Stargardt's Macular DystrophyPhase 2NCT03364153
Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)
A Phase 2 study testing avacincaptad pegol in people with Stargardt's Macular Dystrophy, run by Astellas Pharma Global Development, Inc. at 41 sites in 8 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Stargardt's Macular Dystrophy
- What is being tested?
- avacincaptad pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 121 participants
- Where
- 41 sitesin 8 countries
- Study dates
- 12 Jan 2018to 31 Mar 2025
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 12Show fewer
- 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
Trial locations and contacts
41 sites in 8 countries.
41 sites in 8 countries
United States17 sites
University of Michigan/Kellogg Eye Center
Ann Arbor, Michigan, 48105
Austin Retina Associates
Austin, Texas, 78705
Wilmer Eye Institute, Johns Hopkins
Baltimore, Maryland, 21287
Retina Center of NJ, LLC.
Bloomfield, New Jersey, 07003
Ophthalmic Consultants of Boston
Boston, Massachusetts, 02114
Retina Foundation of the Southwest
Dallas, Texas, 75231
VitreoRetinal Associates
Gainesville, Florida, 32607
Jules Stein Eye Institute/ David Geffen School of Medicine
Los Angeles, California, 90095
The Retina Center
Minneapolis, Minnesota, 55404
Retina Specialty Institute
Pensacola, Florida, 32503
Wills Eye Hospital/Mid Atlantic Retina
Philadelphia, Pennsylvania, 19107
Retinal Research Institute
Phoenix, Arizona, 85053
UPMC Eye Center
Pittsburgh, Pennsylvania, 15213
Casey Eye Institute/Oregon Health & Science University
Portland, Oregon, 97239
University of Utah John A. Moran Eye Center
Salt Lake City, Utah, 84132
Palmetto Retina Center
West Columbia, South Carolina, 29169
Strategic Clinical Research Group
Willow Park, Texas, 76087
Hungary5 sites
Budapest Retina Institute
Budapest, 1133
Semmelweis Egyetem
Budapest, H-1083
University of Debrecen DE KK Szemészeti Klinika
Debrecen, 4032
Ganglion Medical Center
Pécs, 7621
Szegedi Tudomanyegyetem, Szent-Gyorgyi Albert Klinikai Kozpont, Szemeszeti Klinika
Szeged, 6720
Israel5 sites
Rambam Health Care Campus
Haifa, 3109601
Hadassah University Hospital
Jerusalem, 9112001
Rabin Medical Center, Beilinson campus
Petah Tikva, 4941492
Kaplan Medical Center
Rehovot, 7610001
Tel-Aviv Sourasky Medical Center, Ichilov Hospital
Tel Aviv, 6423906
Italy5 sites
AOU Policlinico Sant'Orsola Malpighi, U.O. Oftalmologia,
Bologna, 40138
Azienda Ospedaliera Universitaria Careggi
Florence, 50121
Ospedale San Raffaele
Milan, 20132
University of Campania Luigi Vanvitelli Eye Clinic
Naples, 80131
Fondazione Policlinico Tor Vergata, UOSD Patologie Retiniche
Rome, 00133
France3 sites
Creteil University Eye Clinic University Paris EST
Créteil, 94010
Hopital de la Croix-Rousse
Lyon, Auvergne-Rhône-Alpes, 69004
Centre ophtalmologique des Quinzes Vingts
Paris, 75012
Germany3 sites
University of Bonn
Bonn, 53127
Augenklinik der LMU München
München, 80336
University of Tuebingen
Tübingen, 72076
United Kingdom2 sites
Princess Alexandra Eye Pavillion
Edinburgh, EH3 9HA
Moorfields Eye Hospital
London, EC1V 2PD
About this study
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
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).
- Conditions
- Stargardt's Macular Dystrophy
- Being tested
- avacincaptad pegol, Sham
- Keywords
- Zimura (previous name), ARC1905, complement factor C5 inhibitor, avacincaptad pegol, STGD1, Izervay, Stargardt Disease 1, Stargardt's Macular Dystrophy
- Medical subject headings
- Stargardt Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
avacincaptad pegolDrug
Also called Zimura (previous name), ARC1905, Izervay
Intravitreal Injection
ShamDrug
Intravitreal Injection
| Study group | Role | What they receive |
|---|---|---|
| avacincaptad pegol | Experimental | Participants will receive avacincaptad pegol monthly for up to 17 Months. Receives: Drug: avacincaptad pegol |
| Sham | Sham comparator | Participants will receive a matching sham monthly for up to 17 Months. Receives: Drug: Sham |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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
BCVA in the study eye was assessed using ETDRS visual acuity testing chart. The ETDRS Visual Acuity Score (ETDRS letters) is calculated based on the number of letters read on the ETDRS chart. Minimum and maximum possible scores are 0-100. A higher score represented increased visual functioning. A positive change from Baseline indicates an decrease in symptomology. Change in BCVA from Baseline at Month 18 was estimated using MMRM.
