Clareo Health | A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
argenx
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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.
Pregnant or lactating state or intention to become pregnant during the study.
