A Phase 2 interventional study of avacincaptad pegol and Sham in Stargardt's Macular Dystrophy, sponsored by Astellas Pharma Global Development, Inc.. Completed at 41 sites in 8 countries. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-04-15.
Sponsored by Astellas Pharma Global Development, Inc. (part of Astellas Pharma Inc) · Phase 2, Interventional, and Treatment
Among 42 studies in Stargardt Disease with a site in United States — 16 open to participants now, 25 under way, 3 of them phase 3. Stargardt Disease · United States.
Among the 42 studies in Stargardt Disease with a site in United States, this one is the 7th largest by enrollment of the 26 interventional ones that give a count, is the only one that started in 2018, is one of 7 in phase 2, and was the 18th registered.
66 studies on the registry are indexed under Stargardt Disease; 25 are open to participants now.
Browse Stargardt Disease studies →16 of the 42 studies in Stargardt Disease with a site in United States are open now. Recruiting first, the newest first:
Astellas Institute for Regenerative Medicine · Sites in United States
Ages 6+
Alkeus Pharmaceuticals, Inc. · Sites in United States
Ages 8–45
Cellio Therapeutics Inc · Sites in United States
Ages 18+
Astellas Pharma Global Development, Inc. · Sites in United States
Ages 6+
Ray Therapeutics, Inc. · Sites in United States
Ages 18+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 22 Stargardt Disease studies recruiting now, 14 (64%) set no upper age limit and 7 (32%) stop at 75 or younger; 13 take children under 18; 5 take healthy volunteers.
This study: 18 Years to 60 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
42 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 12 · Phase 2 16 · Phase 3 5
First in 2003; this study’s year is marked. 1 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 121 is above the median of 30 across 43 interventional studies indexed under Stargardt Disease.
Median enrollment 41 across the 26 interventional studies, 63 across the 16 observational. Of the 16 that completed or were terminated, 5 (31%) have results posted.
This tab as a report to print or save as PDF → With an account.
31 of 42 have an industry lead sponsor. The 10 most frequent:
Astellas Pharma Global Development, Inc. is the lead sponsor of 207 studies on the registry; 23 are open to participants now.
Of its 82 completed or terminated interventional studies of FDA-regulated products, 38 (46%) have results posted.
Astellas Pharma Global Development, Inc. has 2 studies filed under Stargardt Disease — fewer than ten, so there is no comparison to draw.
6 of the studies in Stargardt Disease with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06435000Splice BioAn 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 | 2026-12 |
| NCT07502664Ray Therapeutics, Inc.Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) | 2027-02 |
| NCT06048185Nanoscope Therapeutics Inc.Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study | 2027-05 |
| NCT06591806AAVantgarde Bio SrlAn Observational Study in Children and Adults With Stargardt Disease | 2027-06-30 |
| NCT03011541MD Stem CellsStem Cell Ophthalmology Treatment Study II | 2027-07-31 |
Among the 42 studies in Stargardt Disease with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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).
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
HbA1c
A blood test of average blood sugar over the past two to three months, given as a percentage or in mmol/mol. 6.5% (48 mmol/mol) or more is the usual threshold for diabetes, normally confirmed by a second test.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive avacincaptad pegol monthly for up to 17 Months.
Drug: avacincaptad pegol
Participants will receive a matching sham monthly for up to 17 Months.
Drug: Sham
Intravitreal Injection
Also known as: Zimura (previous name), ARC1905, Izervay
Intravitreal Injection
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).
Time frame: Baseline to Month 18
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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 18 months
Participants of either gender, of 18 to 60 years of age (inclusive), with the diagnosis of autosomal recessive Stargardt Disease 1 (STGD1) were enrolled in this study.
| Milestone | Avacincaptad Pegol | Sham |
|---|---|---|
| Started | 61 | 60 |
| Completed | 44 | 53 |
| Not completed | 17 | 7 |
| Withdrew: Adverse event | 4 | 1 |
| Withdrew: Protocol violation | 1 | 2 |
| Withdrew: Subject request | 7 | 3 |
| Withdrew: Lost to follow-up | 2 | 0 |
| Withdrew: Death | 1 | 0 |
| Withdrew: Other | 2 | 1 |
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).
