Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ARGX-113 · 8 trials · 7 indications
An AE was any untoward medical occurrence in a clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. Any clinically significant abnormal laboratory test results (hematology, clinical chemistry, or urinalysis) or other safety assessments (electrocardiogram \[ECG\], radiological scans, vital signs measurements) were collected as AEs. All AEs starting on or after first dose administered and until completion of participant's last visit were considered as TEAEs. A serious AE (SAE) was any AE that resulted in death, was life-threatening, required inpatient hospitalization, resulted in persistent or significant disability/incapacity, was a congenital abnormality, or was medically significant.
The MG-ADL is an 8-item patient-reported scale to assess MG symptoms and their effects on daily activities. The scale comprises 2 items on daily life activities and 6 items on symptoms. The MG-ADL total score range is 0-24, with higher scores indicative of greater disease severity. A patient was considered an MG-ADL responder during C1 if there was a reduction of ≥2 points on the MG-ADL total score (compared to baseline of C1 \[C1B\]) for ≥4 consecutive weeks with the first reduction occurring no later than 1 week after the last infusion of IMP in C1.
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
TEAEs were defined as AEs that first occurred or worsened in severity after the first administration of the treatment. A treatment emergent SAE was any untoward medical occurrence that resulted in death; was life-threatening; required inpatient hospitalization or prolongation of existing hospitalization;resulted in persistent or significant disability or incapacity; was a congenital abnormality or birth defect; or other medically significant events. All TEAEs observed were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 4.03 with descriptions of severity for each AE based on the following general guideline: Grade 1= mild; Grade 2 = moderate; Grade 3 = severe or medically significant but not immediately life-threatening; Grade 4 = life-threatening consequences; Grade 5 = death related to AE.
The patients' diastolic and systolic blood pressure were measured pre-dose on dosing days 1,8,15 and 22 and also during the follow up period. The mean change from baseline at each time point is presented. Baseline is defined as the last non-missing value before first dose of study medication.
The patients' heart rate was measured pre-dose on dosing days 1,8,15 and 22 and also during the follow up period. The mean change from baseline at each time point is presented. Baseline is defined as the last non-missing value before first dose of study medication.
The patients' temperature was measured pre-dose on dosing days 1,8,15 and 22 and also during the follow up period. The mean change from baseline at each time point is presented. Baseline is defined as the last non-missing value before first dose of study medication.
The patients' weight as measured pre-dose on dosing days 1,8,15 and 22 and also during the follow up period. The mean change from baseline at each time point is presented. Baseline is defined as the last non-missing value before first dose of study medication.
ECG parameters of heart rate, PR, QT, and QRS interval were read locally and performed pre-dose on dosing days 1,8,15 and 22 and on the last follow up visit on Day 78. Any patients recording abnormal clinically relevant findings during the study are presented.
Sampling for clinical laboratory tests including hematology, clinical chemistry, and urinalysiswas performed pre-dose on dosing Days 1, 8, 15 and 22 and throughout the follow up period. Patients fasted for at least 8 hours prior to this sampling. Abnormal laboratory values, or test results were not reported as TEAEs unless they were associated with clinical signs and symptoms that were considered clinically relevant, required therapy or led to treatment discontinuation. Patients reporting TEAEs in any of the laboratory parameters during the study are presented.
AUC0-inf
Determining the incidence, severity, and dose relationship of adverse events that are related to treatment with ARGX-113
| Arm | Type | Description |
|---|---|---|
| ARGX-113 | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| ARGX-113 Dose A + SoC | EXPERIMENTAL | Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo |
| ARGX-113 Dose B +SoC | EXPERIMENTAL | Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo |
| Placebo + SoC | PLACEBO_COMPARATOR | Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo |
| Treatment A | EXPERIMENTAL | Single SC injection of Dose A |
| Treatment B | EXPERIMENTAL | Single SC injection of Dose B |
| Treatment C | EXPERIMENTAL | Single SC injection of Dose C |
| Treatment D | EXPERIMENTAL | Single SC injection of Dose D |
| 1 | EXPERIMENTAL | Scheme 1 |
| 2 | EXPERIMENTAL | Scheme 2 |
| 3 | EXPERIMENTAL | Scheme 3 |
| Name | Type | Description |
|---|---|---|
| ARGX-113 | BIOLOGICAL | Intravenous administration of ARGX-113 |
| Placebo | BIOLOGICAL | Intravenous administration of placebo |
| ARGX-113 with rHuPH20 | BIOLOGICAL | subcutaneous administration of efgartigimod with recombinant human hyaluronidase PH20 (rHuPH20) |
Inclusion Criteria: 1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures. 2. Patients who participated in trial ARGX-113-1704 and are eligible for roll over, as specified in the protocol. Other mor...
ARGX-113 is an investigational small molecule being studied for use in Primary Immune Thrombocytopenia, Pemphigus Vulgaris, Generalized Myasthenia Gravis, and Myasthenia Gravis. It has also been evaluated in healthy volunteers and in bioavailability studies. As of the available data, it remains in clinical development and is not approved.
ARGX-113 is being developed by argenx SE, a biopharmaceutical company listed on the stock exchange under the ticker ARGX. The company is conducting clinical trials to evaluate the safety and efficacy of this investigational drug.
ARGX-113 is in Phase 1 and Phase 2 clinical development. The available trials include a Phase 2 study in patients with Primary Immune Thrombocytopenia and multiple Phase 1 studies in healthy volunteers. All trials listed are completed, and the drug remains investigational.
ARGX-113 has been studied in several completed trials, including NCT03102593 (Phase 2 in Primary Immune Thrombocytopenia), NCT03334084 (Phase 1 bioavailability study), NCT03457649 (Phase 1 in healthy volunteers), and NCT04073589 (Phase 1 in healthy subjects). These trials enrolled a total of 318 participants.
Yes, ARGX-113 is also known as efgartigimod. One of the clinical trials, NCT04073589, is titled 'Efgartigimod Co-administered Subcutaneously With rHuPH20 in Healthy Subjects,' confirming that ARGX-113 and efgartigimod refer to the same drug candidate.