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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03446144




Registration number
NCT03446144
Ethics application status
Date submitted
20/02/2018
Date registered
26/02/2018
Date last updated
13/08/2019

Titles & IDs
Public title
Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 and Older With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)
Scientific title
A Phase 2, Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of ISIS 696844, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary (GA) to Age-Related Macular Degeneration (AMD)
Secondary ID [1] 0 0
ISIS 696844-CS3
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Geographic Atrophy 0 0
Age Related Macular Degeneration 0 0
Condition category
Condition code
Eye 0 0 0 0
Diseases / disorders of the eye
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - IONIS-FB-Lrx
Treatment: Drugs - Placebo (sterline saline 0.9%)

Experimental: IONIS-FB-Lrx -

Placebo Comparator: Placebo (sterile saline 0.9%) -


Treatment: Drugs: IONIS-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

Treatment: Drugs: Placebo (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

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Efficacy of IONIS-FB-Lrx - The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level
Timepoint [1] 0 0
Up to 74 weeks
Secondary outcome [1] 0 0
Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx - 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
Timepoint [1] 0 0
Up to 86 weeks
Secondary outcome [2] 0 0
Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B - Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B from Baseline to Post-Treatment
Timepoint [2] 0 0
Up to 74 weeks
Secondary outcome [3] 0 0
Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD - 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
Timepoint [3] 0 0
Up to 74 weeks
Secondary outcome [4] 0 0
Effect of factor B reduction on other components of the complement pathways in AMD patients - 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
Timepoint [4] 0 0
Up to 74 weeks

Eligibility
Key inclusion criteria
Key

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
Minimum age
55 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Withdrawn
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC,WA
Recruitment hospital [1] 0 0
Eye Clinic Albury Wodonga - Albury
Recruitment hospital [2] 0 0
IONIS Investigational Site - Liverpool
Recruitment hospital [3] 0 0
Marsden Eye Specialists - Paramatta
Recruitment hospital [4] 0 0
Strathfield Retina Clinic - Strathfield
Recruitment hospital [5] 0 0
Sydney Eye Hospital - Sydney
Recruitment hospital [6] 0 0
Sydney Retina Clinic Day Surgery - Sydney
Recruitment hospital [7] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [8] 0 0
The Royal Victorian Eye and Ear Hospital - East Melbourne
Recruitment hospital [9] 0 0
Retinology Institute - Glen Iris
Recruitment hospital [10] 0 0
Eye Surgery Associates - Malvern
Recruitment hospital [11] 0 0
Eye Surgery Associates - Parkville
Recruitment hospital [12] 0 0
Lions Eye Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2640 - Albury
Recruitment postcode(s) [2] 0 0
2170 - Liverpool
Recruitment postcode(s) [3] 0 0
2150 - Paramatta
Recruitment postcode(s) [4] 0 0
2135 - Strathfield
Recruitment postcode(s) [5] 0 0
2000 - Sydney
Recruitment postcode(s) [6] 0 0
5000 - Adelaide
Recruitment postcode(s) [7] 0 0
3002 - East Melbourne
Recruitment postcode(s) [8] 0 0
3146 - Glen Iris
Recruitment postcode(s) [9] 0 0
3144 - Malvern
Recruitment postcode(s) [10] 0 0
3050 - Parkville
Recruitment postcode(s) [11] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New York
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
South Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
South Dakota
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
New Zealand
State/province [12] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ionis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/show/NCT03446144
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03446144