Geographic AtrophyPhase 2/3SEATTLENCT01802866
Safety and Efficacy Assessment Treatment Trials of Emixustat Hydrochloride
A Phase 2/3 study testing ACU-4429 in people with Geographic Atrophy, run by Kubota Vision Inc. at 1 site in United States.
Trial summary
The purpose of this study is to determine if emixustat hydrochloride (ACU-4429) reduces the rate of progression of geographic atrophy compared to placebo in subjects with dry 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
- What is being tested?
- ACU-4429
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 508 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2013to May 2016
- Phase
- Phase 2/3
- 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
- Males or females, age ≥55 years.
- Clinical diagnosis of GA associated with AMD
- Able and willing to provide written informed consent.
- Able to reliably administer oral medication by self or with available assistance.
Who cannot join
- Active CNV or presence of an active ocular disease.
- Known serious allergy to the fluorescein sodium for injection in angiography.
- Pre-specified laboratory abnormalities at screening.
- Treatment with any investigational study drug within 30 days of screening or device (within 60 days of screening)
- History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding
- Female subjects who are pregnant or lactating.
- Female subjects of childbearing potential and male subjects who are not surgically sterile who are not willing to practice a medically accepted method of birth control from screening through 30 days after completion of the study.
- Unstable or poorly controlled medical or ophthalmic conditions
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2b/3 Multicenter, Randomized, Double-Masked, Dose-Ranging Study Comparing the Efficacy and Safety of Emixustat Hydrochloride (ACU-4429) With Placebo for the Treatment of Geographic Atrophy Associated With Dry Age-Related Macular Degeneration
The purpose of this study is to determine if emixustat hydrochloride (ACU-4429) reduces the rate of progression of geographic atrophy compared to placebo in subjects with dry age-related macular degeneration.
- Conditions
- Geographic Atrophy
- Being tested
- ACU-4429, Placebo
- Keywords
- Geographic atrophy, GA, Dry AMD
- Medical subject headings
- Geographic Atrophy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACU-4429Drug
Also called emixustat hydrochloride
Take orally once daily for 24 months
PlaceboDrug
Take orally once daily for 24 months
| Study group | Role | What they receive |
|---|---|---|
| ACU-4429 2.5 mg | Experimental | 2.5 mg tablet Receives: Drug: ACU-4429 |
| ACU-4429 5 mg | Experimental | 5 mg tablet Receives: Drug: ACU-4429 |
| ACU-4429 10 mg | Experimental | 10 mg tablet Receives: Drug: ACU-4429 |
| Placebo | Placebo comparator | Includes identical tablets with only inactive ingredients (0 mg). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in the total area of the GA lesion(s)
Measured: 24 months
Secondary outcomes (2)
Change from baseline in BCVA score
Measured: 24 months
Frequency of AEs, discontinuations due to AEs, or dose modifications; severity and seriousness of AEs
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2013 (estimated)
- Study startFeb 2013
- Primary completionApr 2016
- Study completionMay 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Geographic AtrophyStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 508 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kubota Vision Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Otsuka Pharmaceutical Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Acucela Medical Monitor, Study director, Kubota Vision Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01802866
- Sponsor's protocol ID
- 4429-202
- Acronym
- SEATTLE
- Status last verified
- Jun 2017
- First submitted
- 28 Feb 2013
- Last updated
- 2 Jul 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
Yeong JL, Loveman E, Colquitt JL, Royle P, Waugh N, Lois N. Visual cycle modulators versus placebo or observation for the prevention and treatment of geographic atrophy due to age-related macular degeneration. Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD013154. doi: 10.1002/14651858.CD013154.pub2.
PubMed 33331670Rosenfeld PJ, Dugel PU, Holz FG, Heier JS, Pearlman JA, Novack RL, Csaky KG, Koester JM, Gregory JK, Kubota R. Emixustat Hydrochloride for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Clinical Trial. Ophthalmology. 2018 Oct;125(10):1556-1567. doi: 10.1016/j.ophtha.2018.03.059. Epub 2018 Apr 30.
