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โ† Trials
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Study of ALK-001 on the Progression of Stargardt Disease

RecruitingPhase 3Ages 8 Years - 45 YearsAllAlkeus Pharmaceuticals, Inc.
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In plain words

What the trial is, and whether it could suit you

This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria 1. Male or female, 8 to 45 years of age (inclusive) on the day of screening. 2. Female participants of childbearing potential, as well as fertile male participants with female partners of childbearing potential, must be willing to comply with the protocol-defined methods of contraception from the time of consent until 90 days and 30 days, respectively, after the last dose of IMP. 3. Have a clinical diagnosis of typical autosomal recessive STGD macular dystrophy 4. Have provided a genetic report by any regional accredited organization that provides certified testing indicating the presence of disease-causing mutation. 5. Have signed and dated the informed consent forms (ICFs; or assent as appropriate) to participate. Exclusion Criteria 1. Has taken disallowed items (supplements containing vitamin A or beta-carotene, liver-based products, or prescription oral retinoid medications) within 30 days of randomization. 2. Is lactating, pregnant, or has a positive serum or urine pregnancy test at screening or at randomization, or is planning to become pregnant during the course of study. A male participant who wishes to father a child during the course of the study. 3. Has ever participated in any gene therapy, cell therapy, or device study to treat STGD, unless documented confirmation of participation in the placebo arm with no surgery is provided. Has participated in a drug study to treat STGD within the past 6 months. 4. Has participated in any drug study to treat any other condition within 5 half-lives of the investigational drug prior to screening, unless documented confirmation of participation in the placebo arm is provided. Has participated in an investigational device study within 30 days prior to screening or longer if, in the Investigator's judgment, the device could affect study outcomes. 5. Anticipates participating in any other drug or device study within the duration of the study.
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What it tests

  • ALK-001
  • Placebo
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Where

14 sites
Associated Retina Consultants
Phoenix, United States
University of Colorado SOM, Sue-Anschutz-Rodgers Eye Center
Aurora, United States
Vitreo Retinal Associates
Gainesville, United States
Tufts Medical Center - New England Eye Center
Boston, United States
Foundation for Vision Research
Grand Rapids, United States
Ohio State Havener Eye Institute
Columbus, United States
Erie Retina Research
Erie, United States
Mid Atlantic Retina
Philadelphia, United States

โ€ฆand 6 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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