Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
efgartigimod PH20 · 15 trials · 18 indications
Free triiodothyronine: \[fT3\]; free thyroxine: \[fT4\]; ATDs: antithyroid drugs
The ESSDAI was designed to measure systemic disease activity in patients with PSjD and consists of 12 domains. The score varies between 0 (no disease activity) and 123 (max disease activity). The clinESSDAI includes all ESSDAI domains except the biological domain.
The Myasthenia Gravis Impairment Index (MGII) is a scoring tool measuring disease severity. It consists of 22 patient-reported outcomes (PRO) and 6 physical examinations (PE). The Ocular PRO score varies between 0 and 18. The higher the score, the more severe the disease.
AE : adverse event ; SAE : serious adverse event ; AESI : adverse event of special interest.
Adverse events, Serious Adverse event and Adverse events of special interest. Adverse events in the 'Infections and infestations' SOC were defined as AESIs because efgartigimod causes a transient reduction in total IgG levels.
ANCOVA Analysis of Percent Change From Baseline in Total IgG Level at Day 29 (ie, 7 days after the fourth IV or SC administration).
A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.
Proportion of participants with pemphigus vulgaris who had CRmin within 30 weeks, defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.
CASE= Clinical Assessment Scale in Autoimmune Encephalitis; NMDAR=N-methyl-D-aspartate receptor; Neuropsychological Status. The CASE (Clinical Assessment Scale in AIE) includes an assessment of 9 items: seizure, memory dysfunction, psychiatric symptoms, consciousness, language problems, dyskinesia/dystonia, gait instability and ataxia, brainstem dysfunction, weakness. This overall CASE total score ranges from 0 to 27, with a higher score indicating a greater degree of disability.
The Modified Rodnan Skin Score is a scoring tool to assess skin thickness in 17 cutaneous sites across the body. Each site is rated on a semiquantitative score ranging from 0 (normal) to 3 (severe). The total score is the sum of the individual skin scores and can range from 0 to 51.
CRoff = complete remission while receiving efgartigimod PH20 SC or placebo and being off oral corticosteroid therapy for at least 8 weeks. Complete remission is defined as the absence of new lesions, complete healing of existing lesions and absence of pruritus.
| Arm | Type | Description |
|---|---|---|
| Part A - efgartigimod PH20 SC PFS | EXPERIMENTAL | Participants will receive efgartigimod PH20 SC Via Prefilled Syringe (PFS) during the part A double-blinded treatment period. |
| Part A - placebo PH20 SC PFS | PLACEBO_COMPARATOR | Participants will receive placebo PH20 SC Via Prefilled Syringe (PFS) during the part A double-blinded treatment period. |
| Part B - efgartigimod PH20 SC PFS | EXPERIMENTAL | Participants will receive efgartigimod PH20 SC Via Prefilled Syringe (PFS) during the part B treatment period. |
| Part B - placebo PH20 SC PFS | EXPERIMENTAL | Participants will receive placebo PH20 SC Via Prefilled Syringe (PFS) during the part B treatment period. |
| Part C - efgartigimod PH20 SC PFS | EXPERIMENTAL | Participants will receive efgartigimod PH20 SC PFS in the part C open-label treatment period |
| Double-blinded treatment period: Efgartigimod PH20 SC | EXPERIMENTAL | Participants receiving efgartigimod PH20 SC during the double-blinded treatment period |
| Double-blinded treatment period: Placebo PH20 SC | PLACEBO_COMPARATOR | Participants receiving placebo PH20 SC during the double-blinded treatment period |
| Open-label treatment period | EXPERIMENTAL | Participants receiving efgartigimod PH20 SC during the open-label treatment period |
| Efgartigimod PH20 SC in part A+B | EXPERIMENTAL | Participants receiving efgartigimod PH20 SC during part A and part B |
| Placebo PH20 SC in Part A + Efgartigimod PH20 SC in Part B | EXPERIMENTAL | Participants receiving placebo PH20 SC in Part A and Efgartigimod PH20 SC in Part B |
| Efgartigimod PH20 SC | EXPERIMENTAL | Participants receiving efgartigimod PH20 subcutaneously |
| efgartigimod | EXPERIMENTAL | Patients receiving efgartigimod intravenous (IV) treatment |
| Placebo PH20 SC | PLACEBO_COMPARATOR | Patients receiving placebo PH20 SC treatment |
| placebo | EXPERIMENTAL | patients receiving placebo on top of prednisone |
| Part A (Open-Label Lead-in Period): Efgartigimod PH20 SC | EXPERIMENTAL | All participants will receive efgartigimod PH20 SC open label for 8 weeks |
| Part B (Double-blinded treatment period): Efgartigimod PH20 SC | EXPERIMENTAL | Participants will receive efgartigimod PH20 SC for 24 weeks |
| Part B (Maintenance double-blinded treatment period): Placebo PH20 SC | PLACEBO_COMPARATOR | Participants will receive placebo for 24 weeks |
| Part C (Open-Label Extension Period): Efgartigimod PH20 SC | EXPERIMENTAL | Participants who complete Part B will receive efgartigimod PH20 SC for 24 weeks |
| Name | Type | Description |
|---|---|---|
| Efgartigimod PH20 SC | BIOLOGICAL | Subcutaneous injection of Efgartigimod PH20 Via Prefilled Syringe (PFS) |
| Placebo PH20 SC | OTHER | Subcutaneous injection of placebo PH20 via Prefilled Syringe (PFS) |
| efgartigimod IV | BIOLOGICAL | Intravenous infusion of efgartigimod |
| Placebo | OTHER | Subcutaneous injection of placebo |
| prednisone | DRUG | Oral prednisone tablets |
| Efgartigimod PH20 (ARGX-113) SC | BIOLOGICAL | subcutaneous administrations of efgartigimod PH20 SC given by prefilled syringe (PFS). For participants aged 12 to \<18 years with body weight ≤50 kg, the study drug will be administered by vial and syringe. |
Inclusion Criteria: * Is at least 18 years of age and the local legal age of consent for clinical studies when signing the ICF. * Has a documented diagnosis of GD with TRAb (anti-thyrotropin receptor antibody) levels \>=ULN (upper limit of normal) at screening. * Has active hyperthyroidism due to G...