NCT06436742 A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)
| NCT ID | NCT06436742 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | argenx |
| Condition | Congenital Myasthenic Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 16 participants |
| Start Date | 2024-09-24 |
| Primary Completion | 2027-08-24 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 16 participants in total. It began in 2024-09-24 with a primary completion date of 2027-08-24.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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 double-blinded treatment period. Participants will then enter the follow-up period. After the follow-up period, participants may enrol in the active-treatment period, where they will receive open-label ARGX-119. The full duration of the study is approximately 38 months.
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. * Pregnant or lactating state or intention to become pregnant during the study.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06436742 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Congenital Myasthenic Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06436742 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06436742 currently recruiting?
Yes, NCT06436742 is actively recruiting participants. Contact the research team at clinicaltrials@argenx.com for enrollment information.
Where is the NCT06436742 trial being conducted?
This trial is being conducted at Sacramento, United States, Chicago, United States, Ottawa, Canada, Marseille, France and 5 additional locations.
Who is sponsoring the NCT06436742 clinical trial?
NCT06436742 is sponsored by argenx. The trial plans to enroll 16 participants.