NCT04489511

Study of STG-001 in Subjects With Stargardt Disease

A Phase 2a Study of the Safety, Pharmacokinetics and Pharmacodynamics of STG-001 in Subjects With Stargardt Disease (STGD1) Caused by Autosomal Recessive Mutation in ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Stargazer Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is an open-label, multicenter, active treatment study in approximately 12 subjects aged 18 to 55 years (inclusive) with STGD1, genotyped with a minimum of two ABCA4 gene mutations. Treatment course of STG-001 at Dose 1 (Cohort 1) or Dose 2 (Cohort 2) testing doses will be administered once daily for 28 days. Cohorts will run in parallel. Subjects will receive their designated dose daily for 28 days and be monitored for safety measures during the dosing period and for an additional 28 days after dosing (through Day 56).

Conditions

• Stargardt Disease-1

Interventions

Type	Name	Description
DRUG	STG-001	Dose 1 or Dose 2 of STG-001 will be administered daily for 28 days to compare safety, pharmacokinetics and pharmacodynamics in Stargardt disease subjects.

Timeline

• Start date: 2020-10-01
• Primary completion: 2021-04-24
• Completion: 2021-04-24
• First posted: 2020-07-28
• Last updated: 2021-04-27

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study