AI Article Synopsis
- This study reports a 1-year follow-up on a clinical trial that tested a new subretinal implant using human embryonic stem cell-derived retinal pigment epithelium (RPE) cells for patients with advanced non-neovascular age-related macular degeneration (NNAMD).
- The trial involved 16 participants, focused on safety over the course of a year, and included a customized insertion procedure and low-dose immunosuppression to prevent rejection.
- Results indicated the implant was generally safe, with some patients showing slight improvements in vision, though the study was not specifically designed to evaluate efficacy.
Article Abstract
Purpose: To report 1-year follow-up of a phase 1/2a clinical trial testing a composite subretinal implant having polarized human embryonic stem cell (hESC)-derived retinal pigment epithelium (RPE) cells on an ultrathin parylene substrate in subjects with advanced non-neovascular age-related macular degeneration (NNAMD).
Methods: The phase 1/2a clinical trial included 16 subjects in two cohorts. The main endpoint was safety assessed at 365 days using ophthalmic and systemic exams. Pseudophakic subjects with geographic atrophy (GA) and severe vision loss were eligible. Low-dose tacrolimus immunosuppression was utilized for 68 days in the peri-implantation period. The implant was delivered to the worst seeing eye with a custom subretinal insertion device in an outpatient setting. A data safety monitoring committee reviewed all results.
Results: The treated eyes of all subjects were legally blind with a baseline best-corrected visual acuity (BCVA) of ≤ 20/200. There were no unexpected serious adverse events. Four subjects in cohort 1 had serious ocular adverse events, including retinal hemorrhage, edema, focal retinal detachment, or RPE detachment, which was mitigated in cohort 2 using improved hemostasis during surgery. Although this study was not powered to assess efficacy, treated eyes from four subjects showed an increased BCVA of >5 letters (6-13 letters). A larger proportion of treated eyes experienced a >5-letter gain when compared with the untreated eye (27% vs. 7%; P = not significant) and a larger proportion of nonimplanted eyes demonstrated a >5-letter loss (47% vs. 33%; P = not significant).
Conclusions: Outpatient delivery of the implant can be performed routinely. At 1 year, the implant is safe and well tolerated in subjects with advanced dry AMD.
Translational Relevance: This work describes the first clinical trial, to our knowledge, of a novel implant for advanced dry AMD.
Download full-text PDF |
Source |
|---|---|
| http://www.ncbi.nlm.nih.gov/pmc/articles/PMC8496407 | PMC |
| http://dx.doi.org/10.1167/tvst.10.10.13 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Studying the Digital Intervention Engagement-Mediated Relationship Between Intrapersonal Measures and Pre-Exposure Prophylaxis Adherence in Sexual and Gender Minority Youth: Secondary Analysis of a Randomized Controlled Trial.
J Med Internet Res
January 2025
Department of Community Health Sciences, Boston University, Boston, MA, United States.
Background: Improving adherence to pre-exposure prophylaxis (PrEP) via digital health interventions (DHIs) for young sexual and gender minority men who have sex with men (YSGMMSM) is promising for reducing the HIV burden. Measuring and achieving effective engagement (sufficient to solicit PrEP adherence) in YSGMMSM is challenging.
Objective: This study is a secondary analysis of the primary efficacy randomized controlled trial (RCT) of Prepared, Protected, Empowered (P3), a digital PrEP adherence intervention that used causal mediation to quantify whether and to what extent intrapersonal behavioral, mental health, and sociodemographic measures were related to effective engagement for PrEP adherence in YSGMMSM.
Intra-Arterial Tenecteplase Following Endovascular Reperfusion for Large Vessel Occlusion Acute Ischemic Stroke: The POST-TNK Randomized Clinical Trial.
JAMA
January 2025
Department of Neurology, Xinqiao Hospital and The Second Affiliated Hospital, Army Medical University (Third Military Medical University), Chongqing, China.
Importance: The impact of adjunctive intra-arterial tenecteplase administration following near-complete to complete reperfusion by endovascular thrombectomy (EVT) for acute ischemic stroke is unknown.
Objective: To assess the efficacy and adverse events of adjunctive intra-arterial tenecteplase in patients with large vessel occlusion stroke who had achieved near-complete to complete reperfusion (defined as a score on the expanded Thrombolysis in Cerebral Infarction [eTICI] scale of 2c to 3) after EVT.
Design, Setting, And Participants: Investigator-initiated, randomized, open-label, blinded outcome assessment trial implemented at 34 hospitals in China among 540 patients with stroke due to proximal intracranial large vessel occlusion within 24 hours of the time they were last known to be well, with an eTICI score of 2c to 3 after EVT, and without prior intravenous thrombolysis.
Intra-Arterial Urokinase After Endovascular Reperfusion for Acute Ischemic Stroke: The POST-UK Randomized Clinical Trial.
JAMA
January 2025
Department of Neurology, Xinqiao Hospital and The Second Affiliated Hospital, Army Medical University (Third Military Medical University), Chongqing, China.
Importance: Persisting or new thrombi in the distal arteries and the microcirculation have been reported to limit the benefits of successful endovascular thrombectomy for patients with acute ischemic stroke. It remains uncertain whether intra-arterial thrombolysis by urokinase following near-complete to complete reperfusion by thrombectomy improves outcomes among patients with ischemic stroke due to large vessel occlusion.
Objective: To assess the efficacy and adverse events of intra-arterial urokinase after near-complete to complete reperfusion by thrombectomy for acute ischemic stroke due to large vessel occlusion.
Androgen Receptor Pathway Inhibitor Therapy for Advanced Prostate Cancer: Secondary Analysis of a Randomized Clinical Trial.
JAMA Netw Open
January 2025
Latin American Cooperative Oncology Group (LACOG), Porto Alegre, Brazil.
Importance: The open-label randomized phase 2 LACOG0415 trial evaluated 3 treatment strategies for patients with advanced castration-sensitive prostate cancer (CSPC): androgen deprivation therapy (ADT) plus abiraterone acetate and prednisone (AAP), apalutamide (APA) alone, or APA plus AAP.
Objective: To investigate the association of ADT plus AAP, APA alone, or APA plus AAP with health-related quality of life (HRQOL) in patients with advanced CSPC in the LACOG0415 trial.
Design, Setting, And Participants: The LACOG0415 randomized clinical trial comprised 128 patients with advanced CSPC who were randomized (1:1:1) to 1 of 3 treatment arms from October 16, 2017, to April 23, 2019.
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!