Age Related Macular Degeneration, Geographic Atrophy
Conditions
Keywords
Geographic Atrophy, Age Related Macular Degeneration, IONIS-FB-Lrx, Complement Factor B
Brief summary
The purpose of this study is to assess the Safety and Efficacy of IONIS-FB-Lrx for up to 120 patients with Geographic Atrophy secondary to Age Related Macular Degeneration
Detailed description
This study will assess changes in complement factor B over a 69-week treatment period in a patient population 55 and older with well-demarcated Geographic Atrophy secondary to Age Related Macular Degeneration
Interventions
DRUGIONIS-FB-Lrx
Single Dose of IONIS-FB-Lrx administered once weekly for weeks 1-3, and every other week or less frequently until week 70
DRUGPlacebo (sterline saline 0.9%)
Calculated volume to match active comparator. Administered subcutaneously weekly for weeks 1-3, and every other week or less frequently until week 70
Sponsors
Ionis Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Masking description
Double Masked
Eligibility
Sex/Gender
ALL
Age
55 Years to No maximum
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Must have given written informed consent and be able to comply with study requirements 2. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal. Males must be surgically sterile or, if engaged in sexual relations with a female of child bearing potential, the subject must be using an acceptable contraceptive method from the time of signing the informed consent form until at least a period of 13 weeks after the last dose of Study Drug (ISIS 696844 or placebo) 3. Well-demarcated Geographic Atrophy due to Age Related Macular Degeneration Key
Exclusion criteria
1. Clinically-significant abnormalities in medical history 2. Diagnosis of primary or secondary immunodeficiencies of B lymphocyte function, splenectomy, glomerulonephritis or history of recurrent meningococcal disease 3. Diabetes mellitus or thyroid disease unless well controlled for a period of at least 3 months 4. Clinically-significant abnormalities in screening laboratory values 5. Unwillingness to be administered, or history of a serious reaction to protocol required vaccines 6. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B 7. History or presence of a disease other than AMD in study eye
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy of IONIS-FB-Lrx | Up to 74 weeks | The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx | Up to 86 weeks | The safety and tolerability of IONIS-FB-Lrx will be assessed by determining the incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx |
| Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B | Up to 74 weeks | Proportion of patients in each treatment group achieving a \>50% reduction of plasma complement factor B from Baseline to Post-Treatment |
| Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD | Up to 74 weeks | Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD as measured by Fundus Autofluorescence from Baseline to Post-Treatment |
| Effect of factor B reduction on other components of the complement pathways in AMD patients | Up to 74 weeks | Effect of factor B reduction on other components of the complement pathways such as AH50, CH50 and Bb in AMD patients from Baseline to Post-Treatment |
Countries
Australia, New Zealand, United States
Outcome results
None listed