Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ARGX-119 · 4 trials · 5 indications
Adverse Events
Serious Adverse Events
The RHS (Revised Hammersmith Scale) is a validated 36-item scale developed to evaluate the spectrum of gross motor function. Maximum total score; 69 (optimal motor function)
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 |
|---|---|---|
| DBTP - ARGX-119 IV | EXPERIMENTAL | Participants receive ARGX-119 IV during the DBTP |
| DBTP - Placebo IV | PLACEBO_COMPARATOR | Participants receive placebo IV during the DBTP |
| ATEP - ARGX-119 IV | PLACEBO_COMPARATOR | Participants receive ARGX-119 IV during the ATEP. Participants from ARGX-119 IV arm in the DBTP will receive placebo once to maintain the DBTP blinding |
| ARGX-119 - Dose 1 | EXPERIMENTAL | Participants will receive first dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period |
| ARGX-119 - Dose 2 | EXPERIMENTAL | Participants will receive second dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period |
| ARGX-119 - Dose 3 | EXPERIMENTAL | Participants will receive third dosage level of ARGX-119 intravenously during the double blinded treatment period followed by ARGX-119 in active treatment extension period |
| Placebo | PLACEBO_COMPARATOR | Participants will receive placebo intravenously during the double-blinded treatment period followed by ARGX-119 in active treatment extension period |
| 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 |
| ARGX-119 | EXPERIMENTAL | Patients receiving ARGX-119 IV or SC |
| Name | Type | Description |
|---|---|---|
| ARGX-119 IV | BIOLOGICAL | Intravenous infusion of ARGX-119 |
| Placebo IV | OTHER | Intravenous infusion of placebo |
| ARGX-119 | BIOLOGICAL | Intravenous infusion of ARGX-119 |
| Placebo | OTHER | Intravenous infusion of placebo |
Inclusion Criteria: * Is aged ≥5 to \<18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having a parent or guardian sign the ICF, and can comply with protocol * requirements. * Has documented historical genetic diagnosis o...