Phase 3
N=180
Evaluating the Long-Term Safety and Tolerability of Efgartigimod PH20 SC Administered Subcutaneously in Patients With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Bottom Line
View on ClinicalTrials.gov: NCT04818671 ↗Enrolled (actual)
180
Serious AEs
30.6%
Results posted
Jan 2026
Primary outcome: Primary: Number of AEs, SAEs and AESIs — 3325; 107; 420 events
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- efgartigimod PH20 SC (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- argenx
- Primary completion
- Dec 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of AEs, SAEs and AESIs |
3325; 107; 420 | — |
| SECONDARY MG-ADL Total Score Changes From Baseline |
-2.2; -3.4; -3.9; -4.0 | — |
| SECONDARY Percent Change in Total IgG Levels From Baseline |
-62.6 | — |
| SECONDARY Percent Change in AChR-Ab From Baseline in AChR-Ab Seropositive Participants |
-57.6 | — |
| SECONDARY Efgartigimod Serum Concentrations |
21693 | — |
| SECONDARY Incidence of ADAs Against Efgartigimod Over Time |
35 | — |
| SECONDARY Incidence of NAbs Against Efgartigimod Over Time |
11 | — |
| SECONDARY Incidence of Antibodies Against rHuPH20 Over Time |
49 | — |
| SECONDARY Incidence of NAbs Against rHuPH20 Over Time |
1 | — |
| SECONDARY Changes in Total MG-QoL15r From Baseline |
-2.5; -3.7; -4.5; -4.7 | — |
| SECONDARY Changes in EQ-5D-5L VAS Score From Baseline |
13.9 | — |
| SECONDARY Percent of Participants or Caregivers by Number of Training Visits Needed to be Competent to Start Self- or Caregiver-Supported Administration |
4.8; 45.8; 13.9; 11.4; 12.7; 1.8 | — |
| SECONDARY Percent of Self- or Caregiver-supported Study Drug Administration Among All Study Treatment Visits at Home |
49.2 | — |
Summary
The purpose of this study is to evaluate the long-term safety and tolerability of efgartigimod PH20 SC 1000 mg, and the clinical efficacy, PD, pharmacokinetics (PK), immunogenicity, impact on the quality of life (QoL) of the participants, treatment satisfaction, and administration method preference, and the feasibility of self- and caregiver-supported administration of the SC injection.
Treatment duration: 3-week treatment periods, repeated as needed with at least 28 days in between treatment periods
Health measurements: total levels of immunoglobulin G (IgG), Acetylcholine receptor binding autoantibodies (AChR-Ab) levels, Myasthenia Gravis Activities of Daly Living (MG-ADL).
Eligibility Criteria
Inclusion Criteria
- 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.
- Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705 and are eligible for roll over.
- Contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies and:
- Women of Child bearing potential (WOCBP) must have a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered.
Exclusion Criteria
- The participant was discontinued early from studies ARGX-113-2001 or ARGX-113-1705, unless the reason for discontinuation from study ARGX-113-1705 was to roll over into study ARGX-113-2002.
a. Participants who, in the investigator's judgment, are not benefiting from efgartigimod IV in study ARGX-113-1705 Part B are not eligible for roll over into ARGX-113-2002.
- Are pregnant or lactating, or intend to become pregnant during the study or within 90 days after the last dose of investigational medicinal product (IMP)
- Has any of the following medical conditions:
- Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at roll-over
- Any other known autoimmune disease that, in the opinion of the investigator, would interfere with 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 investigational medicinal product (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
- Received a live-attenuated vaccine within 28 days prior to study entry or plan to receive a live-attenuated vaccine during the study
- A known hypersensitivity reaction to efgartigimod, rHuPH20, or any of its excipients
Data sourced from ClinicalTrials.gov (NCT04818671). 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.