Evidence map›Paper›PMID 38842106›Full record

ArticleMuscle & nerve2024

Multicenter expanded access program for access to investigational products for amyotrophic lateral sclerosis.

Dylan V Neel, Clara Baselga-Garriga, Molly Benson, Mackenzie Keegan, Marianne Chase, Derek D'Agostino, Kristin Drake, Jennifer Linn Hagar, Meredith Gibbons Hasenoehrl, Jennifer Kulesa-Kelley and 26 more

Abstract readMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Article in Muscle & nerve, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing 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.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed.

  1. Trial
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  5. Review
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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

36 authors.

Dylan V NeelSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0001-6964-9842
Clara Baselga-GarrigaSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0001-7906-5752
Molly BensonSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Mackenzie KeeganSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Marianne ChaseSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Derek D'AgostinoSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Kristin DrakeSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Jennifer Linn HagarSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Meredith Gibbons HasenoehrlSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Jennifer Kulesa-KelleySean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Alex LeiteSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Silpa MohapatraSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Susanna Marie PortaroSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Lindsay M PothierSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Jesse RosenthalSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Alexander V ShermanSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Hong YuSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Alexandra McCaffreySean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Doreen HoSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Sarah LuppinoSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Richard BedlackDepartment of Neurology, Duke University, Durham, North Carolina, USA.ORCID https://orcid.org/0000-0003-4666-7495
Daragh HeitzmanMDA/ALS Clinic, Texas Neurology, Dallas, Texas, USA.
Senda Ajroud-DrissLes Turner ALS Center, Northwestern University, Chicago, Illinois, USA.
Jonathan KatzCalifornia Pacific Medical Center Research Institute and Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California, USA.
Kevin FeliceDepartment of Neuromuscular Medicine, Hospital for Special Care, New Britain, Connecticut, USA.
Charles WhitakerDepartment of Neuromuscular Medicine, Hospital for Special Care, New Britain, Connecticut, USA.
Shafeeq LadhaGregory W. Fulton ALS and Neuromuscular Center, Barrow Neurological Institute, Phoenix, Arizona, USA.
Gustavo AlamedaHoly Cross Hospital, Silver Spring, Maryland, USA.
Eduardo LocatelliCenter for Collaborative Research, Nova Southeastern University and Dr. Kiran C. Patel College of Allopathic Medicine, Fort Lauderdale, Florida, USA.
Irfan A QureshiBiohaven Pharmaceuticals, Inc., New Haven, Connecticut, USA.
Michael T HotchkinClene Nanomedicine, Inc., Salt Lake, Utah, USA.
Michael R HaydenNaarden, Prilenia Therapeutics, The Netherlands.
Merit E CudkowiczSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0002-7075-1681
Suma BabuSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
James D BerrySean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.
Sabrina PaganoniSean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA.ORCID https://orcid.org/0000-0003-0505-1168

Funding

Medical Scientist Training ProgramT32GM144273 · NIGMS · HARVARD MEDICAL SCHOOL · PI David Shumway Jones, Jacqueline A. Lees · 2022 to 2026
$14.7M
Biohaven Pharmaceuticals, IncI AM ALSNIGMS NIH HHS T32 GM144273
6 · The paper itself

Abstract

INTRODUCTION/

aimsExpanded access (EA) is a Food and Drug Administration-regulated pathway to provide access to investigational products (IPs) to individuals with serious diseases who are ineligible for clinical trials. The aim of this report is to share the design and operations of a multicenter, multidrug EA program for amyotrophic lateral sclerosis (ALS) across nine US centers.

methodsA central coordination center was established to design and conduct the program. Templated documents and processes were developed to streamline study design, regulatory submissions, and clinical operations across protocols. The program included three protocols and provided access to IPs that were being tested in respective regimens of the HEALEY ALS Platform Trial (verdiperstat, CNM-Au8, and pridopidine). Clinical and safety data were collected in all EA protocols (EAPs). The program cohorts comprised participants who were not eligible for the platform trial, including participants at advanced stages of disease progression and with long disease duration.

resultsA total of 85 participants were screened across the 3 EAPs from July 2021 to September 2022. The screen failure rate was 3.5%. Enrollment for the regimens of the platform trial was completed as planned and results informed the duration of the corresponding EAP. The verdiperstat EAP was concluded in December 2022. Mean duration of participation in the verdiperstat EAP was 5.8 ± 4.1 months. The CNM-Au8 and pridopidine EAPs are ongoing. DISCUSSION: Multicenter EAPs conducted in parallel to randomized clinical trials for ALS can successfully enroll participants who do not qualify for clinical trials.

Indexed as

Amyotrophic Lateral SclerosisAdaptive Clinical Trials as TopicAdultAgedDrugs, InvestigationalFemaleHealth Services AccessibilityHumansMaleMiddle AgedUnited StatesUnited States Food and Drug AdministrationDrugs, Investigationalamyotrophic lateral sclerosisexpanded accessinvestigational productmotor neuron disease

Identifiers

PMID38842106
PMCPMC13198442

What OpenQuestion holds

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Registered trials

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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.