Evidence map›Paper›PMID 40857020›Full record

Trial reportMuscle & nerve2025

Multicenter Expanded Access Protocol for Research Through Access to Trehalose in People With Amyotrophic Lateral Sclerosis.

Brooke Krivickas, Erica Scirocco, Elisa Giacomelli, Saloni Sharma, Molly Benson, Mackenzie Keegan, Jennifer Kulesa-Kelley, Lori B Chibnik, Gabriella Casagrande, Lindsay Heyd and 39 more

Registry-linked trialAbstract readMulticenter StudyRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Muscle & nerve, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT05597436 (An Expanded Access Protocol of Intravenous Trehalose Injection 90.5 mg/mL Treatment of Patients With Amyotrophic Lateral Sclerosis), which is not on this map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT05597436 no longer availablenot on this map

An Expanded Access Protocol of Intravenous Trehalose Injection 90.5 mg/mL Treatment of Patients With Amyotrophic Lateral Sclerosis

Typeexpanded_accessSponsorSeelos Therapeutics, Inc.ConditionsAmyotrophic Lateral SclerosisArmsTrehalose
3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Trial
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

49 authors.

Brooke KrivickasSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Erica SciroccoSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0009-0002-9427-1452
Elisa GiacomelliSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0001-9233-235X
Saloni SharmaSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Molly BensonSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Mackenzie KeeganSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0003-1305-8124
Jennifer Kulesa-KelleySean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Lori B ChibnikSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0001-6293-806X
Gabriella CasagrandeSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0009-0006-8731-6383
Lindsay HeydSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0002-2377-1778
Marianne ChaseSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Kristin DrakeSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Silpa MohapatraSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Jennifer Linn HagarSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Meredith Gibbons HasenoehrlSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Derek DagostinoSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Alexander V ShermanSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Alex LeiteSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Hong YuSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Jesse RosenthalSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Timothy MillerWashington University School of Medicine, St. Louis, Missouri, USA.
Alexandra McCaffreySean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Kelly GwathmeyVirginia Commonwealth University, Richmond, Virginia, USA.ORCID https://orcid.org/0000-0002-7589-8826
Eduardo LocatelliNova Southeastern University, Davie, Florida, USA.
Elham BayatGeorge Washington University, Washington, DC, USA.
Daragh HeitzmanTexas Neurology, Dallas, Texas, USA.
Eufrosina YoungUpstate Medical University, Syracuse, New York, USA.
Namita A GoyalUniversity of California Irvine, Orange, California, USA.ORCID https://orcid.org/0000-0002-1713-545X
Jackie WhitesellSaint Alphonsus, Boise, Idaho, USA.
Kevin FeliceHospital for Special Care, New Britain, Connecticut, USA.ORCID https://orcid.org/0000-0002-5084-6827
Hristelina IlievaThomas Jefferson University, Philadelphia, Pennsylvania, USA.ORCID https://orcid.org/0000-0001-8318-7533
Andrea SwensonUniversity of Iowa, Iowa City, Iowa, USA.
David WalkUniversity of Minnesota, Minneapolis, Minnesota, USA.ORCID https://orcid.org/0000-0001-7941-1993
Gustavo AlamedaHoly Cross Hospital, Fort Lauderdale, Florida, USA.
Laura FosterUniversity of Colorado Anschutz, Aurora, Colorado, USA.ORCID https://orcid.org/0000-0002-7804-2345
Courtney E McIlduffBeth Israel Deaconess Medical Center, Boston, Massachusetts, USA.
Alison WalshLehigh Valley Health Network, Allentown, Pennsylvania, USA.
Lindsay ZillioxUniversity of Maryland School of Medicine, Baltimore, Maryland, USA.
Senda Ajroud-DrissNorthwestern University, Chicago, Illinois, USA.
Cynthia BodkinIndiana University, Indianapolis, Indiana, USA.
Jonathan KatzCalifornia Pacific Medical Center, San Francisco, California, USA.
Shafeeq LadhaBarrow Neurological Institute, Phoenix, Arizona, USA.
Michael RivnerAugusta University, Augusta, Georgia, USA.ORCID https://orcid.org/0000-0002-6135-035X
Laura RosowUniversity of California San Francisco, San Francisco, California, USA.
Paul TwydellSpectrum Health, Grand Rapids, Michigan, USA.
Warren WasiewskiSeelos Therapeutics, New York, New York, USA.
Suma BabuSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
James D BerrySean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Sabrina PaganoniSean M. Healey & AMG Center for ALS and Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0003-0505-1168

Funding

An Expanded Access Protocol of Intravenous Trehalose Injection 90 mg/mL Treatment of Patients with Amyotrophic Lateral SclerosisUF1NS131791 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI BABU, SUMA, BERRY, JAMES DALE · 2022 to 2022
$18.1M
NINDS NIH HHS UF1 NS131791Office of the Director of the National Institutes of Health UF1NS131791
6 · The paper itself

Abstract

INTRODUCTION/

aimsExpanded 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).

methodsParticipants received trehalose, a drug studied in a parallel RCT, for up to 24 weeks; clinical and biomarker data were collected throughout the study.

resultsSeventy 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 registrationClinicalTrials.gov: NCT05597436.

Indexed as

Amyotrophic Lateral SclerosisTrehaloseAdultAgedDisease ProgressionFemaleHumansMaleMiddle AgedNeurofilament ProteinsNeurofilament ProteinsTrehaloseamyotrophic lateral sclerosisexpanded accessinvestigational productmotor neuron diseasetrehalose

Identifiers

PMID40857020
PMCPMC12428506

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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.