NCT04489511

Study of STG-001 in Subjects With Stargardt Disease

Completed Phase 2 Last updated 27 April 2021

What this trial tests

Phase 2 trial testing STG-001 in Stargardt Disease-1 in 10 participants. Completed in 24 April 2021.

Timeline

1 October 2020

Primary endpoint
24 April 2021

24 April 2021

Quick facts

Lead sponsor	Stargazer Pharmaceuticals, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	10
Start date	1 October 2020
Primary completion	24 April 2021
Estimated completion	24 April 2021
Sites	5 locations across United States

Drugs / interventions tested

• STG-001 — full drug profile →

Conditions studied

• Stargardt Disease-1 — all drugs for Stargardt Disease-1 →

Sponsor

Stargazer Pharmaceuticals, Inc.

Who can join

Adults 18 to 55, any sex, with Stargardt Disease-1. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Therapy Approaches for Stargardt Disease.
   Piotter E, McClements ME, MacLaren RE. · · 2021 · cited 33× · PMID 34439845 · DOI 10.3390/biom11081179
2. Inherited Retinal Dystrophies: Role of Oxidative Stress and Inflammation in Their Physiopathology and Therapeutic Implications.
   Pinilla I, Maneu V, Campello L, Fernández-Sánchez L, et al · · 2022 · cited 30× · PMID 35739983 · DOI 10.3390/antiox11061086
3. Lipofuscin, Its Origin, Properties, and Contribution to Retinal Fluorescence as a Potential Biomarker of Oxidative Damage to the Retina.
   Różanowska MB. · · 2023 · cited 28× · PMID 38136230 · DOI 10.3390/antiox12122111
4. Stargardt macular dystrophy and therapeutic approaches.
   Fujinami K, Waheed N, Laich Y, Yang P, et al · · 2024 · cited 20× · PMID 37940365 · DOI 10.1136/bjo-2022-323071
5. Emerging Therapeutic Approaches and Genetic Insights in Stargardt Disease: A Comprehensive Review.
   Ghenciu LA, Hațegan OA, Stoicescu ER, Iacob R, et al · · 2024 · cited 19× · PMID 39201545 · DOI 10.3390/ijms25168859
6. The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease.
   Martinez Velazquez LA, Ballios BG. · · 2021 · cited 18× · PMID 34768969 · DOI 10.3390/ijms222111542
7. Updates on Emerging Interventions for Autosomal Recessive <i>ABCA4</i>-Associated Stargardt Disease.
   Wang L, Shah SM, Mangwani-Mordani S, Gregori NZ. · · 2023 · cited 8× · PMID 37834872 · DOI 10.3390/jcm12196229
8. Discovery of Nonretinoid Inhibitors of CRBP1: Structural and Dynamic Insights for Ligand-Binding Mechanisms.
   Plau J, Morgan CE, Fedorov Y, Banerjee S, et al · · 2023 · cited 6× · PMID 37713257 · DOI 10.1021/acschembio.3c00402