Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Krivickas Brooke, Scirocco Erica, Giacomelli Elisa, Sharma Saloni, Benson Molly, Keegan Mackenzie, Kulesa-Kelley Jennifer, Chibnik Lori B, Casagrande Gabriella, Heyd Lindsay, Chase Marianne, Drake Kristin, Mohapatra Silpa, Hagar Jennifer Linn, Hasenoehrl Meredith Gibbons, Dagostino Derek, Sherman Alexander V, Leite Alex, Yu Hong, Rosenthal Jesse, Miller Timothy, McCaffrey Alexandra, Gwathmey Kelly, Locatelli Eduardo, Bayat Elham, Heitzman Daragh, Young Eufrosina, Goyal Namita A, Whitesell Jackie, Felice Kevin, Ilieva Hristelina, Swenson Andrea, Walk David, Alameda Gustavo, Foster Laura, McIlduff Courtney E, Walsh Alison, Zilliox Lindsay, Ajroud-Driss Senda, Bodkin Cynthia, Katz Jonathan, Ladha Shafeeq, Rivner Michael, Rosow Laura, Twydell Paul, Wasiewski Warren, Babu Suma, Berry James D, Paganoni Sabrina
Аннотация
METHODS: Participants received trehalose, a drug studied in a parallel RCT, for up to 24 weeks; clinical and biomarker data were collected throughout the study.
RESULTS: Seventy participants were enrolled at 20 study centers across the United States. Treatment with trehalose did not affect the levels of neurofilament light chain [estimated flat slope per month was -0.005, SE = 0.0078; 95% CI (-0.021, 0.011)] or disease progression [estimated least square mean change of the ALS Functional Rating Scale-Revised total score and slow vital capacity (percent predicted) from baseline to Week 24 were -5.6 (0.67); 95% CI (-7.0, -4.3) and -4.53 (4.308); 95% CI (-13.55, 4.48)], respectively. No unexpected treatment-related risks were identified. Serious adverse events were deemed not related to trehalose (20 occurrences in 13 [18.6%] participants with eight deaths).
DISCUSSION: This EAP establishes a framework for implementing multi-center EAPs that complement data collected from RCTs. Additional NIH-funded EAPs are currently underway. Data and additional serum samples collected in this study are available to the research community for further study.
TRIAL REGISTRATION: ClinicalTrials.gov: NCT05597436.