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A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

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ClinicalTrials.gov Identifier: NCT05019521
Recruitment Status : Recruiting
First Posted : August 25, 2021
Last Update Posted : February 17, 2023
Sponsor:
Information provided by (Responsible Party):
Alexion

Study Description
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Brief Summary:
This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up to 6 weeks, a 104-week masked Treatment Period, followed by a 30-day Follow-up after the last dose. This study will have 4 treatments arms: 100 milligrams (mg) twice daily (bid), 200 mg bid, 400 mg once daily (qd), and matching placebo.

Condition or disease Intervention/treatment Phase
Geographic Atrophy Drug: Danicopan Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 332 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Masking of treatment allocation will be observed until Week 104.
Primary Purpose: Treatment
Official Title: A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Actual Study Start Date : August 20, 2021
Estimated Primary Completion Date : May 31, 2024
Estimated Study Completion Date : June 30, 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Danicopan: 100 mg
Participants will receive danicopan 100 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.
 Drug: Danicopan
Oral tablet.
Other Name: ALXN2040
Experimental: Danicopan: 200 mg
Participants will receive danicopan 200 mg bid during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.
 Drug: Danicopan
Oral tablet.
Other Name: ALXN2040
Experimental: Danicopan: 400 mg
Participants will receive danicopan 400 mg qd during the masked Treatment Period. Once the optimal dose is identified, participants who have at least 52 weeks of treatment will be switched to the optimal dose for the remainder of the study.
 Drug: Danicopan
Oral tablet.
Other Name: ALXN2040
Placebo Comparator: Placebo
Participants will receive matching placebo and will be re-randomized to one of the active treatment groups at Week 52, or to the optimal dose, if already identified.
 Drug: Danicopan
Oral tablet.
Other Name: ALXN2040

Drug: Placebo
Oral tablet.


Outcome Measures
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Primary Outcome Measures :
  1. Change From Baseline To Week 52 In The Square Root (sqrt) Of Total GA Lesion Area In The Study Eye As Measured By Fundus Autofluorescence (FAF) [ Time Frame: Baseline, Week 52 ]

Secondary Outcome Measures :
  1. Change From Baseline To Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye As Measured By FAF [ Time Frame: Baseline, Week 104 ]
  2. Change From Baseline To Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye As Measured By FAF [ Time Frame: Baseline, Week 52 and Week 104 ]
  3. Change From Baseline To Week 52 And Week 104 In Monocular Best-corrected Visual Acuity (BCVA) Scores In The Study Eye As Assessed By The Early Treatment Diabetic Retinopathy Study (ETDRS) Chart [ Time Frame: Baseline, Week 52 and Week 104 ]
  4. Change From Baseline To Week 52 And Week 104 In Monocular Low Luminance Visual Acuity (LLVA) Scores In The Study Eye As Assessed By The ETDRS Chart [ Time Frame: Baseline, Week 52 and Week 104 ]
  5. Change From Baseline To Week 52 And Week 104 In Low Luminance Deficit (BCVA-LLVA) In The Study Eye [ Time Frame: Baseline, Week 52 and Week 104 ]
  6. Change From Baseline To Week 52 And Week 104 In Monocular Reading Speeds In The Study Eye As Assessed By Minnesota Low Vision Reading Test (MNRead) Acuity Charts Or Radner Reading Charts [ Time Frame: Baseline, Week 52 and Week 104 ]
  7. Plasma Concentration Of Danicopan Over Time [ Time Frame: Up to 4 hours postdose ]
  8. PD Biomarkers, Ex Vivo Serum Alternative Pathway Activity, And Plasma Bb Concentration Over Time [ Time Frame: Up to 4 hours postdose ]
  9. Change From Baseline To Week 52 and Week 104 in National Eye Institute Visual Function Questionnaire (NEI VFQ-25) scores [ Time Frame: Baseline, Week 52 and Week 104 ]
  10. Incidence Of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), And Ocular TEAEs, SAEs, And Clinical Laboratory Abnormalities, And Events Leading To Discontinuation Of Study Drug Throughout The Study [ Time Frame: Day 1 through Week 104 ]

Eligibility Criteria
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Ages Eligible for Study:   60 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Vaccination for Neisseria meningitidis.
  • Capable of giving signed informed consent.
  • Presentation of GA secondary to AMD in at least 1 eye
  • The entire GA lesion must be > 1 μm outside of the foveal center

Key Exclusion Criteria:

  • GA in the study eye due to cause other than AMD.
  • Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye for intraocular vascular disease.
  • Have previously received any stem cell/gene therapy for any ophthalmological condition in either eye.
  • Previous participation in interventional clinical studies for any ophthalmic indications in the study eye regardless of route of administration within the last 3 months or 5 half-lives of the investigational product (whichever is longer).
  • Presence of active ocular diseases in the study 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 medical or psychological condition that, in the opinion of the Principal Investigator, would make the patient inappropriate for the study.
  • Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients.

Note: Additional inclusion/exclusion criteria may apply, per protocol.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05019521


Contacts
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Contact: Alexion Pharmaceuticals, Inc. 855-752-2356 clinicaltrials@alexion.com

Locations
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Sponsors and Collaborators
Alexion
More Information
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Responsible Party: Alexion
ClinicalTrials.gov Identifier: NCT05019521    
Other Study ID Numbers: ALXN2040-GA-201
First Posted: August 25, 2021    Key Record Dates
Last Update Posted: February 17, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion:
Geographic Atrophy
GA
Danicopan
 ALXN2040
Complement Inhibition
AMD
Additional relevant MeSH terms:
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Macular Degeneration
Geographic Atrophy
Atrophy
Retinal Degeneration
 Retinal Diseases
Eye Diseases
Pathological Conditions, Anatomical