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

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argenx

Status

Enrolling

Conditions

Congenital Myasthenic Syndrome

Study type

Observational

Funder types

Industry

Identifiers

NCT06078553
ARGX-119-NIS-2301

Details and patient eligibility

About

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.

Enrollment

30 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Can understand the requirements of the study and can provide written informed consent, and willingness and ability to comply with the study protocol procedures
  • Is male or female and ≥18 years of age at the time of providing informed consent
  • Has a diagnosis of DOK7-CMS due to biallelic pathogenic mutations in DOK7
  • Has a total Quantitative Myasthenia Gravis (QMG) score of ≥3
  • For participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine), participant must have been receiving the medication for ≥3 months before screening/baseline

Exclusion criteria

  • Known medical condition that would interfere with an accurate assessment of DOK7-CMS, in the investigator's opinion
  • Is currently participating in any interventional clinical study with a study drug at the time of providing informed consent
  • Diagnosis of CMS due to mutation of any gene other than DOK7

Trial contacts and locations

10

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Central trial contact

Sabine Coppieters, MD

Data sourced from clinicaltrials.gov

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