Geographic AtrophyPhase 2NCT03446144
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)
A Phase 2 study testing IONIS-FB-Lrx in people with Geographic Atrophy and Age Related Macular Degeneration, run by Ionis Pharmaceuticals, Inc. at 37 sites in 3 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 55 years and older
- Conditions the study is for
- Geographic Atrophy, Age Related Macular Degeneration
- What is being tested?
- IONIS-FB-Lrx
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 37 sitesin 3 countries
- Study dates
- 16 Mar 2018to 10 Oct 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 55 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must have given written informed consent and be able to comply with study requirements
- 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)
- Well-demarcated Geographic Atrophy due to Age Related Macular Degeneration
Who cannot join
- Clinically-significant abnormalities in medical history
- Diagnosis of primary or secondary immunodeficiencies of B lymphocyte function, splenectomy, glomerulonephritis or history of recurrent meningococcal disease
- Diabetes mellitus or thyroid disease unless well controlled for a period of at least 3 months
- Clinically-significant abnormalities in screening laboratory values
- Unwillingness to be administered, or history of a serious reaction to protocol required vaccines
- Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B
- History or presence of a disease other than AMD in study eye
Trial locations and contacts
37 sites in 3 countries.
37 sites in 3 countries
United States24 sites
IONIS Investigative Site
Arlington, Texas, 76012-2505
IONIS Investigational Site
Augusta, Georgia, 30909
IONIS Investigational Site
Austin, Texas, 78705
IONIS Investigational Site
Beverly Hills, California, 90211
IONIS Investigative Site
Chesterfield, Missouri, 63017
IONIS Investigative Site
Clearwater, Florida, 33761
IONIS Investigative Site
Dallas, Texas, 75231-5078
IONIS Investigative Site
Encino, California, 91436
IONIS Investigative Site
Houston, Texas, 77030-2727
IONIS Investigative Site
Irvine, California, 92697
IONIS Investigational Site
Ladson, South Carolina, 29456
IONIS Investigational Site
Leawood, Kansas, 66211
IONIS Investigative Site
McAllen, Texas, 78503-1518
IONIS Investigational Site
Mountain View, California, 94040
IONIS Investigative Site
New York, New York, 10021
IONIS Investigative Site
Philadelphia, Pennsylvania, 19104
IONIS Investigative Site
Philadelphia, Pennsylvania, 19107-5109
IONIS Investigative Site
Phoenix, Arizona, 85014-2709
IONIS Investigative Site
Pittsburgh, Pennsylvania, 15213
IONIS Investigative Site
Rapid City, South Dakota, 57701-7374
IONIS Investigative Site
San Antonio, Texas, 78240-1502
IONIS Investigational Site
Santa Barbara, California, 93103
IONIS Investigational Site
Tampa, Florida, 33612
IONIS Investigative Site
The Woodlands, Texas, 77384-8018
Australia12 sites
Royal Adelaide Hospital
Adelaide, South Australia, 5000
Eye Clinic Albury Wodonga
Albury, New South Wales, 2640
The Royal Victorian Eye and Ear Hospital
East Melbourne, Victoria, 3002
Retinology Institute
Glen Iris, Victoria, 3146
IONIS Investigational Site
Liverpool, New South Wales, 2170
Eye Surgery Associates
Malvern, Victoria, 3144
Lions Eye Institute
Nedlands, Western Australia, 6009
Eye Surgery Associates
Parkville, Victoria, 3050
Marsden Eye Specialists
Parramatta, New South Wales, 2150
Strathfield Retina Clinic
Strathfield, New South Wales, 2135
Sydney Eye Hospital
Sydney, New South Wales, 2000
Sydney Retina Clinic Day Surgery
Sydney, New South Wales, 2000
About this study
Official 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)
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
Read the 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
- Being tested
- IONIS-FB-Lrx, Placebo (sterline saline 0.9%)
- Keywords
- Geographic Atrophy, Age Related Macular Degeneration, IONIS-FB-Lrx, Complement Factor B
- Medical subject headings
- Geographic Atrophy, Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IONIS-FB-LrxDrug
Single Dose of IONIS-FB-Lrx administered once weekly for weeks 1-3, and every other week or less frequently until week 70
Placebo (sterline saline 0.9%)Drug
Calculated volume to match active comparator. Administered subcutaneously weekly for weeks 1-3, and every other week or less frequently until week 70
| Study group | Role | What they receive |
|---|---|---|
| IONIS-FB-Lrx | Experimental | Not described Receives: Drug: IONIS-FB-Lrx |
