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Also known as ARGX-119 IV
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...
ARGX-119 is an investigational monoclonal antibody being developed for neurological conditions including Spinal Muscular Atrophy (SMA), Congenital Myasthenic Syndrome, and Amyotrophic Lateral Sclerosis (ALS). It is also studied in healthy volunteers for safety and tolerability. The drug is administered intravenously and is currently in clinical development.
ARGX-119 targets MuSK, a protein involved in neuromuscular signaling. By modulating MuSK activity, the antibody aims to address underlying mechanisms in diseases like SMA and congenital myasthenic syndromes. This target is central to the drug's proposed therapeutic approach in neurology.
ARGX-119 is being developed by argenx SE, a biopharmaceutical company listed on the stock exchange under the ticker ARGX. The company is conducting clinical trials for the drug across multiple neurological indications, including SMA and ALS.
ARGX-119 is in Phase 2 clinical development. It has completed a Phase 1 trial in healthy volunteers and is currently being evaluated in Phase 1b and Phase 2 studies for congenital myasthenic syndrome, ALS, and pediatric SMA. The drug is investigational and not yet approved.
ARGX-119 is being studied in several trials: NCT05670704 (completed Phase 1 in healthy volunteers), NCT06436742 (Phase 1b in DOK7-congenital myasthenic syndrome), NCT06441682 (Phase 2 in ALS), and NCT07287982 (Phase 2 in pediatric SMA). These trials assess safety, efficacy, and pharmacokinetics.
Yes, ARGX-119 is also known as ARGX-119 IV, referring to its intravenous route of administration. The alternative name is used in clinical trial contexts to specify how the drug is given. Both names refer to the same investigational monoclonal antibody developed by argenx.