Geographic Atrophy (AD) secondary to Age-related Macular Degeneration (AMD). MedDRA version: 20.1 Level: LLT Classification code 10063947 Term: Geographic atrophy System Organ Class: 100000004853
Conditions
Interventions
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: DANICOPAN
CAS Number: 1903768-17-1
Current Sponsor code: ACH-0144471
Other des
Sponsors
Alexion Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: • Age = 70 years, male or female • Presentation of GA secondary to AMD in at least 1 eye. • Study eye must have the specified VA (range of 84 to 4 letters; 20/20 to 20/800) using Early Treatment Diabetic Retinopathy Study charts at starting distance of 4 meters. • GA area of 0.5 to 17.76 mm2 (~0.25 to 7 disc area [DA]) per eye measured by FAF • The entire GA lesion must be extrafoveal without foveal involvement Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 330
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria: • GA in either eye due to cause other than AMD. • Have previously received intravitreal anti-vascular endothelial growth factor 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. • Presence of active ocular diseases in either eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments. • Known or suspected complement deficiency. • History or presence of any clinically relevant co-morbidities or any uncontrolled conditions. • Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients. Note: Additional inclusion/exclusion criteria may apply, per protocol.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD.;Secondary Objective: To evaluate the effect of danicopan on disease progression utilizing anatomical measures compared to placebo To evaluate the effect of danicopan on disease progression utilizing functional measures compared to placebo To evaluate the PK and PD of danicopan in patients with GA secondary to AMD To evaluate the safety and tolerability of danicopan in patients with GA secondary to AMD;Primary end point(s): Mean rate of change from Baseline at Week 52 in the square root (sqrt) of total GA lesion area (mm/year) in the study eye as measured by fundus autofluorescence (FAF);Timepoint(s) of evaluation of this end point: week 52 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 2. Mean Rate Of Change From Baseline At Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye And From Baseline At Week 52 And Week 104 In The Fellow Eye And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104] 3. Mean Rate Of Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104] 4. Mean Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104] 5. Percent Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104] 6. Mean Change From Baseline At Week 52 And Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104] 7. Percent Change From Baseline At Week 52 And Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104] 8. Mean Change From Baseline At Week 52 And Week 104 In Monocular Best-corrected Visual Acuity (BCVA) Scores In The Study Eye And Fellow Eye As Assessed By The Early Treatment Diabetic Retinopathy Study (ETDRS) Chart At Four Meters [Time Frame: Baseline, Week 52 and Week 104] 9. Mean Change From Baseline At Week 52 And Week 104 In Monocular Low Luminance Visual Acuity (LLVA) Scores In The Study Eye And Fellow Eye As Assessed By The ETDRS Chart At F | — |
Countries
Australia, Canada, Czechia, France, Germany, Hungary, Italy, Japan, Korea, Republic of, Latvia, New Zealand, Slovakia, Spain, United Kingdom, United States
Contacts
Public ContactAnna Anguera
Alexion Pharma Spain S.L.
Outcome results
None listed