Study to Asses the Safety and Efficacy of Multiple Doses of Ionis-FB-LRX in patients with Geographic Atrophy Secondary to Age-Related Macular Degeneratio

Phase 1

Conditions: Geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
MedDRA version: 20.1Level: PTClassification code 10064930Term: Age-related macular degenerationSystem Organ Class: 10015919 - Eye disorders
MedDRA version: 20.1Level: LLTClassification code 10063947Term: Geographic atrophySystem Organ Class: 10015919 - Eye disorders
Therapeutic area: Diseases [C] - Eye Diseases [C11]

Registration Number: EUCTR2020-005174-94-CZ

Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 330

Inclusion Criteria

1. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal.
2. Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at least 2 weeks prior to first dose of investigational product
3. Well-demarcated geographic atrophy (GA) due to AMD
4. Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart
5. Must have clear ocular media and adequate pupillary dilation in the study eye to permit high-quality fundus imaging

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 25
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 330

Exclusion Criteria

1.Clinically-significant abnormalities in medical history
2. A lack of full recovery from any infection for at least 14 days prior to the Study Drug administration
3. Chronic treatment with steroids, including topically or intravitreally administered
4. History or presence of diabetic retinopathy or diabetic macular edema (DME)
5. History or presence of a disease other than AMD that could affect vision or safety assessments
6. Prior treatment with another investigational drug, biological agent, or device
7. Other protocol-specified inclusion/exclusion criteria may apply

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effect of ISIS 696844 on the rate of change of the area of Geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF);Secondary Objective: To evaluate the effect of ISIS 696844 on plasma FB levels and serum AH50 activity in AMD patients<br><br>To assess the effect of ISIS 696844 to slow reduction of low luminance visual acuity (LLVA)<br>;Primary end point(s): The primary efficacy endpoint is the absolute change in the GA area at Week 49 compared to baseline, as measured by FAF in study eye.;Timepoint(s) of evaluation of this end point: Week 49

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): The secondary efficacy endpoints include:<br>• Percent change in levels of plasma FB from Baseline<br>• Percent change in levels of serum AH50 activity from Baseline<br>• Absolute change in LLVA score from Baseline at Week 49<br>;Timepoint(s) of evaluation of this end point: Please, refer to Appendix A in the protocol for a detailed schedule of procedures

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