A Long-Term, Single-Arm, Open-label, Multicenter Phase 3 Study to Evaluate the Safety and Tolerability of Multiple Subcutaneous Injections of Efgartigimod PH20 SC in Patients With Generalized Myasthenia Gravis
- Conditions
- 1000381610029317Disease of the neuro muscular junctionSerious muscle weakness
- Registration Number
- NL-OMON54450
- Lead Sponsor
- PPD
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 2
Participants are eligible to be included in the study only if all of the
following criteria apply:
1. 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
2. Previously participated in antecedent studies ARGX-113-2001 or ARGX-113-1705
and are eligible for roll over
3. Contraceptive use by men and women should be consistent with local
regulations regarding the methods of contraception for those participating in
clinical studies and:
a. Male participants: No male contraception is required
b. Female participants:
* Women of Child bearing potential (WOCBP) must have a negative urine pregnancy
test at baseline before IMP can be administered.
Participants are excluded from the study if any of the following criteria apply:
1. 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.
2. Are pregnant or lactating, or intend to become pregnant during the study or
within 90 days after the last dose of IMP
3. Has any of the following medical conditions:
a. Clinically significant uncontrolled chronic bacterial, viral, or fungal
infection at rollover
b. 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
c. History of malignancy unless deemed cured by adequate treatment with no
evidence of reoccurrence for >=3 years before the first administration of 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)
d. 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
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>• Incidence and severity of adverse events (AEs), incidence of serious adverse<br /><br>events (SAEs), and changes in laboratory test results, physical examination<br /><br>results, vital signs, and electrocardiogram results</p><br>
- Secondary Outcome Measures
Name Time Method <p>• Myasthenia Gravis Activities of Daily Living (MG-ADL) total score changes<br /><br>from baseline and cycle baseline over time by cycle<br /><br>• Percentage reduction in levels of total immunoglobulin G (IgG) from baseline<br /><br>and cycle baseline over time by cycle<br /><br>• Percentage reduction of acetylcholine receptor binding autoantibodies<br /><br>(AChR-Ab) from baseline and cycle baseline over time by cycle in AChR-Ab<br /><br>seropositive participants<br /><br>• Incidence and prevalence of anti-drug antibodies (ADAs) to efgartigimod over<br /><br>time</p><br>