Geographic AtrophyPhase 1NCT02130531
Pharmacokinetic and Pharmacodynamic Study of Emixustat in Subjects With Geographic Atrophy Associated With Dry Age-Related Macular Degeneration
A Phase 1 study testing Emixustat HCl Tablet in people with Geographic Atrophy and Age-Related Macular Degeneration, run by Kubota Vision Inc. at 1 site in United States.
Trial summary
The purpose of this study is to characterize emixustat hydrochloride pharmacokinetic and pharmacodynamic parameters in subjects with geographic atrophy associated with dry age-related macular degeneration. Read more about this study
- Who is it for?
- Ages 65 years and older
- Conditions the study is for
- Geographic Atrophy, Age-Related Macular Degeneration
- What is being tested?
- Emixustat HCl Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2014to Nov 2015
- Phase
- Phase 1
- 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 65 years and older
- All sexes
- No healthy volunteers
- Older adult
Who may be able to join
- Males or females age ≥65 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
- Geographic atrophy associated with a condition other than AMD
- History of, active or high risk of developing choroidal neovascularization (CNV) in either eye
- 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
- Prior participation in any clinical study of emixustat
- History of other disease, metabolic dysfunction, chronic immunosuppression, physical examination finding
- Male subjects who are not surgically sterile and are not willing to practice a medically accepted method of birth control with their female partner of childbearing potential from screening through 21 days after the last dose of study drug
Trial locations and contacts
1 site in 1 country.
United States1 site
Call Acucela Clinical Trials Helpdesk for locations
Seattle, Washington, 98101
About this study
Official title: Pharmacokinetic and Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Geographic Atrophy Associated With Dry Age-Related Macular Degeneration
The purpose of this study is to characterize emixustat hydrochloride pharmacokinetic and pharmacodynamic parameters in subjects with geographic atrophy associated with dry age-related macular degeneration.
Read the detailed description
This is a multi-center, randomized, masked, multiple-dose, crossover study of the pharmacokinetics of emixustat hydrochloride (HCl) in subjects with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD). Subjects will receive study drug during multiple dose periods in a crossover design, and serve as their own controls. There is a 21-28 day washout period between dose periods. The final Study Exit visit is 21 to 28 days after the subject's last dose of study drug at the end of the final dosing period.
- Being tested
- Emixustat HCl Tablet, Placebo Tablet
- 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.
Emixustat HCl TabletDrug
Also called ACU-4429
Tablet for oral administration
Placebo TabletOther
Tablet for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | Two dose periods in any sequence (subjects will be assigned to 1 of 2 possible sequences):
* Emixustat HCl Tablet Strength B (mid dose); 1 tablet once daily \& 1 placebo tablet once daily for 7 days
* Emixustat HCl Tablet Strength A (low dose); 1 tablet twice daily for 7 days Receives: Drug: Emixustat HCl Tablet; Other: Placebo Tablet |
| Cohort 2 | Experimental | Three dose periods in any sequence (subjects will be assigned to 1 of 6 possible sequences):
* Emixustat HCl Tablet Strength A (low dose); 1 tablet daily for 7 days
* Emixustat HCl Tablet Strength B (mid dose); 1 tablet daily for 7 days
* Emixustat HCl Tablet Strength C (high dose); 1 tablet daily for 7 days Receives: Drug: Emixustat HCl Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Area Under the Concentration-Time Curve from time zero to 24 hours [(AUC(0-24h)]
Measured: Day 1 and Day 7 of each dosing period
Degree of suppression of the ERG b-wave response post-photobleaching.
Measured: Screening through Day 10 of each dosing period and study exit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2014 (estimated)
- Study startApr 2014
- Primary completionSep 2015
- Study completionNov 2015
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.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kubota Vision Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Acucela Medical Monitor, Study director, Kubota Vision Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02130531
- Sponsor's protocol ID
- 4429-101
- Status last verified
- Jan 2016
- First submitted
- 1 May 2014
- Last updated
- 18 Jan 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Jan 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pharmacokinetic and Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Geographic Atrophy Associated With Dry Age-Related Macular Degeneration
- ClinicalTrials.gov ID
- NCT02130531
- Acronym / sponsor ID
- 4429-101
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Kubota Vision Inc.Industry (pharma, biotech, devices)
- Study start
- Apr 2014
- Primary completion
- Sep 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 5 May 2014 (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 characterize emixustat hydrochloride pharmacokinetic and pharmacodynamic parameters in subjects with geographic atrophy associated with dry age-related macular degeneration.
Detailed description
This is a multi-center, randomized, masked, multiple-dose, crossover study of the pharmacokinetics of emixustat hydrochloride (HCl) in subjects with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD). Subjects will receive study drug during multiple dose periods in a crossover design, and serve as their own controls. There is a 21-28 day washout period between dose periods. The final Study Exit visit is 21 to 28 days after the subject's last dose of study drug at the end of the final dosing period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: Emixustat HCl Tablet, Other: Placebo TabletTwo dose periods in any sequence (subjects will be assigned to 1 of 2 possible sequences): * Emixustat HCl Tablet Strength B (mid dose); 1 tablet once daily \& 1 placebo tablet once daily for 7 days * Emixustat HCl Tablet Strength A (low dose); 1 tablet twice daily for 7 days |
| Cohort 2 | Experimental | Drug: Emixustat HCl TabletThree dose periods in any sequence (subjects will be assigned to 1 of 6 possible sequences): * Emixustat HCl Tablet Strength A (low dose); 1 tablet daily for 7 days * Emixustat HCl Tablet Strength B (mid dose); 1 tablet daily for 7 days * Emixustat HCl Tablet Strength C (high dose); 1 tablet daily for 7 days |
Who may qualify
- Ages
- 65 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Older adult
Key inclusion criteria (4 listed)
- Males or females age ≥65 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)
- Geographic atrophy associated with a condition other than AMD
- History of, active or high risk of developing choroidal neovascularization (CNV) in either eye
- 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
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 |
|---|---|---|
| Emixustat HCl TabletAlso: ACU-4429Tablet for oral administration | Drug | Cohort 1, Cohort 2 |
| Placebo TabletTablet for oral administration | Other | Cohort 1 |
Primary outcome
Area Under the Concentration-Time Curve from time zero to 24 hours [(AUC(0-24h)]
Time frame: Day 1 and Day 7 of each dosing period
Degree of suppression of the ERG b-wave response post-photobleaching.
Time frame: Screening through Day 10 of each dosing period and study exit
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 18 Jan 2016.
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
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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 1 study testing Emixustat HCl Tablet in people with Geographic Atrophy and Age-Related Macular Degeneration, 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 18 Jan 2016. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Area Under the Concentration-Time Curve from time zero to 24 hours [(AUC(0-24h)] (time frame: Day 1 and Day 7 of each dosing period). The study lists 2 primary outcomes in all. 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 18 Jan 2016.