The Data Safety and Monitoring Board (DSMB) has granted approval for progression to the medium dose in Ocugen’s OCU410ST clinical trial for Stargardt disease.
The decision follows the establishment of the low dose of modifier gene therapy OCU410ST as safe and tolerable, marking a significant step in the trial’s dose-escalation phase.
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Ocugen has reported that three patients have been dosed with the low dose in the Phase I/II clinical trial for Stargardt disease.
Named GARDian, the trial is set to continue with the enrolment and dosing of three patients with the medium dose of the therapy in cohort two and three subjects with the high dose in cohort three in the dose-escalation phase.
It is designed to evaluate the safety and efficacy of OCU410ST when administered subretinally to individuals with Stargardt disease.
The trial comprises two phases, with Phase I involving a dose-ranging study across three levels and Phase II focusing on a dose-expansion study with randomised, outcome accessor-blinded groups.
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By GlobalDataStargardt disease, a genetic condition leading to retinal degeneration and vision loss, is the focus of the OCU410ST therapy.
Using the AAV delivery platform, OCU410ST will deliver the RAR Related Orphan Receptor A (RORA gene), which is implicated in several pathways associated with the disease such as oxidative stress and inflammation.
Ocugen chief medical officer Huma Qamar said: “We are pleased to see the continued favourable safety and tolerability profile exhibited by OCU410ST, allowing us to evaluate a higher dose in patients with Stargardt retinal dystrophy.
“We recognise the high unmet medical need for Stargardt patients, as there are no current FDA-approved therapies for the indication, and we look forward to sharing the interim safety data from our Phase I trial in the second quarter of 2024.”
Last year, Ocugen announced plans to start Phase I/II trials for gene therapies, OCU410 and OCU410ST, by the end of last year in geographic atrophy and Stargardt disease.
Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.
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