Evidence map›Paper›PMID 38491364›Full record

ArticleBMC neurology2024

Defining clinical endpoints in limb girdle muscular dystrophy: a GRASP-LGMD study.

Amy Doody, Lindsay Alfano, Jordi Diaz-Manera, Linda Lowes, Tahseen Mozaffar, Katherine D Mathews, Conrad C Weihl, Matthew Wicklund, Man Hung, Jeffrey Statland and 2 more

Registry-linked trialOpen access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in BMC neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03981289 (GRASP-LGMD), which is not on this map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
2.2field-weighted citation impact, top 13% of its field
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.

NCT03981289 completednot on this map

GRASP-LGMD: Defining Clinical Endpoints in LGMD

TypeobservationalSponsorVirginia Commonwealth UniversityRan2019 to 2025Enrolled116ConditionsLimb Girdle Muscular Dystrophy, Muscular Dystrophies
3 · Its place in the literature

Who cites it

3 citing papers in PubMed, 5 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

12 authors at 9 institutions in 2 countries.

Amy DoodyVirginia Commonwealth University, Richmond, VA, USA.
Lindsay AlfanoNationwide Children's Hospital, Columbus, OH, USA.
Jordi Diaz-ManeraNewcastle University, Newcastle, UK.
Linda LowesNationwide Children's Hospital, Columbus, OH, USA.
Tahseen MozaffarUniversity of California, Irvine, CA, USA.
Katherine D MathewsUniversity of Iowa Carver College of Medicine, Iowa City, IA, USA.
Conrad C WeihlWashington University, St. Louis, MO, USA.
Matthew WicklundUT Health San Antonio, San Antonio, TX, USA.
Man HungRoseman University, Salt Lake City, UT, USA.
Jeffrey StatlandUniversity of Kansas Medical Center, Kansas City, KS, USA.
Nicholas E JohnsonVirginia Commonwealth University, Richmond, VA, USA. Nicholas.johnson@vcuhealth.org.
GRASP-LGMD Consortium
Virginia Commonwealth University · USNationwide Children's Hospital · USUniversity of Iowa · USNewcastle University · GBRoseman University of Health Sciences · USThe University of Texas Health Science Center at Houston · USUniversity of California, Irvine · USUniversity of Kansas Medical Center · USWashington University in St. Louis · US

Funding

Development and validation of clinical outcome assessments in LGMD2AR21TR003184 · NCATS · VIRGINIA COMMONWEALTH UNIVERSITY · PI JOHNSON, NICHOLAS ELWOOD, STATLAND, JEFFREY · 2020 to 2021
$452k
NCATS NIH HHS 1R21TR003184NCATS NIH HHS R21 TR003184
6 · The paper itself

Abstract

backgroundThe Limb Girdle Muscular Dystrophies (LGMDs) are characterized by progressive weakness of the shoulder and hip girdle muscles as a result of over 30 different genetic mutations. This study is designed to develop clinical outcome assessments across the group of disorders. METHODS/

designThe primary goal of this study is to evaluate the utility of a set of outcome measures on a wide range of LGMD phenotypes and ability levels to determine if it would be possible to use similar outcomes between individuals with different phenotypes. We will perform a multi-center, 12-month study of 188 LGMD patients within the established Genetic Resolution and Assessments Solving Phenotypes in LGMD (GRASP-LGMD) Research Consortium, which is comprised of 11 sites in the United States and 2 sites in Europe. Enrolled patients will be clinically affected and have mutations in CAPN3 (LGMDR1), ANO5 (LGMDR12), DYSF (LGMDR2), DNAJB6 (LGMDD1), SGCA (LGMDR3), SGCB (LGMDR4), SGCD (LGMDR6), or SGCG (LGMDR5, or FKRP-related (LGMDR9). DISCUSSION: To the best of our knowledge, this will be the largest consortium organized to prospectively validate clinical outcome assessments (COAs) in LGMD at its completion. These assessments will help clinical trial readiness by identifying reliable, valid, and responsive outcome measures as well as providing data driven clinical trial decision making for future clinical trials on therapeutic agents for LGMD. The results of this study will permit more efficient clinical trial design. All relevant data will be made available for investigators or companies involved in LGMD therapeutic development upon conclusion of this study as applicable.

trial registrationClinicaltrials.gov NCT03981289; Date of registration: 6/10/2019.

Indexed as

Muscular Dystrophies, Limb-GirdleSarcoglycanopathiesAnoctaminsHSP40 Heat-Shock ProteinsHumansMolecular ChaperonesMuscle, SkeletalMutationNerve Tissue ProteinsPentosyltransferasesPhenotypeANO5 protein, humanAnoctaminsDNAJB6 protein, humanFKRP protein, humanHSP40 Heat-Shock ProteinsMolecular ChaperonesNerve Tissue ProteinsPentosyltransferasesClinical outcome assessmentsClinical trialsLimb girdle muscular dystrophyMuscular dystrophyTherapeutic development

Identifiers

PMID38491364
PMCPMC10941356
OpenAlexW4392863536

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

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.