Measured: Baseline and Month 18
Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18
Photopic macular sensitivity or mesopic macular sensitivity were measured by microperimetry. Participants either had a photopic or mesopic measurement taken depending on the resources available at their site. Researchers were provided with one measurement regardless of the type of lighting conditions the assessment was conducted in. A higher score represented an increased retinal sensitivity. A positive change from Baseline indicates an improvement in symptomology. Change in Photopic or Mesopic Macular Sensitivity from Baseline at Month 18 was estimated using MMRM.
Measured: Baseline and Month 18
Number of Participants With Adverse Events (AEs)
An AE is defined as any untoward medical occurrence in a participant including unfavorable and unintended signs, symptoms or disease temporally associated with the use of a medicinal product and which does not necessarily have to have a causal relationship to this treatment.
AEs include illnesses with onset during the trial, or exacerbations of pre-existing illnesses. Exacerbation of pre-existing illness is defined as a significant increase in the severity of the illness as compared to the start of the trial and was considered when a participant requires new or additional treatment for that illness. Lack of or insufficient clinical response or efficacy was not recorded as an AE.
Measured: Up to 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2017
- Study start12 Jan 2018
- Primary completion31 Mar 2025
- Study completion31 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Stargardt's Macular DystrophyStudies of the same condition, open and under way.
- More trials run by Astellas Pharma Global Development, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 121 participants (actual)
Sponsor and investigators
- Lead sponsor
- Astellas Pharma Global Development, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Astellas Pharma Global Development, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03364153
- Sponsor's protocol ID
- OPH2005
- Other IDs
- 2017-004783-35, 2017
- Status last verified
- Apr 2026
- First submitted
- 1 Dec 2017
- Last updated
- 15 Apr 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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/.
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03364153
- Acronym / sponsor ID
- OPH2005
- Conditions
- Stargardt's Macular Dystrophy
- Keywords
- Zimura (previous name), ARC1905, complement factor C5 inhibitor, avacincaptad pegol, STGD1, Izervay, Stargardt Disease 1, Stargardt's Macular Dystrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 121 participants (actual)
- Lead sponsor
- Astellas Pharma Global Development, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Jan 2018 (actual)
- Primary completion
- 31 Mar 2025 (actual)
- Study completion
- 31 Mar 2025 (actual)
- First posted
- 6 Dec 2017 (actual)
- Results
- Posted 12 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| avacincaptad pegol | Experimental | Drug: avacincaptad pegolParticipants will receive avacincaptad pegol monthly for up to 17 Months. |
| Sham | Sham comparator | Drug: ShamParticipants will receive a matching sham monthly for up to 17 Months. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| avacincaptad pegolAlso: Zimura (previous name), ARC1905, IzervayIntravitreal Injection | Drug | avacincaptad pegol |
| ShamIntravitreal Injection | Drug | Sham |
Primary outcome
Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18
Time frame: Baseline to 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).
3 secondary outcomes
Change in Best Corrected Visual Acuity (BCVA) Using Early Treatment Diabetic Retinopathy Study (ETDRS) Letters From Baseline at Month 18
Time frame: Baseline and Month 18
Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18
Time frame: Baseline and Month 18
Number of Participants With Adverse Events (AEs)
Time frame: Up to 18 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Link to plain language summary of the study on the Trial Results Summaries website.Link provided by the sponsor.
- Link to results and other applicable study documents on the Astellas Clinical Trials website.Link provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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/.
Related trials for Stargardt's Macular Dystrophy
RecruitingObservational study
Rod and Cone Mediated Function in Retinal Disease
Retinal Degeneration, Retinitis Pigmentosa
Active, not recruitingPhase 2
Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration
Stargardt's Disease
RecruitingObservational study
An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene
Stargardt, Stargardt's Disease
Not yet recruiting
Feasibility and Tolerability Study of Smart Contact Lens With Healthy Subjects and Patients With Stargardt's Disease
Stargardt Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Astellas Pharma Global Development, Inc.
41 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing avacincaptad pegol in people with Stargardt's Macular Dystrophy, run by Astellas Pharma Global Development, Inc. at 41 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in United States, Hungary, Israel, Italy, France and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18 (time frame: Baseline to Month 18). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.