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

A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt Disease


Lead Sponsor: IVERIC bio, Inc.

Source IVERIC bio, Inc.
Brief Summary

To evaluate the safety and efficacy of Zimura™ (complement factor C5 inhibitor) compared to Sham in subjects with autosomal recessive Stargardt disease 1 (STGD1).

Overall Status Recruiting
Start Date 2018-01-12
Completion Date 2024-09-01
Primary Completion Date 2024-09-01
Phase Phase 2
Study Type Interventional
Primary Outcome
Measure Time Frame
Spectral Domain-Optical Coherence Tomography (SD-OCT) 18 months
Enrollment 120

Intervention Type: Drug

Intervention Name: Zimura

Description: Zimura Intravitreal Injection

Arm Group Label: Cohort 1

Other Name: avacincaptad pegol

Intervention Type: Other

Intervention Name: Sham

Description: Sham Intravitreal Injection

Arm Group Label: Cohort 2



Inclusion Criteria: - At least two pathogenic mutations of ABCA4 gene confirmed by a 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 - 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 Zimura formulation



Minimum Age:

18 Years

Maximum Age:

60 Years

Healthy Volunteers:


Overall Contact

Last Name: Sonia Cruz

Phone: 347-322-8722

Email: [email protected]

Facility: Status: Contact:
Retinal Consultants of Arizona | Phoenix, Arizona, 85053, United States Recruiting 602-222-2221
Jules Stein Eye Institute/ David Geffen School of Medicine | Los Angeles, California, 90095, United States Recruiting 310-267-5278
VitreoRetinal Associates | Gainesville, Florida, 32607, United States Recruiting 352-300-8412
Retinal Specialty Institute | Pensacola, Florida, 32503, United States Recruiting
Wilmer Eye Institute, Johns Hopkins | Baltimore, Maryland, 21287, United States Completed
Ophthalmic Consultants of Boston | Boston, Massachusetts, 02114, United States Completed
Kellogg Eye Center | Ann Arbor, Michigan, 48105, United States Recruiting 734-936-9798
Retina Center P.A. | Minneapolis, Minnesota, 55404, United States Recruiting 612-747-5644
Envision Ocular, LLC | Bloomfield, New Jersey, 07003, United States Recruiting 973-707-5632
Oregon Health & Science University | Portland, Oregon, 97239, United States Recruiting 503-494-3115
Mid Atlantic Retina | Philadelphia, Pennsylvania, 19107, United States Recruiting 215-928-3092
UPMC Eye Center | Pittsburgh, Pennsylvania, 15213, United States Completed
Palmetto Retina Center | West Columbia, South Carolina, 29169, United States Recruiting 803-404-5038
Austin Retina Associates | Austin, Texas, 78705, United States Recruiting 512-451-0103
Retina Foundation of the Southwest | Dallas, Texas, 75231, United States Recruiting 214-363-3911
Strategic Clinical Research Group | Willow Park, Texas, 76087, United States Recruiting 682-703-4324
University of Utah John A. Moran Eye Center | Salt Lake City, Utah, 84132, United States Recruiting 801-581-6265
Montreal Children's Hospital | Montréal, H4A 3J1, Canada Withdrawn
Hopital de la Croix-Rousse | Lyon, Rhone-Alpes, 69317, France Completed
Hopital Intercommunal de Verdun, University Paris Est | Créteil, 94000, France Completed
Centre ophtalmologique des Quinzes Vingts | Paris, 75012, France Completed
University of Bonn | Bonn, 53127, Germany Completed
Augenklinik und Poliklinik des Klinikums der Universitat München | München, 80336, Germany Completed
Universitätsklinikum Tübingen | Tübingen, 72076, Germany Completed
Budapest Retina Associates | Budapest, 1133, Hungary Completed
Semmelweis University, Ophthalmology Clinics | Budapest, H-1085, Hungary Completed
Debreceni Egyetem Klinikai Kozpont Szemklinika | Debrecen, 4032, Hungary Completed
Ganglion Medical Center | Pécs, 7621, Hungary Completed
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika | Szeged, 6720, Hungary Completed
Rambam Health Care Campus | Haifa, 310960, Israel Completed
Hadassah Medical Center | Jerusalem, 91120, Israel Completed
Rabin Medical Center, Beilinson campus | Petah tikva, 4941492, Israel Completed
Kaplan Medical Center | Reẖovot, 7610001, Israel Completed
Tel-Aviv Sourasky Medical Center, Ichilov Hospital | Tel Aviv, 64239, Israel Completed
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia, | Bologna, 40138, Italy Completed
AOU Careggi | Florence, 50134, Italy Completed
IRCCS Ospedale San Raffaele | Milano, 20132, Italy Completed
AOU Universita degli studi della Campania Luigi Vanvitelli - UOC Oculistica | Naples, 80131, Italy Completed
Foundation Policlinico Tor Vergata, UOSD Patologie Retiniche | Rome, 00133, Italy Completed
Institut de la Macula | Barcelona, 08022, Spain Completed
Princess Alexandra Eye Pavillion | Edinburgh, EH3 9HA, United Kingdom Completed
Moorfields Eye Hospital | London, EC1V 2PD, United Kingdom Completed
Location Countries








United Kingdom

United States

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Cohort 1

Type: Experimental

Description: Zimura dose group

Label: Cohort 2

Type: Sham Comparator

Description: Sham dose group

Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Triple (Participant, Investigator, Outcomes Assessor)

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