Stargardt DiseasePhase 2NCT03033108
Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
A Phase 2 study testing Emixustat in people with Stargardt Disease and Macular Atrophy, run by Kubota Vision Inc. at 1 site in United States.
Trial summary
This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Stargardt Disease, Macular Atrophy
- What is being tested?
- Emixustat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2017to Dec 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of macular atrophy (MA) secondary to Stargardt disease (STGD) in one or both eyes
- At least 2 pathogenic mutations of the ABCA4 gene
- Early Treatment Diabetic Retinopathy Study BCVA of ≥ 20 letters (approximately ≥ 20/400 Snellen) in the study eye
- Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of MA in the study eye
- Able and willing to provide written informed consent before undergoing any study-related procedures
- Able to reliably administer oral medication by self or with available assistance
Who cannot join
- Macular atrophy associated with a condition other than STGD in either eye.
- Presence in either eye of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function.
- History of any intraocular or ocular surface surgery in either eye within 3 months of screening.
- Current or previous participation in an interventional study to treat STGD using gene therapy or stem cell therapy at any time, or participation in an interventional study of a vitamin A derivative ≤3 months prior to screening.
- Pre-specified laboratory abnormalities at screening
- Presence of other medical or ophthalmic disease, physical examination finding, or clinical laboratory finding that in the opinion of the Investigator may contraindicate the use of an investigational drug and place the subject at risk
Show all 12Show fewer
- Current or history of cancer (except for adequately treated basal cell or squamous cell carcinoma of the skin) within 1 year of screening
- History of myocardial infarction, stroke, unstable ischemic heart disease, uncontrolled cardiac arrhythmia, or hospitalization for congestive heart failure within 6 months of screening.
- Anticipated hospitalization for a medical/surgical procedure(s) that could result in interruption/premature cessation of study treatment or participation.
- Electrocardiogram with a clinically significant abnormal finding
- Female subjects who are pregnant or lactating
- Female subjects of childbearing potential or male subjects who are not surgically sterile who are not willing to practice a medically accepted method of birth control with their sexual partner from screening through 30 days after the final dose of study drug
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.
Read the detailed description
This is a multicenter, randomized, masked study to characterize the pharmacodynamics, safety and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt disease.
- Conditions
- Stargardt Disease, Macular Atrophy
- Being tested
- Emixustat
- Medical subject headings
- Stargardt Disease, Anetoderma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EmixustatDrug
Also called emixustat hydrochloride
Once daily, tablet for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Emixustat Dose 1 | Experimental | lowest dose of once-daily oral emixustat Receives: Drug: Emixustat |
| Emixustat Dose 2 | Experimental | middle dose of once-daily oral emixustat Receives: Drug: Emixustat |
| Emixustat Dose 3 | Experimental | highest dose of once-daily oral emixustat Receives: Drug: Emixustat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
Measured: Baseline and 1 month
Secondary outcomes (1)
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Assessment of safety profile
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jan 2017 (estimated)
- Study startJan 2017
- Primary completionNov 2017
- Study completionDec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Stargardt DiseaseStudies 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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kubota Vision Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Acucela Medical Director, MD, Study director, Kubota Vision Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03033108
- Sponsor's protocol ID
- 4429-204
- Status last verified
- Apr 2021
- First submitted
- 20 Jan 2017
- Last updated
- 19 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Publications
Kubota R, Birch DG, Gregory JK, Koester JM. Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. Br J Ophthalmol. 2022 Mar;106(3):403-408. doi: 10.1136/bjophthalmol-2020-317712. Epub 2020 Nov 19.
PubMed 33214244
Study at a glance
From the registry record, last updated by the sponsor on 19 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease
- ClinicalTrials.gov ID
- NCT03033108
- Acronym / sponsor ID
- 4429-204
- Conditions
- Stargardt Disease, Macular Atrophy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Kubota Vision Inc.Industry (pharma, biotech, devices)
- Study start
- Jan 2017 (actual)
- Primary completion
- Nov 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 26 Jan 2017 (estimated)
- Results
- Posted 27 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a pharmacodynamics study of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease.
Detailed description
This is a multicenter, randomized, masked study to characterize the pharmacodynamics, safety and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Emixustat Dose 1 | Experimental | Drug: Emixustatlowest dose of once-daily oral emixustat |
| Emixustat Dose 2 | Experimental | Drug: Emixustatmiddle dose of once-daily oral emixustat |
| Emixustat Dose 3 | Experimental | Drug: Emixustathighest dose of once-daily oral emixustat |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Clinical diagnosis of macular atrophy (MA) secondary to Stargardt disease (STGD) in one or both eyes
- At least 2 pathogenic mutations of the ABCA4 gene
- Early Treatment Diabetic Retinopathy Study BCVA of ≥ 20 letters (approximately ≥ 20/400 Snellen) in the study eye
- Adequate clarity of ocular media and adequate pupillary dilation to permit good quality imaging of MA in the study eye
- Able and willing to provide written informed consent before undergoing any study-related procedures
Key exclusion criteria (12 listed)
- Macular atrophy associated with a condition other than STGD in either eye.
- Presence in either eye of an active ocular disease that in the opinion of the Investigator compromises or confounds visual function.
- History of any intraocular or ocular surface surgery in either eye within 3 months of screening.
- Current or previous participation in an interventional study to treat STGD using gene therapy or stem cell therapy at any time, or participation in an interventional study of a vitamin A derivative ≤3 months prior to screening.
- Pre-specified laboratory abnormalities at 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 |
|---|---|---|
| EmixustatAlso: emixustat hydrochlorideOnce daily, tablet for oral administration | Drug | Emixustat Dose 1, Emixustat Dose 2, Emixustat Dose 3 |
Primary outcome
Change in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram
Time frame: Baseline and 1 month
Percent suppression compared to baseline of rod b-wave amplitude recovery after a photobleaching light.
1 secondary outcome
Percentage of Subjects With Adverse Events, by Severity and Seriousness
Time frame: 1 month
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33214244Kubota R, Birch DG, Gregory JK, Koester JM. Randomised study evaluating the pharmacodynamics of emixustat hydrochloride in subjects with macular atrophy secondary to Stargardt disease. Br J Ophthalmol. 2022 Mar;106(3):403-408. doi: 10.1136/bjophthalmol-2020-317712. Epub 2020 Nov 19.
- Individual participant dataSharing planned: No
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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.
Acucela Medical Director, MD 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 study testing Emixustat in people with Stargardt Disease and Macular 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 6 inclusion and 12 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 19 May 2021. 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 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?
Yes. Results are posted in the registry (first posted 27 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 in Electrical Response of the Retina to a Flash of Light, as Measured by Electroretinogram (time frame: Baseline and 1 month). 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 NCT03033108, which also lists 1 linked publication.
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 19 May 2021.