A Phase 3b, Randomized, Open-label, Parallel-Group Study to Evaluate Different Dosing Regimens of Intravenous Efgartigimod to Maximize and Maintain Clinical Benefit in Patients With Generalized Myasthenia Gravis (gMG)
- Conditions
- Generalized Myasthenia Gravis (gMG)MedDRA version: 20.0Level: HLTClassification code 10071942Term: Myasthenia gravis and related conditionsSystem Organ Class: 100000004859Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2021-002504-12-IT
- Lead Sponsor
- ARGENX BV
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 72
1. Capable of giving signed informed consent as described in Section
10.1.3 of the protocol, which includes compliance with the requirements
and restrictions listed in the informed consent form (ICF) and in this
protocol
2. At least 18 years of age, at the time of signing the informed consent
3. Diagnosed with gMG with confirmed documentation and supported by
a physical exam and confirmed seropositivity for AChR-Abs
4. Meets the clinical criteria as defined by the Myasthenia Gravis
Foundation of America (MFGA) class II, III, or IV
5. Has an MG-ADL total score =5 at screening and the day 1 visit, with
more than 50% of the score due to nonocular symptoms
6. Concomitant gMG treatment is permitted. Permitted concomitant gMG
treatment includes nonsteroidal immunosuppressive drugs (NSIDs),
steroids, and/or acetylcholinesterase (AChE) inhibitors. If receiving
corticosteroids and/or NSIDs, must be on a stable dose for at least 1
month before screening.
7. 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:
- Male participants are not allowed to donate sperm from signing the
ICF until the end of the study.
b. Female participants:
- WOCBP (defined in Section 10.4.1 of protocol) must have a negative
serum pregnancy test at screening and a negative urine pregnancy test
at baseline before IMP can be administered.
- The contraception requirements for WOCBP are described in Section
10.4.2.1 of the protocol.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 43
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 29
1. Clinically significant uncontrolled active or chronic bacterial, viral, or
fungal infection at screening
2. A positive test for SARS-CoV-2 at screening
3. Any other known autoimmune disease that, in the opinion of the
investigator, would interfere with an accurate assessment of the clinical
symptoms of gMG and/or put the participant at undue risk
4. 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, provided they are adequately treated at screening:
a. Basal cell or squamous cell skin cancer
b. Carcinoma in situ of the cervix
c. Carcinoma in situ of the breast
d. Incidental histological finding of prostate cancer (TNM stage T1a or
T1b)
5. Clinical evidence of other significant serious diseases, a recent (<3
months) major surgery, or any other condition that, in the opinion of the
investigator, could confound the results of the study or put the
participant at undue risk
6. A thymectomy within 3 months of screening
7. Pregnant or lactating females and those who intend to become
pregnant during the study or within 90 days of the last dose of IMP
8. Use of the following prior or concomitant therapies:
a. IVIg or subcutaneous (SC)Ig within 14 days of day 1
b. Rituximab within 6 months of day 1
c. Eculizumab within 1 month of day 1
d. Other monoclonal antibodies (eg, adalimumab, tocilizumab,
ixekizumab) within 5 half-lives of the monoclonal antibodies before day
1
e. Use of any other investigational product within 3 months or 5 halflives,
whichever is longer, before day 1
f. Receipt of a live or live-attenuated vaccine within 4 weeks of
screening. The receipt of any inactivated, subunit, polysaccharide,
conjugate vaccine at any time before screening is not considered
exclusionary.
9. Previous participation in a clinical study or patient access program
during which they were treated with efgartigimod
10. Positive serum test at screening for an active viral infection with any
of the following conditions:
a. Hepatitis B virus (HBV) that is indictive of an acute or chronic
infection
b. Hepatitis C virus (HCV) based on HCV antibody assay (unless
associated with a negative HCV RNA test)
c. HIV based on test results that are associated with an AIDS-defining
condition or a CD4 count =200 cells/mm3
11. Total IgG <6 g/L at screening
12. Known hypersensitivity reaction to efgartigimod or any of its excipients
13. The participant stands in any relationship of dependency with the
sponsor.
14. The participant has been institutionalized due to an official or judicial
order.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the clinical efficacy of efgartigimod IV 10 mg/kg administered<br>in a q2w continuous regimen compared to that administered in a cyclic<br>regimen;Primary end point(s): Mean of the average MG-ADL total score change from baseline during the<br>visit of week (W)1 through W21 by regimen arm;Timepoint(s) of evaluation of this end point: 21 weeks;Secondary Objective: To evaluate the safety and tolerability of both treatment regimens<br>used throughout the study<br>• To assess the clinical efficacy of efgartigimod IV in both treatment<br>regimens over time<br>• To compare the number of participants who achieve maximal clinical<br>effect during different treatment regimens
- Secondary Outcome Measures
Name Time Method