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

Launched by ARGENX · May 24, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called ARGX-119 for adults with a specific type of genetic condition known as DOK7-Congenital Myasthenic Syndromes (CMS). The main goal of the study is to see if ARGX-119 is safe to use and how well it works for improving symptoms in patients. Researchers will also look at how the body processes this treatment and how the immune system responds to it.

To participate, individuals must be at least 18 years old and have a confirmed diagnosis of DOK7-CMS. Participants will receive either the new treatment or a placebo (a substance with no active medication) through an intravenous infusion. The study will last about 11 months, during which participants will be monitored closely for any side effects and changes in their condition. It’s important for potential participants to know that they should not be involved in other clinical studies or have certain health conditions that could interfere with the results. This trial is currently recruiting, so those who meet the criteria may have the opportunity to contribute to important research in this area.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 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.
• Exclusion Criteria:
• • 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.