Phase 3
N=110
Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Bottom Line
View on ClinicalTrials.gov: NCT04735432 ↗Enrolled (actual)
110
Serious AEs
10.9%
Results posted
Feb 2023
Primary outcome: Primary: Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set) — -66.4; -62.2 percent — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- efgartigimod PH20 SC (Biological); efgartigimod IV (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- argenx
- Primary completion
- Nov 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Total IgG Levels at Day 29 (mITT Analysis Set) |
-66.4; -62.2 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline in Total IgG Levels Over Time (mITT Analysis Set) |
-40.1; -39.6; -56.9; -55.1; -62.2; -59 | — |
| SECONDARY Percent Change From Baseline in AChR-Ab Levels Over Time in AChR- Ab Positive Patients (mITT Analysis Set) |
-42.5; -43.7; -57.4; -55.1; -61.8; -59.2 | — |
| SECONDARY Percent Change From Baseline in IgG Subtype Levels Over Time (mITT Analysis Set) |
-71; -68.4; -65.6; -64.5; -69.6; -64.7 | — |
| SECONDARY AUEC of the Percent Change From Baseline in Total IgG Level (mITT Analysis Set) |
-138.9; -139.1; -341.9; -328.3; -416.0; -399.8 | — |
| SECONDARY Еfgartigimod IV and PH20 SC Serum Pharmacokinetic Parameter Ctrough |
18.3; 16.4; 21.4; 14.0; 22.5; 15.2 | — |
| SECONDARY Efgartigimod IV Serum Pharmacokinetic Parameter Cmax |
199; 215; 211; 206 | — |
| SECONDARY Incidence of ADA Against Efgartigimod (Safety Analysis Set) |
34.5; 20.0 | — |
| SECONDARY Incidence of Antibodies Against rHuPH20 in the SC Treatment Arm (Safety Analysis Set) |
5.5 | — |
| SECONDARY Incidence and Severity of AEs and SAEs (Safety Analysis Set) |
67.3; 50.9; 14.5; 7.3; 16.4; 7.3 | — |
| SECONDARY MG-ADL Responders (ITT Analysis Set) |
69.1; 69.1 | — |
| SECONDARY QMG Responders (ITT Analysis Set) |
65.5; 51.9 | — |
| SECONDARY Change From Baseline in MG-ADL Total Score Over Time (ITT Analysis Set) |
-2.2; -2.0; -3.6; -3.2; -4.7; -4.3 | — |
| SECONDARY Change From Baseline in QMG Score Over Time (ITT Analysis Set) |
-3; -2; -4.3; -3.4; -5.7; -4.5 | — |
Summary
The purpose of this study is to investigate the Pharmacodynamics (PD), Pharmacokinetics (PK), safety, tolerability, immunogenicity, and clinical efficacy of efgartigimod coformulated with recombinant human hyaluronidase PH20 (rHuPH20) as compared to efgartigimod IV infused in patients with generalized myasthenia gravis (gMG). The study duration is approximately 12 weeks. After screening, patients will be randomized to receive either efgartigimod infusions or efgartigimod PH20 subcutaneously (SC)
Eligibility Criteria
Inclusion Criteria
Bullet list of each inclusion criterium:
- Must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- At least 18 years of age at the time of signing the informed consent form.
- Diagnosed with generalized Myasthenia Gravis (gMG) with confirmed documentation and supported by at least 1 of the following:
- History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation
- History of positive edrophonium chloride test
- Demonstrated improvement in Myasthenia Gravis (MG) signs upon treatment with oral acetylcholinesterase (AChE) inhibitors as assessed by the treating physician
- Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, IVa, or IVb
Exclusion Criteria
Bullet list of each exclusion criterium:
- Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of Investigational Medicinal Product.
- Has any of the following medical conditions:
- Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
- Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.
- History of malignancy unless deemed cured by adequate treatment with no evidence of reoccurrence for ≥3 years before the first administration of the IMP. Participants with the following cancers can be included at any time:
- adequately treated basal cell or squamous cell skin cancer
- carcinoma in situ of the cervix
- carcinoma in situ of the breast
- incidental histological findings of prostate cancer (TNM Classification of Malignant Tumors stage T1a or T1b).
- Clinical evidence of other significant serious diseases, or the participant has had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the study or put the participant at undue risk.
Data sourced from ClinicalTrials.gov (NCT04735432). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.