Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06436742 | A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS) | PHASE1 | RECRUITING | 16 | — | — | Sep 24, 2024 | Jan 24, 2028 | Apr 23, 2026 | 9 | United States, Canada +4 |
The 6-minute walk test (6MWT) measures the distance a participant walks in 6 minutes. Before and after the 6MWT assessment, the participant's blood pressure, heart rate, and SPO2 will be recorded, and the participant's perception of fatigue and dyspnea will be measured.
| Arm | Type | Description |
|---|---|---|
| Double-blinded treatment period - ARGX-119 IV | EXPERIMENTAL | Participants receive ARGX-119 during the double-blinded treatment period |
| Double-blinded treatment period - Placebo IV | PLACEBO_COMPARATOR | Participants receive placebo during the double-blinded treatment period |
| Active-treatment period - ARGX-119 IV | EXPERIMENTAL | Participants receive ARGX-119 during the active-treatment period |
| Name | Type | Description |
|---|---|---|
| ARGX-119 | BIOLOGICAL | Intravenous infusion of ARGX-119 |
| Placebo | OTHER | Intravenous infusion of placebo |
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 m...