| mm^2/18 months | Avacincaptad Pegol | Sham |
|---|---|---|
| Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18 | 0.6383 ± 0.1113 | 0.6644 ± 0.1081 |
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.
| ETDRS letters | Avacincaptad Pegol | Sham |
|---|---|---|
| Change in Best Corrected Visual Acuity (BCVA) Using Early Treatment Diabetic Retinopathy Study (ETDRS) Letters From Baseline at Month 18 | -0.1326 ± 1.2080 | -1.3358 ± 1.1622 |
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.
| dB | Avacincaptad Pegol | Sham |
|---|---|---|
| Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18 | -1.1604 ± 0.7964 | -1.9184 ± 0.8128 |
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.
| Participants | Avacincaptad Pegol | Sham |
|---|---|---|
| Number of Participants With Adverse Events (AEs) | 53 | 45 |
Collected over All-cause mortality (ACM): From randomization up to 18 months. Serious Adverse events (SAEs) and non-SAEs: From first dose up to 18 months.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Avacincaptad Pegol | 1/61 (1.6%) | 3/61 (4.9%) | 47/61 (77%) |
| Sham | 0/60 (0%) | 2/58 (3.4%) | 41/58 (70.7%) |
| Event | Avacincaptad Pegol | Sham |
|---|---|---|
| Ligament ruptureInjury, poisoning and procedural complications | 0/61 | 1/58 |
| SepsisInfections and infestations | 0/61 | 1/58 |
| Road traffic accidentInjury, poisoning and procedural complications | 1/61 | 0/58 |
| EndophthalmitisEye disorders | 1/61 | 0/58 |
| Hypotony of eyeEye disorders | 1/61 | 0/58 |
| Rhegmatogenous retinal detachmentEye disorders | 1/61 | 0/58 |
| Event | Avacincaptad Pegol | Sham |
|---|---|---|
| Conjunctival haemorrhageEye disorders | 31/61 | 10/58 |
| NasopharyngitisInfections and infestations | 12/61 | 11/58 |
| Intraocular pressure increasedInvestigations | 10/61 | 0/58 |
| HeadacheNervous system disorders | 9/61 | 9/58 |
| Eye painEye disorders | 9/61 | 7/58 |
| Eye irritationEye disorders | 8/61 | 5/58 |
| Punctate keratitisEye disorders | 8/61 | 4/58 |
| Vitreous floatersEye disorders | 7/61 | 1/58 |
| Urinary tract infectionInfections and infestations | 6/61 | 4/58 |
| Conjunctival hyperaemiaEye disorders | 6/61 | 5/58 |
Intent to Treat (ITT) included all participants who were randomized. Participants were analyzed according to the treatment to which they were assigned at the time of randomization regardless of the actual study drug the participant may have received during the participation in the study.
| Age, Continuous(Years) | Avacincaptad Pegol | Sham | Total |
|---|---|---|---|
| Mean | 33.8 ± 7.90 | 36.1 ± 10.58 | 34.9 ± 9.36 |
| Sex: Female, Male(Participants) | Avacincaptad Pegol | Sham | Total |
|---|---|---|---|
| Female | 41 | 31 | 72 |
| Male | 20 | 29 | 49 |
| Ethnicity (NIH/OMB)(Participants) | Avacincaptad Pegol | Sham | Total |
|---|---|---|---|
| Hispanic or Latino | 12 | 14 | 26 |
| Not Hispanic or Latino | 49 | 46 | 95 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Avacincaptad Pegol | Sham | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 3 | 1 | 4 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 3 | 6 | 9 |
| White | 55 | 53 | 108 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Area of Ellipsoid Zone Defect(mm^2) | Avacincaptad Pegol | Sham | Total |
|---|---|---|---|
| Mean | 4.31 ± 4.85 | 3.94 ± 2.46 | 4.13 ± 3.86 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: 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/.
Supporting information: Study protocol, Statistical analysis plan, Clinical study report
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.
Data sharing details ↗ · www.clinicaltrials.astellas.com
Ran from Jan 2018 to primary completion in Mar 2025, 86 months.
Results were first posted in Mar 2026, 11 months after primary completion.
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Studies of the drug tested here: Avacincaptad pegol
Astellas Pharma Global Development, Inc. →