Clinical Trials

Inclusion Criteria:

• Age ≥ 60 years, male or female.
• Documentation of vaccination for Neisseria meningitidis: All patients must be vaccinated against meningococcal infections within 3 years prior to, or at the time of, initiating study drug. Patients who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics until 2 weeks after vaccination.
• For female patients, confirmation of non-childbearing potential based on follicle-stimulating hormone (FSH) test at Screening only.
• For nonsterile male patients, agreement to use a highly effective or acceptable method of contraception with their partner(s) of childbearing potential from the first day of dosing to 90 days after their last dose of study drug. Males who are surgically sterile do not need to employ additional contraception.
• For male patients, agreement not to donate sperm while enrolled in this study and for 90 days after their last dose of study drug.
• Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

Ocular Inclusion Criteria:

• Presentation of GA secondary to AMD in at least one eye, characterized by:
  • The study eye must have the specified VA (range of 84 to 4 letters; 20/20 to 20/800) using Early Treatment Diabetic Retinopathy Study (ETDRS) charts at starting distance of 4 meters;
  • Adequate clarity of ocular media, adequate pupillary dilation, and fixation assessed by slit lamp examination and indirect ophthalmoscopy to permit the collection of good quality images as determined by the Investigator;
  • Axial length ≤ 26.0 mm measured by biometry (if available) OR spherical equivalent refractive error ≤ 6.0 diopter of myopia:
    • For patients with history of refractive or cataract surgery in the study eye, axial length ≤ 26.0 mm measured by biometry (if available); OR
    • Preoperative spherical equivalent refractive error ≤ 6.0 diopter of myopia.
• The entire GA lesion must be completely visualized on the macula centered color fundus photography (CFP) image and must be able to be imaged in its entirety.
• GA area of 0.5 to 17.76 mm^2 (~0.25 to 7 disc area [DA]) per eye measured by FAF.

Exclusion Criteria:

Ocular Exclusion Criteria:

• GA in either eye due to cause other than AMD (eg, pathological myopia, monogenetic macular dystrophies eg, Stargardt/cone-rod dystrophy) or toxic maculopathies (eg, chloroquine/ hydroxychloroquine maculopathy) per Investigator’s judgment.
• Any eye with GA and concomitant exudative nAMD.
• Have previously received intravitreal anti-vascular endothelial growth factor (VEGF) injections in study eye.
• Have previously received any complement/stem cell/gene therapy for any ophthalmological condition.
• Previous participation in interventional clinical studies for treatment of drusen, nascent GA or GA (except vitamins or minerals) irrespective of route of administration (ocular or systemic) in either eye.
• Previous laser photocoagulation for nAMD, diabetic macular edema, retinal vein occlusion and proliferative diabetic retinopathy in either eye.
• Previous photodynamic therapy (visudyne), external beam radiation therapy and/or any other irradiation (eg, isotope, charged particle, photon, x-ray) to the eye, orbit, head, and/or neck or transpupillary thermotherapy in either eye.
• Previous intravitreal delivery of steroid, anti-complement, or device implantation in either eye. A single steroid for cystoid macular edema after cataract ≥ 3 months is permitted.
• Any concurrent ocular or intraocular condition (cataract, diabetic retinopathy) in either eye per Investigator’s judgement that could result in surgery to prevent vision loss during the study; if allowed to progress could contribute to ≥ 2 Snellen equivalent lines of BCVA loss during the study.
• Presence of an active ocular diseases in either eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments, including but not limited to, uveitis, keratitis, scleritis or endophthalmitis, vitreous hemorrhage, other macular diseases (eg, clinically significant epiretinal membrane, full thickness macular hole, RPE tear in macula), central serous retinopathy, uncontrolled glaucoma/ocular hypertension (intraocular pressure [IOP] ≥ 30 mmHg on anti-glaucoma medications), proliferative diabetic retinopathy.
• History of any of the following in either eye:
  • recurrent infectious or inflammatory eye disease;
  • retinal detachment or macular hole;
  • glaucoma-filtering surgery;
  • corneal transplantation;
  • citrectomy, submacular surgery, or any surgical intervention for AMD;
  • prophylactic subthreshold laser treatment for AMD;
  • intraocular surgery (including lens replacement surgery) within 3 months prior to randomization.
• If the following changes occur between Screening and Day 1, as determined by eligibility review at Day 1:
  • VA change of ≥ 5 letters; OR
  • the Snellen equivalent is no longer between 20/20 to 20/800; OR
  • significant anatomical changes (i.e., large subretinal hemorrhage, RPE rip, pigment epithelial detachment, or other conditions that meet the exclusion criteria per Investigator discretion).

Exclusion Criteria:

• Known or suspected complement deficiency
• History of N meningitidis infection.
• Active bacterial or viral infection, a body temperature > 38°C (100.4°F) on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study drug administration.
• History of malignant disease within the past 5 years or ongoing, including solid tumors and hematologic malignancies (with the exception of basal cell and squamous cell carcinomas of the skin that have been completely excised and are considered cured).
• Abnormal liver function tests, defined as:
  • ALT, aspartate aminotransferase (AST), alkaline phosphatase (ALP) or direct bilirubin > 2 × upper limit of normal (ULN) at Screening;
  • Patients with Gilbert's Syndrome will not be excluded. If increased bilirubin is suggestive of Gilbert's syndrome, the patient has to provide documentation of the diagnosis or will be tested for this condition.
• History or presence of any of the following:
  • any clinically relevant co-morbidities;
  • any uncontrolled conditions;
  • any medical or psychological abnormality that, in the opinion of the Principal Investigator, would make the patient inappropriate for the study or unable to comply with study procedures or put the patient at undue risk or confound the results of the study.
• Current evidence of hepatobiliary cholestasis.
• Evidence of infection of hepatitis B virus (positive hepatitis B surface antigen [HBsAg] or positive core antibody (anti-HBc) with negative surface antibody [anti-HBs]) or hepatitis C virus (HCV antibody positive) or human immunodeficiency virus (HIV antibody positive) at Screening.
• Estimated glomerular filtration rate < 30 mL/min/1.73 m^2 and/or are on dialysis.
• Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients.
• Participation in other clinical trials that entailed receipt of another investigational medicinal product within 30 days for systemic non-biologics or 6 months for biologics and intravitreal treatments.

Eligibility last updated 1/25/22.  Questions regarding updates should be directed to the study team contact.