Trial Outcomes & Findings for Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1) (NCT NCT03364153)

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.

Participants who met all inclusion criteria and none of the exclusion criteria were enrolled in the study.

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.

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).

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.

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.

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.