Evidence map›Paper›PMID 33533663›Full record

ArticleAmyotrophic lateral sclerosis & frontotemporal degeneration2021

Challenges and opportunities for Multi-National Investigator-Initiated clinical trials for ALS: European and United States collaborations.

Paul Lingor, Jan C Koch, Jeffrey M Statland, Sumaira Hussain, Christiane Hennecke, Joanne Wuu, Thomas Langbein, Raees Ahmed, René Günther, Benjamin Ilse and 9 more

Registry-linked trialAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Amyotrophic lateral sclerosis & frontotemporal degeneration, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03792490 (Inhibition of Rho Kinase), which is not on this map. Cited by 4 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed, 1 pooled it
–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.

NCT03792490 phase2completednot on this map

Inhibition of Rho Kinase (ROCK) With Fasudil as Disease-modifying Treatment for ALS

TypeinterventionalSponsorUniversity Medical Center GoettingenRan2019 to 2023Enrolled120ConditionsAmyotrophic Lateral SclerosisArmsFasudil, Placebo
3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Article
  3. Article
  4. Article
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

19 authors.

Paul LingorDepartment of Neurology, School of Medicine, Technical University of Munich, Klinikum rechts der Isar, München, Germany.ORCID 0000-0001-9362-7096
Jan C KochDepartment of Neurology, University Medical Center Göttingen, Göttingen, Germany.
Jeffrey M StatlandDepartment of Neurology, University of Kansas Medical Center, Kansas City, Kansas, USA.
Sumaira HussainDepartment of Neurology, University of Miami, Miami, Florida, USA.
Christiane HenneckeDepartment for International Cooperations, University Medical Center Göttingen, Göttingen, Germany.
Joanne WuuDepartment of Neurology, University of Miami, Miami, Florida, USA.
Thomas LangbeinClinical Trials Unit, University Medical Center Göttingen, Göttingen, Germany.
Raees AhmedDepartment for International Cooperations, University Medical Center Göttingen, Göttingen, Germany.
René GüntherDepartment of Neurology, Technische Universität Dresden and German Center for Neurodegenerative Diseases, Dresden, Germany.
Benjamin IlseDepartment of Neurology, Jena University Hospital, Jena, Germany.
Jan KassubekDepartment of Neurology, RKU, University of Ulm, Ulm, Germany.
Katja KolleweDepartment of Neurology, Hannover Medical School, Hannover, Germany.
Josua KuttlerDepartment of Neurology, University Medical Center Göttingen, Göttingen, Germany.
Andreas LehaDepartment of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany.
Teresa LengenfeldDepartment of Neurology, University Medical Center Göttingen, Göttingen, Germany.
Thomas MeyerCenter for ALS and other Motor Neuron Disorders, Charité - Universitätsmedizin Berlin, Berlin, Germany.
Christoph NeuwirthNeuromuscular Disease Unit/ALS Clinic, Kantonsspital St. Gallen, St. Gallen, Switzerland.
Ralf TostmannDepartment for International Cooperations, University Medical Center Göttingen, Göttingen, Germany.
Michael BenatarDepartment of Neurology, University of Miami, Miami, Florida, USA.ORCID 0000-0003-4241-5135

Funding

Uncovering new genes and disease modifiers for ALS and related disordersU54NS092091 · NINDS · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI LINDSAY N ALFANO · 2014 to 2026
$19.8M
NINDS NIH HHS U54 NS092091
6 · The paper itself

Abstract

An inherent challenge to clinical trials that aim to test the efficacy of experimental therapeutics for patients with amyotrophic lateral sclerosis (ALS) is the relative rarity of the disease. A promising solution to this problem is a multi-center approach that ideally includes sites distributed across a broad geographic area. In support of such an approach, the European E-RARE program and the United States National Institutes of Health (NIH) partnered to support the investigator-initiated ROCK-ALS trial (Eudra-CT-Nr.: 2017-003676-31, NCT03792490) as a multi-national collaboration between centers in Europe and North America that is led by European investigators. During the set-up of this international trial, however, a number of unanticipated legal, administrative, and financial complexities emerged that required significant adaptation of the proposed trial scheme. Here, we report our experience navigating these obstacles and describe the potential solutions that we explored. Our experience may inform future efforts to implement multi-national investigator-initiated trials that involve both European and United States centers.

Indexed as

Amyotrophic Lateral SclerosisClinical Trials as TopicEuropeHumansUnited StatesClinical trialinternationalrare diseaseregulatorysister trials

Identifiers

PMID33533663
PMCPMC8289747

What OpenQuestion holds

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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.