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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03815825




Registration number
NCT03815825
Ethics application status
Date submitted
22/01/2019
Date registered
24/01/2019
Date last updated
29/07/2019

Titles & IDs
Public title
A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Scientific title
A Phase 2, Randomized Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Secondary ID [1] 0 0
ISIS 696844-CS5
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Macular Degeneration 0 0
Geographic Atrophy 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

Experimental: IONIS-FB-LRx - Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.

Experimental: Placebo - Stage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.


Treatment: Drugs: IONIS-FB-LRx
IONIS-FB-LRx at multiple ascending doses, administered subcutaneously every 4 weeks

Treatment: Drugs: Placebo
Placebo matching solution, administered subcutaneously every 4 weeks

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

Outcomes
Primary outcome [1] 0 0
Absolute Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
Timepoint [1] 0 0
Week 49
Secondary outcome [1] 0 0
Percentage Change from Baseline in Levels of Factor B (FB) in Plasma
Timepoint [1] 0 0
Baseline and up to Week 49
Secondary outcome [2] 0 0
Percentage Change from Baseline in Levels of Serum AH50 Activity
Timepoint [2] 0 0
Baseline and up to Week 49
Secondary outcome [3] 0 0
Absolute Change from Baseline in Low Luminance Visual Acuity (LLVA)
Timepoint [3] 0 0
Baseline and up to Week 49

Eligibility
Key inclusion criteria
- Females must be non-pregnant and non-lactating, and either surgically sterile or
post-menopausal.

- Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at
least 2 weeks prior to first dose of investigational product

- Well-demarcated geographic atrophy (GA) due to AMD

- Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen
equivalent) or better on the ETDRS chart

- Must have clear ocular media and adequate pupillary dilation in the study eye to
permit high-quality fundus imaging
Minimum age
50 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Clinically-significant abnormalities in medical history

- A lack of full recovery from any infection for at least 14 days prior to the Study
Drug administration

- Chronic treatment with steroids, including topically or intravitreally administered

- History or presence of diabetic retinopathy or diabetic macular edema (DME)

- History or presence of a disease other than AMD that could affect vision or safety
assessments

- Prior treatment with another investigational drug, biological agent, or device

- Other protocol-specified inclusion/exclusion criteria may apply

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

Recruitment
Recruitment status
Recruiting
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
IONIS Investigative Site - Albury
Recruitment hospital [2] 0 0
IONIS Investigative Site - Parramatta
Recruitment hospital [3] 0 0
IONIS Investigative Site - Sydney
Recruitment hospital [4] 0 0
IONIS Investigative Site - Adelaide
Recruitment hospital [5] 0 0
IONIS Investigative Site - East Melbourne
Recruitment hospital [6] 0 0
IONIS Investigative Site - Essendon
Recruitment hospital [7] 0 0
IONIS Investigative Site - Glen Iris
Recruitment hospital [8] 0 0
IONIS Investigative Site - Malvern
Recruitment hospital [9] 0 0
IONIS Investigative Site - Parkville
Recruitment hospital [10] 0 0
IONIS Investigative Site - Nedlands
Recruitment postcode(s) [1] 0 0
2640 - Albury
Recruitment postcode(s) [2] 0 0
2150 - Parramatta
Recruitment postcode(s) [3] 0 0
2000 - Sydney
Recruitment postcode(s) [4] 0 0
5000 - Adelaide
Recruitment postcode(s) [5] 0 0
3002 - East Melbourne
Recruitment postcode(s) [6] 0 0
3040 - Essendon
Recruitment postcode(s) [7] 0 0
3146 - Glen Iris
Recruitment postcode(s) [8] 0 0
3144 - Malvern
Recruitment postcode(s) [9] 0 0
3050 - Parkville
Recruitment postcode(s) [10] 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
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Kansas
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
Nevada
Country [11] 0 0
United States of America
State/province [11] 0 0
New Jersey
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Oregon
Country [15] 0 0
United States of America
State/province [15] 0 0
Pennsylvania
Country [16] 0 0
United States of America
State/province [16] 0 0
South Carolina
Country [17] 0 0
United States of America
State/province [17] 0 0
Texas
Country [18] 0 0
United States of America
State/province [18] 0 0
Utah
Country [19] 0 0
United States of America
State/province [19] 0 0
Virginia
Country [20] 0 0
United States of America
State/province [20] 0 0
Wisconsin
Country [21] 0 0
New Zealand
State/province [21] 0 0
Christchurch
Country [22] 0 0
New Zealand
State/province [22] 0 0
Remuera

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 the study is to evaluate the effect of IONIS-FB-LRx on the rate of change of
the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
measured by fundus autofluorescence (FAF).
Trial website
https://clinicaltrials.gov/show/NCT03815825
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
Ionis Pharmaceuticals
Address 0 0
Country 0 0
Phone 0 0
800-679-4747
Fax 0 0
Email 0 0
patients@ionisph.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03815825