| Placebo (sterile saline 0.9%) | Placebo comparator | Not described Receives: Drug: Placebo (sterline saline 0.9%) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of IONIS-FB-Lrx
The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level
Measured: Up to 74 weeks
Secondary outcomes (4)
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
Measured: Up to 86 weeks
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
Measured: Up to 74 weeks
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
Measured: Up to 74 weeks
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
Measured: Up to 74 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2018
- Study start16 Mar 2018
- Primary completion10 Oct 2018
- Study completion10 Oct 2018
Why it stopped: Business objective change, no safety or efficacy concerns
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- More trials run by Ionis Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03446144
- Sponsor's protocol ID
- ISIS 696844-CS3
- Status last verified
- Aug 2019
- First submitted
- 20 Feb 2018
- Last updated
- 13 Aug 2019
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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)
- ClinicalTrials.gov ID
- NCT03446144
- Acronym / sponsor ID
- ISIS 696844-CS3
- Keywords
- Geographic Atrophy, Age Related Macular Degeneration, IONIS-FB-Lrx, Complement Factor B
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Business objective change, no safety or efficacy concerns
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Ionis Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 16 Mar 2018 (actual)
- Primary completion
- 10 Oct 2018 (actual)
- Study completion
- 10 Oct 2018 (actual)
- First posted
- 26 Feb 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IONIS-FB-Lrx | Experimental | Drug: IONIS-FB-Lrx |
| Placebo (sterile saline 0.9%) | Placebo comparator | Drug: Placebo (sterline saline 0.9%) |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Must have given written informed consent and be able to comply with study requirements
- 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)
- Well-demarcated Geographic Atrophy due to Age Related Macular Degeneration
Key exclusion criteria (7 listed)
- Clinically-significant abnormalities in medical history
- Diagnosis of primary or secondary immunodeficiencies of B lymphocyte function, splenectomy, glomerulonephritis or history of recurrent meningococcal disease
- Diabetes mellitus or thyroid disease unless well controlled for a period of at least 3 months
- Clinically-significant abnormalities in screening laboratory values
- Unwillingness to be administered, or history of a serious reaction to protocol required vaccines
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IONIS-FB-LrxSingle Dose of IONIS-FB-Lrx administered once weekly for weeks 1-3, and every other week or less frequently until week 70 | Drug | IONIS-FB-Lrx |
| 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 | Drug | Placebo (sterile saline 0.9%) |
Primary outcome
Efficacy of IONIS-FB-Lrx
Time frame: Up to 74 weeks
The Efficacy of IONIS-FB-Lrx will be measured by the percent change in plasma complement factor B level
4 secondary outcomes
Incidence and severity of adverse events that are related to treatment with IONIS-FB-Lrx
Time frame: Up to 86 weeks
Proportion of patients in each treatment group achieving a >50% reduction of plasma complement factor B
Time frame: Up to 74 weeks
Effect of IONIS-FB-Lrx on the rate of change of the area of GA secondary to AMD
Time frame: Up to 74 weeks
Effect of factor B reduction on other components of the complement pathways in AMD patients
Time frame: Up to 74 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2019.
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Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
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RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
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Phase 1 Study of C.001 in Retinal Degeneration
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RecruitingPhase 2/3
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
37 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IONIS-FB-Lrx in people with Geographic Atrophy and Age Related Macular Degeneration, run by Ionis Pharmaceuticals, Inc. at 37 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 13 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Australia, New Zealand. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Efficacy of IONIS-FB-Lrx (time frame: Up to 74 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2019.