Stargardt's Macular Dystrophy
Conditions
Keywords
Zimura (previous name), ARC1905, complement factor C5 inhibitor, avacincaptad pegol, STGD1, Izervay, Stargardt Disease 1, Stargardt's Macular Dystrophy
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).
Interventions
Intravitreal Injection
DRUGSham
Intravitreal Injection
Sponsors
Astellas Pharma Global Development, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 60 Years
Healthy volunteers
No
Inclusion criteria
* 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
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 * 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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean Rate of Change in the Area of Ellipsoid Zone Defect From Baseline Through Month 18 | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Best Corrected Visual Acuity (BCVA) Using Early Treatment Diabetic Retinopathy Study (ETDRS) Letters From Baseline at Month 18 | Baseline and 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. |
| Change in Photopic or Mesopic Macular Sensitivity Measured by Microperimetry From Baseline at Month 18 | Baseline and 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. |
| Number of Participants With Adverse Events (AEs) | Up to 18 months | 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. |
Countries
France, Germany, Hungary, Israel, Italy, Spain, United Kingdom, United States
Contacts
STUDY_DIRECTORMedical Director
Astellas Pharma Global Development, Inc.
Participant flow
Recruitment details
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.
Pre-assignment details
Participants who met all inclusion criteria and none of the exclusion criteria were enrolled in the study.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 34.9 Years STANDARD_DEVIATION 9.36 |
| Area of Ellipsoid Zone Defect | 4.13 mm^2 STANDARD_DEVIATION 3.86 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 26 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 95 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 6 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 108 Participants |
| Sex: Female, Male Female | 41 Participants |
| Sex: Female, Male Male | 20 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 61 | 0 / 60 |
| other Total, other adverse events | 47 / 61 | 41 / 58 |
| serious Total, serious adverse events | 3 / 61 | 2 / 58 |
Outcome results
None listed