PubMed 29716784
Study at a glance
From the registry record, last updated by the sponsor on 2 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2b/3 Multicenter, Randomized, Double-Masked, Dose-Ranging Study Comparing the Efficacy and Safety of Emixustat Hydrochloride (ACU-4429) With Placebo for the Treatment of Geographic Atrophy Associated With Dry Age-Related Macular Degeneration
- ClinicalTrials.gov ID
- NCT01802866
- Acronym / sponsor ID
- SEATTLE / 4429-202
- Conditions
- Geographic Atrophy
- Keywords
- Geographic atrophy, GA, Dry AMD
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 508 participants (actual)
- Lead sponsor
- Kubota Vision Inc.Industry (pharma, biotech, devices)
- Collaborators
- Otsuka Pharmaceutical Co., Ltd.
- Study start
- Feb 2013
- Primary completion
- Apr 2016 (actual)
- Study completion
- May 2016 (actual)
- First posted
- 4 Mar 2013 (estimated)
- 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 determine if emixustat hydrochloride (ACU-4429) reduces the rate of progression of geographic atrophy compared to placebo in subjects with dry age-related macular degeneration.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| ACU-4429 2.5 mg | Experimental | Drug: ACU-44292.5 mg tablet |
| ACU-4429 5 mg | Experimental | Drug: ACU-44295 mg tablet |
| ACU-4429 10 mg | Experimental | Drug: ACU-442910 mg tablet |
| Placebo | Placebo comparator | Drug: PlaceboIncludes identical tablets with only inactive ingredients (0 mg). |
Who may qualify
- Ages
- 55 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Males or females, age ≥55 years.
- Clinical diagnosis of GA associated with AMD
- Able and willing to provide written informed consent.
- Able to reliably administer oral medication by self or with available assistance.
Key exclusion criteria (8 listed)
- Active CNV or presence of an active ocular disease.
- Known serious allergy to the fluorescein sodium for injection in angiography.
- Pre-specified laboratory abnormalities at screening.
- Treatment with any investigational study drug within 30 days of screening or device (within 60 days of screening)
- History of other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding
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 |
|---|---|---|
| ACU-4429Also: emixustat hydrochlorideTake orally once daily for 24 months | Drug | ACU-4429 10 mg, ACU-4429 2.5 mg, ACU-4429 5 mg |
| PlaceboTake orally once daily for 24 months | Drug | Placebo |
Primary outcome
Change from baseline in the total area of the GA lesion(s)
Time frame: 24 months
2 secondary outcomes
Change from baseline in BCVA score
Time frame: 24 months
Frequency of AEs, discontinuations due to AEs, or dose modifications; severity and seriousness of AEs
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Jul 2017.
- Derived from this study: PubMed 33331670Yeong JL, Loveman E, Colquitt JL, Royle P, Waugh N, Lois N. Visual cycle modulators versus placebo or observation for the prevention and treatment of geographic atrophy due to age-related macular degeneration. Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD013154. doi: 10.1002/14651858.CD013154.pub2.
- Derived from this study: PubMed 29716784Rosenfeld PJ, Dugel PU, Holz FG, Heier JS, Pearlman JA, Novack RL, Csaky KG, Koester JM, Gregory JK, Kubota R. Emixustat Hydrochloride for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Clinical Trial. Ophthalmology. 2018 Oct;125(10):1556-1567. doi: 10.1016/j.ophtha.2018.03.059. Epub 2018 Apr 30.
- Individual participant dataSharing planned: Undecided
Related trials for Geographic Atrophy
Active, not recruitingPhase 1/2
Phase 1/2 Study of VOY-101 in Patients With Advanced Non-Neovascular Age-Related Macular Degeneration
Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 1/2
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration
Dry Age-Related Macular Degeneration, Geographic Atrophy
RecruitingPhase 3
A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy
Age-related Macular Degeneration (AMD), Geographic Atrophy (GA)
RecruitingPhase 1
Phase 1 Study of C.001 in Retinal Degeneration
Geographic Atrophy, Stargardt Disease
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)
Geographic Atrophy, Macular Degeneration
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.
Acucela Medical Monitor study director
Kubota Vision Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing ACU-4429 in people with Geographic Atrophy, run by Kubota Vision Inc. at 1 site in United States.
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 4 inclusion and 8 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: finished, as of the sponsor's last update on 2 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Change from baseline in the total area of the GA lesion(s) (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01802866, which also lists 2 linked publications.
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 2 Jul 2017.