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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03364153
Recruitment Status : Active, not recruiting
First Posted : December 6, 2017
Last Update Posted : March 4, 2019
Sponsor:
Information provided by (Responsible Party):
Ophthotech Corporation

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).

Condition or disease Intervention/treatment Phase
Stargardt Disease 1 Drug: Zimura Other: Sham Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 95 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : January 12, 2018
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2020


Arm Intervention/treatment
Experimental: Cohort 1
Zimura dose group
Drug: Zimura
Zimura Intravitreal Injection
Other Name: avacincaptad pegol

Sham Comparator: Cohort 2
Sham dose group
Other: Sham
Sham Intravitreal Injection




Primary Outcome Measures :
  1. Spectral Domain-Optical Coherence Tomography (SD-OCT) [ Time Frame: 18 months ]
    Mean rate of change in the area of ellipsoid zone defect measured by en face SD-OCT



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Ages Eligible for Study:   18 Years to 50 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03364153


  Hide Study Locations
Locations
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United States, Arizona
Retinal Consultants of Arizona
Phoenix, Arizona, United States, 85053
United States, California
Jules Stein Eye Institute/ David Geffen School of Medicine
Los Angeles, California, United States, 90095
United States, Florida
VitreoRetinal Associates
Gainesville, Florida, United States, 32607
United States, Maryland
Wilmer Eye Institute, Johns Hopkins
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Ophthalmic Consultants of Boston
Boston, Massachusetts, United States, 02114
United States, Michigan
Kellogg Eye Center
Ann Arbor, Michigan, United States, 48105
United States, Minnesota
Retina Center P.A.
Minneapolis, Minnesota, United States, 55404
United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Mid Atlantic Retina
Philadelphia, Pennsylvania, United States, 19107
UPMC Eye Center
Pittsburgh, Pennsylvania, United States, 15213
United States, South Carolina
Palmetto Retina Center
West Columbia, South Carolina, United States, 29169
United States, Texas
Austin Retina Associates
Austin, Texas, United States, 78705
Retina Foundation of the Southwest
Dallas, Texas, United States, 75231
Strategic Clinical Research Group
Willow Park, Texas, United States, 76087
United States, Utah
University of Utah John A. Moran Eye Center
Salt Lake City, Utah, United States, 84132
France
Hopital de la Croix-Rousse
Lyon, Rhone-Alpes, France, 69317
Hopital Intercommunal de Verdun, University Paris Est
Créteil, France, 94000
Centre ophtalmologique des Quinzes Vingts
Paris, France, 75012
Germany
University of Bonn
Bonn, Germany, 53127
Augenklinik und Poliklinik des Klinikums der Universitat München
München, Germany, 80336
Universitätsklinikum Tübingen
Tübingen, Germany, 72076
Hungary
Budapest Retina Associates
Budapest, Hungary, 1133
Semmelweis University, Ophthalmology Clinics
Budapest, Hungary, H-1085
Debreceni Egyetem Klinikai Kozpont Szemklinika
Debrecen, Hungary, 4032
Ganglion Medical Center
Pécs, Hungary, 7621
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
Szeged, Hungary, 6720
Israel
Rambam Health Care Campus
Haifa, Israel, 310960
Hadassah Medical Center
Jerusalem, Israel, 91120
Rabin Medical Center, Beilinson campus
Petah tikva, Israel, 4941492
Kaplan Medical Center
Reẖovot, Israel, 7610001
Tel-Aviv Sourasky Medical Center, Ichilov Hospital
Tel Aviv, Israel, 64239
Italy
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
Bologna, Italy, 40138
AOU Careggi
Florence, Italy, 50134
IRCCS Ospedale San Raffaele
Milano, Italy, 20132
AOU Universita degli studi della Campania Luigi Vanvitelli - UOC Oculistica
Naples, Italy, 80131
Foundation Policlinico Tor Vergata, UOSD Patologie Retiniche
Rome, Italy, 00133
Spain
Institut de la Macula
Barcelona, Spain, 08022
United Kingdom
Princess Alexandra Eye Pavillion
Edinburgh, United Kingdom, EH3 9HA
Moorfields Eye Hospital
London, United Kingdom, EC1V 2PD
Sponsors and Collaborators
Ophthotech Corporation

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Responsible Party: Ophthotech Corporation
ClinicalTrials.gov Identifier: NCT03364153     History of Changes
Other Study ID Numbers: OPH2005
First Posted: December 6, 2017    Key Record Dates
Last Update Posted: March 4, 2019
Last Verified: August 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ophthotech Corporation:
Zimura
ARC1905
complement factor C5 inhibitor
avacincaptad pegol
STGD1

Additional relevant MeSH terms:
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Macular Degeneration
Retinal Degeneration
Retinal Diseases
Eye Diseases
Complement System Proteins
Immunologic Factors
Physiological Effects of Drugs