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| ID | Type | Description | Link |
|---|---|---|---|
| 2017-004783-35 | EudraCT Number |
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| avacincaptad pegol | Experimental | Participants will receive avacincaptad pegol monthly for up to 17 Months. |
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| Sham | Sham Comparator | Participants will receive a matching sham monthly for up to 17 Months. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| avacincaptad pegol | Drug | Intravitreal Injection |
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| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Astellas Pharma Global Development, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Retinal Research Institute | Phoenix | Arizona | 85053 | United States | ||
| Jules Stein Eye Institute/ David Geffen School of Medicine |
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| Label | URL |
|---|---|
| Link to plain language summary of the study on the Trial Results Summaries website. | View source |
| Link to results and other applicable study documents on the Astellas Clinical Trials website. | View source |
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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/.
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.
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.
Participants who met all inclusion criteria and none of the exclusion criteria were enrolled in the study.
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.
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| ID | Title | Description |
|---|---|---|
| FG000 | Avacincaptad Pegol | The participants received Avacincaptad pegol (ACP) 2 mg/eye on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
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| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Mar 21, 2025 | Feb 4, 2026 |
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| Sham | Drug | Intravitreal Injection |
|
| 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. | 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. | Up to 18 months |
| 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 |
| FG001 |
| Sham |
The participants received Sham on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
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| COMPLETED |
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| NOT COMPLETED |
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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.
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| ID | Title | Description |
|---|---|---|
| BG000 | Avacincaptad Pegol | The participants received ACP 2 mg/eye on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
|
| BG001 | Sham | The participants received Sham on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
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| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Area of Ellipsoid Zone Defect | The area of ellipsoid zone defect was measured by en face spectral domain-optical coherence tomography. | 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. Only participants with available data at Baseline were included. | Mean | Standard Deviation | mm^2 |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | 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. Participants with either a non-missing baseline or non-missing post-baseline assessment were included. | Posted | Least Squares Mean | Standard Error | mm^2/18 months | Baseline to Month 18 |
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| Secondary | 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. | 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. Participants with a non-missing baseline and at least one non-missing post-baseline assessment were included. | Posted | Least Squares Mean | Standard Error | ETDRS letters | Baseline and Month 18 |
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| Secondary | 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. | 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. Participants with a non-missing baseline and at least one non-missing post-baseline assessment were included. | Posted | Least Squares Mean | Standard Error | dB | Baseline and Month 18 |
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| Secondary | 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. | Safety Analysis Set (SAF) included all participants who received at least one dose of study drug. Participants were analyzed in the ACP group if they ever received ACP at any time during the study. Participants that received Sham and never received ACP were analyzed in the Sham group. | Posted | Number | Participants | Up to 18 months |
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All-cause mortality (ACM): From randomization up to 18 months. Serious Adverse events (SAEs) and non-SAEs: From first dose up to 18 months.
Enrolled Population included all participants who were enrolled. SAEs and non-SAEs: SAF included all participants who received at least one dose of study drug.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Avacincaptad Pegol | The participants received ACP 2 mg/eye on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
| 1 | 61 | 3 | 61 | 47 | 61 |
| EG001 | Sham | The participants received Sham on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
| 0 | 60 | 2 | 58 | 41 | 58 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Ligament rupture | Injury, poisoning and procedural complications | MedDRA v24.1 | Systematic Assessment |
| |
| Road traffic accident | Injury, poisoning and procedural complications | MedDRA v24.1 | Systematic Assessment |
| |
| Endophthalmitis | Eye disorders | MedDRA v24.1 | Systematic Assessment |
| |
| Hypotony of eye | Eye disorders | MedDRA v24.1 | Systematic Assessment |
| |
| Rhegmatogenous retinal detachment | Eye disorders | MedDRA v24.1 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA v24.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Dry eye | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Eye irritation | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Eye pain | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Ocular hyperaemia | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Punctate keratitis | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Seasonal allergy | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Vision blurred | Eye disorders | MedDRA v24.1 | Systematic Assessment |
| |
| Vitreous detachment | Eye disorders | MedDRA v24.1 | Systematic Assessment |
| |
| Vitreous floaters | Eye disorders | MedDRA v24.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v24.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v24.1 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA v24.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA v24.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.1 | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA v24.1 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA v24.1 | Systematic Assessment |
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| Intraocular pressure increased | Investigations | MedDRA v24.1 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v24.1 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA v24.1 | Systematic Assessment |
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| Hypertension | Vascular disorders | MedDRA v24.1 | Systematic Assessment |
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| Conjunctival haemorrhage | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Conjunctival hyperaemia | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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| Conjunctival oedema | Eye disorders | MedDRA v24.1 | Systematic Assessment |
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Institute and/or Principal Investigator may publish trial data generated at their specific study site after Sponsor publication of the multi-center data. Sponsor must receive a site's manuscript prior to publication for review and comment as specified in the Investigator Agreement.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Transparency | Astellas Pharma Global Development, Inc | 800-888-7704 | astellas.resultsdisclosure@astellas.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 3, 2025 | Feb 4, 2026 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000080362 | Stargardt Disease |
| ID | Term |
|---|---|
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D008268 | Macular Degeneration |
| D012162 | Retinal Degeneration |
| D012164 | Retinal Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| C005703 | salicylhydroxamic acid |
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The participants received Sham on Day 1, Month 1, and Month 2 in the following sequence, 14 days apart in the Induction Phase:
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