Evidence map›Paper›PMID 30451888›Full record

Trial reportScientific reports2018

Mavoglurant in Fragile X Syndrome: Results of two open-label, extension trials in adults and adolescents.

Randi Hagerman, Sebastien Jacquemont, Elizabeth Berry-Kravis, Vincent Des Portes, Andrew Stanfield, Barbara Koumaras, Gerd Rosenkranz, Alessandra Murgia, Christian Wolf, George Apostol and 1 more

Open access · goldAbstract readClinical Trial, Phase IIMulticenter StudyRandomized Controlled Trial
In one paragraph

Trial report in Scientific reports, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed
4.0field-weighted citation impact, top 6% 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.

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

30 citing papers in PubMed, 46 citations in OpenAlex.

  1. Effects of AFQ056 on language learning in fragile X syndrome.The Journal of clinical investigation · 2023
    Trial
  2. Article
  3. Review
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  5. Review
  6. Article
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  11. Bayesian PBPK modeling using R/Stan/Torsten and Julia/SciML/Turing.Jl.CPT: pharmacometrics & systems pharmacology · 2023
    Article
  12. Review
  13. Review
  14. Targeted Treatments for Fragile X Syndrome.Advances in neurobiology · 2023
    Article
  15. Review
  16. Article
  17. Observational
  18. Article
  19. Article
  20. Electrophysiological Biomarkers in Genetic Epilepsies.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2021
    Review
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

11 authors at 7 institutions in 6 countries.

Randi HagermanMIND Institute and Department of Pediatrics, UC Davis Medical Center, Sacramento, CA, USA.
Sebastien JacquemontCentre Hospitalier Universitaire Vaudois, Lausanne, Switzerland.
Elizabeth Berry-KravisRush University Medical Centre, Department of Pediatrics, Neurological Sciences, and Biochemistry, Chicago, IL, USA.
Vincent Des PortesNational Reference Center for Fragile X and Other XLID, CIC 1407 INSERM - Hospices Civils de Lyon, Université de Lyon and CNRS UMR 5304 (L2C2), Bron, France.
Andrew StanfieldPatrick Wild Centre, Division of Psychiatry, University of Edinburgh, Royal Edinburgh Hospital, Edinburgh, UK.
Barbara KoumarasNeurodegeneration Global Development, Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA.
Gerd RosenkranzNeuroscience Development, Novartis Pharma AG, Basel, Switzerland.
Alessandra MurgiaLaboratory of Molecular Genetics of Neurodevelopment, Department of Women's and Children's Health, University of Padova, Padova, Italy.
Christian WolfLycalis sprl, Brussels, Belgium.ORCID http://orcid.org/0000-0002-4683-0507
George ApostolNeuroscience Development, Novartis Pharma AG, Basel, Switzerland.
Florian von RaisonNeuroscience Development, Novartis Pharma AG, Basel, Switzerland. florian.von_raison@novartis.com.
Novartis (Switzerland) · CHRoyal Edinburgh Hospital · GBRush University Medical Center · USUniversité Claude Bernard Lyon 1 · FRUniversity Hospital of Lausanne · CHUniversity of California Davis Medical Center · USUniversity of Padua · IT

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Fragile X syndrome (FXS) is the most common monogenic cause of inherited intellectual and developmental disabilities. Mavoglurant, a selective metabotropic glutamate receptor subtype-5 antagonist, has shown positive neuronal and behavioral effects in preclinical studies, but failed to demonstrate any behavioral benefits in two 12-week, randomized, placebo-controlled, double-blind, phase IIb studies in adults and adolescents with FXS. Here we report the long-term safety (primary endpoint) and efficacy (secondary endpoint) results of the open-label extensions. Adolescent (n = 119, aged 12-19 years) and adult (n = 148, aged 18-45 years) participants received up to 100 mg bid mavoglurant for up to 34 months. Both extension studies were terminated prematurely due to lack of proven efficacy in the core studies. Mavoglurant was well tolerated with no new safety signal. Five percent of adults and 16.9 percent of adolescents discontinued treatment due to adverse events. Gradual and consistent behavioral improvements as measured by the ABC-C

Indexed as

AdolescentAdultChildDose-Response Relationship, DrugDouble-Blind MethodExcitatory Amino Acid AntagonistsFemaleFragile X SyndromeHumansIndolesMaleMental DisordersMiddle AgedPlacebosReceptor, Metabotropic Glutamate 5Young AdultExcitatory Amino Acid AntagonistsIndolesmavoglurantPlacebosReceptor, Metabotropic Glutamate 5

Identifiers

PMID30451888
PMCPMC6242849
OpenAlexW2901742569

What OpenQuestion holds

Textmetadata
LicenceCC BY
Read underepoch 390

Registered trials

None linked

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.