Congenital Myasthenic Syndrome clinical trials at University of California Health

3 in progress, 1 open to eligible people

Showing trials for

  • Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)

    open to eligible people ages 18 years and up

    Participants will attend up to 3 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with DOK7-CMS better and may inform future study design.

    at UC Davis

  • ARGX-119 in Adult Participants with DOK7-Congenital Myasthenic Syndromes (CMS)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to assess the safety and tolerability of ARGX-119 in adult participants with DOK7- Congenital Myasthenic Syndromes. The study will also assess how ARGX-119 is processed by the body (pharmacokinetics), how the immune system reacts to it (immunogenicity), and how it may improve the way patients feel and function. After the screening period, eligible participants will be randomized in a 4:1 ratio to receive intravenous infusions of ARGX-119 or placebo during the treatment period. Participants will then enter the follow-up period. The full duration of the study is approximately 11 months.

    at UC Davis

  • Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome

    Sorry, not accepting new patients

    Primary: The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus until the product became commercially available. Secondary: The secondary objective of this study under the original protocol was to provide additional long-term safety data on amifampridine phosphate in patients. Primary The primary objective of this study after its fifth amendment was to provide access to amifampridine phosphate therapy to pediatric patients with LEMS, and pediatric and adult patients with CMS until the product became commercially available for these indications or development of the product for the indication was terminated. Secondary: The secondary objective of this study after its fifth amendment was to assess the long-term safety of amifampridine phosphate in pediatric patients with LEMS, and pediatric and adult patients with CMS.

    at UCLA

Our lead scientists for Congenital Myasthenic Syndrome research studies include .

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