory Improvement Amendments (CLIA)-certified laboratory
* Best corrected visual acuity in the study eye between 20/20 - 20/200 Snellen equivalent, inclusive

Exclusion Criteria

* Macular atrophy secondary to any condition other than STGD1 in either eye
* Any prior treatment for STGD1 including gene therapy, stem cell therapy or any prior intravitreal treatment for any indication in either eye
* Participation in an interventional study of a vitamin A derivative \</= 3 months prior to screening
* Presence of intraocular inflammation, macular hole, pathologic myopia, epiretinal membrane, evidence of significant vitreo-macular traction, vitreous hemorrhage or aphakia
* Any intraocular surgery or thermal laser within 3 months of trial entry. Any prior thermal laser in the macular region
* Diabetes mellitus
* Hemoglobin A1c (HbA1c) value of \>/=6.5%
* Stroke within 12 months of trial entry
* Any major surgical procedure within one month of trial entry or anticipated during the trial
* Any treatment with an investigational agent in the past 60 days for any condition
* Women who are pregnant or nursing
* Known serious allergies to the fluorescein dye used in angiography, povidone iodine, or to the components of the avacincaptad pegol formulation
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Astellas Pharma Global Development, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_DIRECTOR

Astellas Pharma Global Development, Inc.

Locations

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Retinal Research Institute

Phoenix, Arizona, United States

Site Status

Jules Stein Eye Institute/ David Geffen School of Medicine

Los Angeles, California, United States

Site Status

VitreoRetinal Associates

Gainesville, Florida, United States

Site Status

Retina Specialty Institute

Pensacola, Florida, United States

Site Status

Wilmer Eye Institute, Johns Hopkins

Baltimore, Maryland, United States

Site Status

Ophthalmic Consultants of Boston

Boston, Massachusetts, United States

Site Status

University of Michigan/Kellogg Eye Center

Ann Arbor, Michigan, United States

Site Status

The Retina Center

Minneapolis, Minnesota, United States

Site Status

Retina Center of NJ, LLC.

Bloomfield, New Jersey, United States

Site Status

Casey Eye Institute/Oregon Health & Science University

Portland, Oregon, United States

Site Status

Wills Eye Hospital/Mid Atlantic Retina

Philadelphia, Pennsylvania, United States

Site Status

UPMC Eye Center

Pittsburgh, Pennsylvania, United States

Site Status

Palmetto Retina Center

West Columbia, South Carolina, United States

Site Status

Austin Retina Associates

Austin, Texas, United States

Site Status

Retina Foundation of the Southwest

Dallas, Texas, United States

Site Status

Strategic Clinical Research Group

Willow Park, Texas, United States

Site Status

University of Utah John A. Moran Eye Center

Salt Lake City, Utah, United States

Site Status

Hopital de la Croix-Rousse

Lyon, Auvergne-Rhône-Alpes, France

Site Status

Creteil University Eye Clinic University Paris EST

Créteil, , France

Site Status

Centre ophtalmologique des Quinzes Vingts

Paris, , France

Site Status

University of Bonn

Bonn, , Germany

Site Status

Augenklinik der LMU München

München, , Germany

Site Status

University of Tuebingen

Tübingen, , Germany

Site Status

Budapest Retina Institute

Budapest, , Hungary

Site Status

Semmelweis Egyetem

Budapest, , Hungary

Site Status

University of Debrecen DE KK Szemészeti Klinika

Debrecen, , Hungary

Site Status

Ganglion Medical Center

Pécs, , Hungary

Site Status

Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika

Szeged, , Hungary

Site Status

Rambam Health Care Campus

Haifa, , Israel

Site Status

Hadassah University Hospital

Jerusalem, , Israel

Site Status

Rabin Medical Center, Beilinson campus

Petah Tikva, , Israel

Site Status

Kaplan Medical Center

Rehovot, , Israel

Site Status

Tel-Aviv Sourasky Medical Center, Ichilov Hospital

Tel Aviv, , Israel

Site Status

AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,

Bologna, , Italy

Site Status

Azienda Ospedaliera Universitaria Careggi

Florence, , Italy

Site Status

Ospedale San Raffaele

Milan, , Italy

Site Status

University of Campania Luigi Vanvitelli Eye Clinic

Naples, , Italy

Site Status

Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche

Rome, , Italy

Site Status

Institut de la Macula

Barcelona, , Spain

Site Status

Princess Alexandra Eye Pavillion

Edinburgh, , United Kingdom

Site Status

Moorfields Eye Hospital

London, , United Kingdom

Site Status

Countries

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United States France Germany Hungary Israel Italy Spain United Kingdom

Other Identifiers

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2017-004783-35

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

OPH2005

Identifier Type: -

Identifier Source: org_study_id

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