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A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long-Term Safety, Tolerability, Pharmocokinetics and Effects of ALK-001 on the Progression of Stargardt Disease

Primary Objective

This is one trial in a series of trials studying an investigational drug called ALK-001 in people who have Stargardt Disease. Stargardt Disease can cause vision loss over time and currently has no treatment. We hope that the drug being studied, ALK-001, will slow or stop vision loss in patients with Stargardt Disease.

Study category: Eyes

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Description

The TEASE studies (Tolerability and Effects of ALK-001 in Stargardt Disease) are a series of clinical trials evaluating an investigational drug called ALK-001 in people who have Stargardt Disease. There are no FDA-approved treatments to slow the progression of Stargardt Disease. Research studies are conducted to determine if an investigational drug is safe and effective.

Main Procedures Involved: Details on Short Visits: Visit 2, Visit 3, Visit 4, Visit 6, Visit 8, Visit 10 and Visit 12 The major activities that take place during short visits are listed below: • Medications • Study Drug Supply • Discussion about your health • Vital Signs • Laboratory Tests • Pregnancy Test • Attestation on Contraception Requirements • At Visit 2 only, we may also perform Eye Imaging, Microperimetry, Visual Acuity and Reading Speed or procedures not completed during Visit 1. Details on Long Visits: Visit 1, Visit 5, Visit 7, Visit 9, and Visit 11 Long visits include all activities that take place during short visits, plus the following: • Demographics and Medical History • Electrocardiogram (ECG) • General Exams • Visual Acuity and Reading Speed • Questionnaires

Duration of Participation: There are a total of up to 12 scheduled visits. Seven visits are short (approximately 1 to 2 hours) and five visits last longer (we will attempt to make these long visits last less than 5 to 6 hours). Participation in the study will last up to approximately 28 months.

Details
Age

8 to years

Eligibility

Participants in this study must be at least 8 years old and diagnosed with Stargardt Disease, as confirmed by genetic testing, and must meet all other eligibility criteria.

Type of Study

Treatment

Location

University of Colorado Hospital

Principal Investigator
Marc Mathias,  MD

Marc Mathias, MD

Study ID

Protocol Number: 19-1103

More information available at ClinicalTrials.gov: NCT02402660

Categories

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