A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

Open to people ages 18 years and up

• At least 18 years of age.
• Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS).
• Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study.

• Diagnosis of CMS due to mutation of any gene other than DOK7.
• Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator.
• History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer.
• Different study drug received in another clinical study within 12 weeks or 5 half-lives before screening.
• Current participation in another interventional clinical study or prior participation in any gene therapy or cell therapy study.
• Pregnant or lactating state or intention to become pregnant during the study.

The complete list of exclusion criteria can be found in the protocol.