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
2 other identifiers
interventional
121
8 countries
41
Brief Summary
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Jan 2018
Longer than P75 for phase_2
41 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 1, 2017
CompletedFirst Posted
Study publicly available on registry
December 6, 2017
CompletedStudy Start
First participant enrolled
January 12, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 31, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
March 31, 2025
CompletedResults Posted
Study results publicly available
March 12, 2026
CompletedApril 15, 2026
April 1, 2026
7.2 years
December 1, 2017
February 4, 2026
April 13, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18
The area of ellipsoid zone defect was measured by en face spectral domain-optical coherence tomography. Rate of change (slope) in the area of ellipsoid zone defect from Baseline through Month 18 was estimated using mixed model for repeated measures (MMRM).
Baseline to Month 18
Secondary Outcomes (3)
Change in Best Corrected Visual Acuity (BCVA) Using Early Treatment Diabetic Retinopathy Study (ETDRS) Letters From Baseline at Month 18
Baseline and Month 18
Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18
Baseline and Month 18
Number of Participants With Adverse Events (AEs)
Up to 18 months
Study Arms (2)
avacincaptad pegol
EXPERIMENTALParticipants will receive avacincaptad pegol monthly for up to 17 Months.
Sham
SHAM COMPARATORParticipants will receive a matching sham monthly for up to 17 Months.
Interventions
Intravitreal Injection
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (41)
Retinal Research Institute
Phoenix, Arizona, 85053, United States
Jules Stein Eye Institute/ David Geffen School of Medicine
Los Angeles, California, 90095, United States
VitreoRetinal Associates
Gainesville, Florida, 32607, United States
Retina Specialty Institute
Pensacola, Florida, 32503, United States
Wilmer Eye Institute, Johns Hopkins
Baltimore, Maryland, 21287, United States
Ophthalmic Consultants of Boston
Boston, Massachusetts, 02114, United States
University of Michigan/Kellogg Eye Center
Ann Arbor, Michigan, 48105, United States
The Retina Center
Minneapolis, Minnesota, 55404, United States
Retina Center of NJ, LLC.
Bloomfield, New Jersey, 07003, United States
Casey Eye Institute/Oregon Health & Science University
Portland, Oregon, 97239, United States
Wills Eye Hospital/Mid Atlantic Retina
Philadelphia, Pennsylvania, 19107, United States
UPMC Eye Center
Pittsburgh, Pennsylvania, 15213, United States
Palmetto Retina Center
West Columbia, South Carolina, 29169, United States
Austin Retina Associates
Austin, Texas, 78705, United States
Retina Foundation of the Southwest
Dallas, Texas, 75231, United States
Strategic Clinical Research Group
Willow Park, Texas, 76087, United States
University of Utah John A. Moran Eye Center
Salt Lake City, Utah, 84132, United States
Hopital de la Croix-Rousse
Lyon, Auvergne-Rhône-Alpes, 69004, France
Creteil University Eye Clinic University Paris EST
Créteil, 94010, France
Centre ophtalmologique des Quinzes Vingts
Paris, 75012, France
University of Bonn
Bonn, 53127, Germany
Augenklinik der LMU München
München, 80336, Germany
University of Tuebingen
Tübingen, 72076, Germany
Budapest Retina Institute
Budapest, 1133, Hungary
Semmelweis Egyetem
Budapest, H-1083, Hungary
University of Debrecen DE KK Szemészeti Klinika
Debrecen, 4032, Hungary
Ganglion Medical Center
Pécs, 7621, Hungary
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
Szeged, 6720, Hungary
Rambam Health Care Campus
Haifa, 3109601, Israel
Hadassah University Hospital
Jerusalem, 9112001, Israel
Rabin Medical Center, Beilinson campus
Petah Tikva, 4941492, Israel
Kaplan Medical Center
Rehovot, 7610001, Israel
Tel-Aviv Sourasky Medical Center, Ichilov Hospital
Tel Aviv, 6423906, Israel
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
Bologna, 40138, Italy
Azienda Ospedaliera Universitaria Careggi
Florence, 50121, Italy
Ospedale San Raffaele
Milan, 20132, Italy
University of Campania Luigi Vanvitelli Eye Clinic
Naples, 80131, Italy
Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
Rome, 00133, Italy
Institut de la Macula
Barcelona, 08022, Spain
Princess Alexandra Eye Pavillion
Edinburgh, EH3 9HA, United Kingdom
Moorfields Eye Hospital
London, EC1V 2PD, United Kingdom
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Clinical Transparency
- Organization
- Astellas Pharma Global Development, Inc
Study Officials
- STUDY DIRECTOR
Medical Director
Astellas Pharma Global Development, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- TRIPLE
- Who Masked
- PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 1, 2017
First Posted
December 6, 2017
Study Start
January 12, 2018
Primary Completion
March 31, 2025
Study Completion
March 31, 2025
Last Updated
April 15, 2026
Results First Posted
March 12, 2026
Record last verified: 2026-04
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, CSR
- Time Frame
- Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data.
- Access Criteria
- Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement.
Access to anonymized individual participant level data collected during the study, in addition to study-related supporting documentation, is planned for studies conducted with approved product indications and formulations, as well as products terminated during development. Studies conducted with product indications or formulations that remain active in development are assessed after study completion to determine if Individual Participant Data can be shared. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.