NCT02402660
Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease
Enrolling by Invitation
PHASE2Ages 8–70InterventionalTreatmentAlkeus Pharmaceuticals, Inc.
~160 participants
Updated 2025-04-27 on ClinicalTrials.gov
What's tested:ALK-001Placebo
At a glance
Recruiting sites
0 of 14 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Safety and tolerability of 24 months of daily dosing of ALK-001 assessed by incidence and/or clinically significant changes of a combination of ocular and non-ocular adverse events
Measured over From baseline to 24 months
Conditions
Where it's being run
14 sites across 12 statesFlorida2
New York2
Arizona1
California1
Colorado1
Indiana1
Maryland1
Michigan1
Who to contact
This trial hasn't published a contact. View it on ClinicalTrials.gov
Do you actually qualify for this trial?
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Eligibility criteria
Inclusion
Male or female between 8 and 70 years old (inclusive), with any visual acuity
Has a clinical diagnosis of typical autosomal recessive Stargardt macular dystrophy (STGD1)
Has provided a genetic report indicating at least two ABCA4 disease-causing mutations. When only one ABCA4 disease-causing mutation is reported, sponsor's permission will be required.
At least one eye (called the "primary study eye") must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF), have decreased retinal sensitivity as measured by microperimetry, or have maculopathy expected to progress over the duration of the study
Primary study eye must have clear ocular media and adequate pupillary dilation, including no allergy to dilating eyedrops, to permit good quality retinal imaging
Healthy as judged by investigator
Able and willing to comply with study requirements, restrictions and instructions and is likely to complete the 24-month study
Has signed and dated the informed consent forms (or assent where appropriate) to participate
Female of childbearing potential has signed the informed consent about birth defects or attestation on contraception requirements
Exclusion
Has taken disallowed items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) over the past 30 days
Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization
Has concurrent medical condition or history, which in the opinion of the investigator, is likely to prevent compliance with the protocol and/or interfere with absorption of ALK-001 or study procedures
Has clinically significant abnormal laboratory result(s) at screening
Has active or historical acute or chronic liver disorder
Has active or historical ocular disorder in the primary study eye that, in the opinion of the investigator, may confound assessment of the retina morphologically or functionally (this could include for example cataract surgery within the past 6 months, choroidal neovascularization (CNV), glaucoma, recurring uveitis, diabetic retinopathy, other retinal disease, etc.)
Has had intraocular surgery or injections in the primary study eye within 90 days of the screening visit
Has a clinically significant abnormal electrocardiogram (ECG), or has a corrected QT interval (QTc) that is 450 ms or greater
What this trial measures
- Safety and tolerability of 24 months of daily dosing of ALK-001 assessed by incidence and/or clinically significant changes of a combination of ocular and non-ocular adverse eventsFrom baseline to 24 months