Journal article
Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis
Muscle & nerve, Vol.72(5), pp.1108-1116
11/2025
DOI: 10.1002/mus.70011
PMCID: PMC12428506
PMID: 40857020
Abstract
Introduction/Aims
Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)-funded EAP for amyotrophic lateral sclerosis (ALS).
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.
Details
- Title: Subtitle
- Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis
- Creators
- Brooke Krivickas - Massachusetts General HospitalErica Scirocco - Harvard UniversityElisa Giacomelli - Harvard UniversitySaloni Sharma - Massachusetts General HospitalMolly Benson - Massachusetts General HospitalMackenzie Keegan - Massachusetts General HospitalJennifer Kulesa-Kelley - Massachusetts General HospitalLori B. Chibnik - Massachusetts General HospitalGabriella Casagrande - Massachusetts General HospitalLindsay Heyd - Harvard Medical SchoolMarianne Chase - Massachusetts General HospitalKristin Drake - Massachusetts General HospitalSilpa Mohapatra - Massachusetts General HospitalJennifer Linn Hagar - Massachusetts General HospitalMeredith Gibbons Hasenoehrl - Massachusetts General HospitalDerek Dagostino - Harvard Medical SchoolAlexander V. Sherman - Harvard UniversityAlex Leite - Massachusetts General HospitalHong Yu - Harvard UniversityJesse Rosenthal - Massachusetts General HospitalTimothy Miller - Washington University in St. Louis School of MedicineAlexandra McCaffrey - Massachusetts General HospitalKelly Gwathmey - Virginia Commonwealth UniversityEduardo Locatelli - Nova Southeastern UniversityElham Bayat - George Washington UniversityDaragh Heitzman - Texas NeurologyEufrosina Young - SUNY Upstate Medical UniversityNamita A. Goyal - University of California, IrvineJackie Whitesell - Saint Alphonsus Medical CenterKevin Felice - Hospital for Special CareHristelina Ilieva - Thomas Jefferson UniversityAndrea Swenson - University of IowaDavid Walk - University of MinnesotaGustavo Alameda - Holy Cross HospitalLaura Foster - University of Colorado Anschutz Medical CampusCourtney E. McIlduff - Beth Israel Deaconess Medical CenterAlison Walsh - Lehigh Valley Hospital-PoconoLindsay Zilliox - University of Maryland, BaltimoreSenda Ajroud-Driss - Northwestern UniversityCynthia Bodkin - Indiana University – Purdue University IndianapolisJonathan Katz - California Pacific Medical CenterShafeeq Ladha - Barrow Neurological InstituteMichael Rivner - Augusta UniversityLaura Rosow - University of California, San FranciscoPaul Twydell - Corewell Health Blodgett HospitalWarren Wasiewski - Seelos Therapeutics (United States)Suma Babu - Massachusetts General HospitalJames D. Berry - Massachusetts General HospitalSabrina Paganoni - Massachusetts General Hospital
- Resource Type
- Journal article
- Publication Details
- Muscle & nerve, Vol.72(5), pp.1108-1116
- DOI
- 10.1002/mus.70011
- PMID
- 40857020
- PMCID
- PMC12428506
- NLM abbreviation
- Muscle Nerve
- ISSN
- 0148-639X
- eISSN
- 1097-4598
- Publisher
- Wiley
- Grant note
- Office of the Director of the National Institutes of Health: UF1NS131791
This work was supported by the Office of the Director of the National Institutes of Health (NIH) (UF1NS131791)
- Language
- English
- Electronic publication date
- 08/26/2025
- Date published
- 11/2025
- Academic Unit
- Neurology
- Record Identifier
- 9984949232702771
Metrics
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