CompletedPhase 2

Study of STG-001 in Subjects With Stargardt Disease

United States10 participantsStarted 2020-10-01

Plain-language summary

This is an open-label, multicenter study in subjects with Stargardt disease, comparing 2 doses of STG-001 with regard to safety, pharmacokinetics and pharmacodynamics.

Who can participate

Age range18 Years – 55 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Understand the study procedures and agree to participate by providing written informed consent.
✓. STGD1 caused by autosomal recessive mutation in the ABCA4 gene (i.e. at least 2 mutations), confirmed genotypically by a fully accredited certified central genotyping laboratory.
✓. 18 to 55 years of age inclusive.
✓. Negative pregnancy testing for women of childbearing potential and highly effective double barrier contraception before, during, and for a period after study treatment as described in the clinical protocol.
✓. Primary study eye must have at least one well-demarcated area of significantly reduced autofluorescence as imaged by fundus autofluorescence (FAF)
✓. In at least one eye and in the same eye:
✓. ETDRS BCVA of less than or equal to 0.4 logMAR (≤68 letters; 20/50 Snellen equivalent) and greater than or equal to 1.3 logMAR (≥19 letters; 20/400 Snellen equivalent); and
✓. Clinical evidence of a macular lesion phenotypically consistent with Stargardt Disease

Exclusion criteria

✕. The subject is an employee of the Sponsor or study site or immediate family member (e.g., spouse, parent, child, sibling) of the Sponsor or study site.
✕. Use of prescription or nonprescription drugs and dietary supplements within 7 days or 5 half-lives (whichever is longer) prior to Day 1 first dose. Herbal supplements and HRT must be discontinued 7 days prior to Day 1 first dose.
✕. Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).
✕. Presence of any meaningful retinopathy/maculopathy/atrophy other than as a result of STGD1 in either eye as determined by the Investigator.
✕. Has taken non-approved items (supplement containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of Screening.
✕. Use of medications that may interact with Vitamin A metabolism within 60 days of Screening.
✕. Participation in an oral interventional study of a vitamin A derivative up to 3 months prior to Screening.
✕. The subject had a history of vitamin A deficiency as defined based upon serum values less than 20 mcg/dl (0.7 μmol/L) or clinical signs during slit lamp examination (conjunctival or corneal xerosis; Bitot's spots; corneal ulcers or scarring not due to trauma or other secondary causes).

What they're measuring

Safety events and Tolerability of STG-001 from baseline to 28 days: adverse events

Timeframe: From baseline to 28 days

Trial details

NCT IDNCT04489511

SponsorStargazer Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2021-04-24

View on ClinicalTrials.gov More Stargardt Disease-1 trials