Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 5 February 2024
• Main ID: NCT02402660
• Date of registration: 09/03/2015
• Prospective Registration: Yes
• Primary sponsor: Alkeus Pharmaceuticals, Inc.
• Public title: Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease TEASE
• Scientific title: A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease
• Date of first enrolment: August 2015
• Target sample size: 140
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02402660
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Leonide Saad, PhD
• Affiliation: Alkeus Pharmaceuticals, Inc.

• Name: Leonide Saad, PhD

• Name: Hendrik Scholl, MD
• Affiliation: University of Basel

Key inclusion & exclusion criteria

Simplified Inclusion Criteria:

- 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

Main Exclusion Criteria:

- 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



Age minimum: 8 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Stargardt Disease

Stargardt Macular Degeneration

Stargardt Macular Dystrophy

Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

Intervention(s)

Drug: Placebo

Drug: ALK-001

Primary Outcome(s)

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 [Time Frame: From baseline to 24 months]

Secondary Outcome(s)

Pharmacokinetic profile of ALK-001 derived from the concentrations of ALK-001 and metabolites in plasma [Time Frame: Up to 24 months]

Effects of ALK-001 on the progression of Stargardt disease [Time Frame: From baseline to 24 months]

Secondary ID(s)

R01FD004098

ALK001-P1002

R01FD006016

Source(s) of Monetary Support

Please refer to primary and secondary sponsors