Clinical trials remain rare
There are few clinical trials on Stargardt disease.
They focus on different technologies: gene therapy, cell therapy, pharmacology.
Some of these are underway.
(updated 2018 may, Clinical trials)
Gene therapy
Phase I/IIa Study of SAR422459 in Patients With Stargardt’s Macular Degeneration (Phase I/II* – Sponsor : Sanofi – Assay USA and France)
A Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt’s Macular Degeneration (Phase I/II* – Sponsor : Sanofi – Assay USA and France)
Forcoming : future Phase II trial« Gene therapy (AAV-ABCA4) to treat blindness caused by Stargardt disease »- Oxford University Innovation
http://innovation.ox.ac.uk/licence-details/gene-therapy-treat-blindness-caused-stargardt-disease/
Cell therapy
Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt’s Macular Dystrophy (Phase I/II* – Sponsor : Astellas Institute for Regenerative Medicine – Assay in USA)
Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients (Phase I/II* – Biological: MA09-hRPE -Sponsor : Astellas Institute for Regenerative Medicine – Assay in USA)
Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt’s Macular Dystrophy (SMD) (Phase I/II* – Biological: MA09-hRPE – Sponsor : Astellas Institute for Regenerative Medicine – Assay in UK))
A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt’s Macular Dystrophy (SMD) ( Phase I/II- Sponsor : Astellas Institute for Regenerative Medicine – Assay in UK)
Pharmacology
Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease (Phase II – Sponsor Acucela Inc., Assay in USA)
Zimura (Complement C5 Inhibitor) Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1) (Phase II -Sponsor : Ophthotech Corporation. Assay USA)
Saffron Supplementation in Stargardt’s Disease (Pase I/II* – Sponsor : Catholic University of the Sacred Heart – Assay in Italy)
DHA Supplementation in Patients With STGD3 (Phase I/II* ; Sponsor: University of Utah. Assay in USA)
Phase 2 Tolerability and Effects of ALK-001 (C20-D3-retinyl acetate) on Stargardt Disease (Phase II – Sponsor : Alkeus Pharmaceuticals, Inc. Assay in USA)
Other techniques
Microcurrent Stimulation to Treat Macular Degeneration ( Sonsor : Retina Institute of Hawaii – Assay in USA)
Other Studies Programs
ProgStar (http://progstar.org) :
Sponsored by the Foundation Fighting Blindness, the Natural History of the Progression of Atrophy Secondary to Stargardt Disease (ProgStar) studies are comprised of retrospective and prospective longitudinal observational studies. The retrospective study includes clinical examination findings and images collected between 2008 (and earlier) and 2014 and progression will be retrospectively evaluated. The prospective study consists of a 24 month observational period from 2013/2014 for two years, with one visit every six months.
ProgStar Study : A natural History of the progression of Stargardt Disease : retrospective and prospective Studies (Learn more > Clinicaltrials.gov). ” With the ultimate goal of determining the best outcome measures for clinical trials, the ProgStar study currently enrolls patients with Stargardt disease type 1 (STGD1) caused by mutations in the ABCA4 gene.”
*Phase I/II = Safety/Efficacy Study
Publication
To find out more about current research, we advise you to consult the following websites which group together many international clinical trials.
- Clinical trials
- Pubmed
http://www.ncbi.nlm.nih.gov/pubmed
