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Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling

Author

Listed:
  • Josephine Elia

    (Nemours, du Pont Hospital for Children
    Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University
    Department of Psychiatry Sidney Kimmel Medical College of Thomas Jefferson University)

  • Grace Ungal

    (Drexel University College of Medicine)

  • Charlly Kao

    (The Children’s Hospital of Philadelphia)

  • Alexander Ambrosini

    (University of Pennsylvania)

  • Nilsa Jesus-Rosario

    (Department of Psychiatry Sidney Kimmel Medical College of Thomas Jefferson University)

  • Lene Larsen

    (Department of Psychiatry Sidney Kimmel Medical College of Thomas Jefferson University)

  • Rosetta Chiavacci

    (The Children’s Hospital of Philadelphia)

  • Tiancheng Wang

    (The Children’s Hospital of Philadelphia)

  • Christine Kurian

    (Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Kanani Titchen

    (Nemours, du Pont Hospital for Children
    Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Brian Sykes

    (Nemours, du Pont Hospital for Children
    Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Sharon Hwang

    (Nemours, du Pont Hospital for Children
    Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Bhumi Kumar

    (Nemours, du Pont Hospital for Children
    Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Jacqueline Potts

    (Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Joshua Davis

    (Nemours, du Pont Hospital for Children
    Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Jeffrey Malatack

    (Nemours, du Pont Hospital for Children)

  • Emma Slattery

    (Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Ganesh Moorthy

    (University of Pennsylvania)

  • Athena Zuppa

    (University of Pennsylvania)

  • Andrew Weller

    (Department of Psychiatry Sidney Kimmel Medical College of Thomas Jefferson University)

  • Enda Byrne

    (The Children’s Hospital of Philadelphia)

  • Yun R. Li

    (The Children’s Hospital of Philadelphia
    University of California San Francisco)

  • Walter K. Kraft

    (Department of Pediatrics Sidney Kimmel Medical College of Thomas Jefferson University)

  • Hakon Hakonarson

    (The Children’s Hospital of Philadelphia
    University of Pennsylvania
    The Children’s Hospital of Philadelphia)

Abstract

The glutamatergic neurotransmitter system may play an important role in attention-deficit hyperactivity disorder (ADHD). This 5-week, open-label, single-blind, placebo-controlled study reports the safety, pharmacokinetics and responsiveness of the metabotropic glutamate receptor (mGluR) activator fasoracetam (NFC-1), in 30 adolescents, age 12–17 years with ADHD, harboring mutations in mGluR network genes. Mutation status was double-blinded. A single-dose pharmacokinetic profiling from 50–800 mg was followed by a single-blind placebo at week 1 and subsequent symptom-driven dose advancement up to 400 mg BID for 4 weeks. NFC-1 treatment resulted in significant improvement. Mean Clinical Global Impressions-Improvement (CGI-I) and Severity (CGI-S) scores were, respectively, 3.79 at baseline vs. 2.33 at week 5 (P

Suggested Citation

  • Josephine Elia & Grace Ungal & Charlly Kao & Alexander Ambrosini & Nilsa Jesus-Rosario & Lene Larsen & Rosetta Chiavacci & Tiancheng Wang & Christine Kurian & Kanani Titchen & Brian Sykes & Sharon Hwa, 2018. "Fasoracetam in adolescents with ADHD and glutamatergic gene network variants disrupting mGluR neurotransmitter signaling," Nature Communications, Nature, vol. 9(1), pages 1-9, December.
  • Handle: RePEc:nat:natcom:v:9:y:2018:i:1:d:10.1038_s41467-017-02244-2
    DOI: 10.1038/s41467-017-02244-2
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    Cited by:

    1. Hain HS & Connolly JJ & Glessner JT & Elia J & Hakonarson H, 2018. "Toward Individualized Treatments for Neurodevelopmental Disorders Targeting Metabotropic Glutamate Receptor (mGluR) Genes Impacted by Copy Number Variants," Open Access Journal of Neurology & Neurosurgery, Juniper Publishers Inc., vol. 8(2), pages 37-42, August.

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