- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04777734
Efgartigimod Expanded Access for Generalized Myasthenia Gravis
An Expanded Access Program for Efgartigimod Treatment in Patients With Generalized Myasthenia Gravis
This expanded access protocol applies to patients with gMG who are not enrolled in an ongoing clinical trial. The aim of the trial is to provide patients with generalized myasthenia gravis (gMG), who are ineligible to participate in a clinical trial, access to efgartigimod treatment before regulatory approval.
There are country-specific protocols and also individual use EAP. Recruitment for the treatment protocol in US is now closed (ARGX-113-EAP-2101).
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Expanded Access Type
- Treatment IND/Protocol
Contacts and Locations
Study Locations
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Illinois
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Chicago, Illinois, United States, 60611
- Investigator site 12 - Approved for Marketing
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Kentucky
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Lexington, Kentucky, United States, 40506
- Investigator site 16 - Approved for Marketing
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Michigan
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Ann Arbor, Michigan, United States, 41809
- Investigator site 1 - Approved for Marketing
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New York
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New Hyde Park, New York, United States, 11042
- Investigator Site 6 - Approved for Marketing
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Pennsylvania
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Philadelphia, Pennsylvania, United States, 19104
- Investigator site 14 - Approved for Marketing
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Virginia
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Henrico, Virginia, United States, 23233
- Investigator site 2 - Approved for Marketing
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
- Patient is ≥18 years of age, at the time of signing the informed consent
- Patient has a diagnosis of MG (AChR-Ab seropositive or seronegative) with generalized muscle weakness
- Patient has been vaccinated against COVID-19 or has had a negative COVID-19 test result in the 2 weeks before enrollment
- Patient has documented IgG >6 g/L within one month of screening
Patient agrees to contraceptive use consistent with local regulations and scientific rationale regarding the methods of contraception and:
- Male patients: must agree to use an acceptable method of contraception and to not donate sperm from the time of providing informed consent until the end of the program
- Female patients: Women of childbearing potential must use a highly effective or acceptable method of contraception and have a negative serum pregnancy test at screening
- Patient provides signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and this protocol
- Patients with a history of hepatitis B, hepatitis C, or HIV must have a documented negative test for an active viral infection.
Exclusion Criteria:
- Patient has clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening
- Patient has a known autoimmune disease that, in the opinion of the treating physician, would interfere with an accurate assessment of clinical symptoms of gMG or put the patient at undue risk
- Patient has a history of malignancy unless it is deemed to be cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of the IMP. Patients with documentation of adequate treatment of the following cancers can be included at any time: basal cell or squamous cell skin cancer; carcinoma in situ of the cervix; carcinoma in situ of the breast; incidental histological finding of prostate cancer (TNM stage T1a or T1b)
- Patient has clinical evidence of other significant serious diseases, has recently had major surgery, or has any other condition that, in the opinion of the treating physician, could put the patient at undue risk
- Patient may be excluded based upon review of clinical medical records and screening clinical safety laboratory test results
- Patient has received a live or a live-attenuated vaccination during the month before screening
- Patient is pregnant and/or lactating or intends to become pregnant during the program or within 90 days after the last dose
- Patient is an unsterilized male who is sexually active while participating in the program and does not intend to use effective methods of contraception during the program through 90 days after the last dose or plans to donate sperm during the program or through 90 days after the last dose.
- The patient has previously received rituximab, and the last rituximab dose was received less than 6 months before the first dose of efgartigimod
Study Plan
How is the study designed?
Collaborators and Investigators
Sponsor
Study record dates
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Immune System Diseases
- Neoplasms
- Autoimmune Diseases of the Nervous System
- Autoimmune Diseases
- Neoplasms by Site
- Neurologic Manifestations
- Musculoskeletal Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Neuromuscular Manifestations
- Nervous System Neoplasms
- Paraneoplastic Syndromes, Nervous System
- Paraneoplastic Syndromes
- Neuromuscular Junction Diseases
- Muscle Weakness
- Myasthenia Gravis
Other Study ID Numbers
- ARGX-113-EAP-2101
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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Assiut UniversityRecruitingNervous System Diseases | Autoimmune Diseases of the Nervous System | Thymoma | Myasthenia Gravis | Neuromuscular Junction Diseases | Myasthenia Gravis, Generalized | Myasthenia Gravis Crisis | Myasthenia Gravis, Ocular | Myasthenia Gravis, Juvenile Form | Thymus Hyperplasia | Myasthenia Gravis With Exacerbation... and other conditionsEgypt
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Alexion Pharmaceuticals, Inc.CompletedMyasthenia Gravis | Myasthenia Gravis, Generalized | Myasthenia Gravis, Juvenile FormUnited States, Japan, Netherlands
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Catalyst Pharmaceuticals, Inc.CompletedMyasthenia Gravis, GeneralizedUnited States
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COUR Pharmaceutical Development Company, Inc.Not yet recruitingMyasthenia Gravis | Generalized Myasthenia | AChR Myasthenia Gravis | MuSK MGUnited States
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Cabaletta BioNot yet recruitingGeneralized Myasthenia Gravis (gMG)
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argenxRecruiting
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Kyverna TherapeuticsNot yet recruitingGeneralized Myasthenia Gravis | Myasthenia Gravis
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UCB Biopharma SRLNot yet recruitingGeneralized Myasthenia Gravis
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Tang-Du HospitalRecruitingMyasthenia Gravis, GeneralizedChina
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Alexion Pharmaceuticals, Inc.RecruitingGeneralized Myasthenia Gravis | gMGSpain, Italy, United States, France, Switzerland, Serbia, Japan, Netherlands
Clinical Trials on efgartigimod
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argenxCompleted
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argenxRecruitingGeneralized Myasthenia GravisUnited States, Netherlands, Belgium, Poland, United Kingdom
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argenxCompletedGeneralized Myasthenia GravisSpain, United States, Belgium, Georgia, Germany, Hungary, Italy, Japan, Netherlands, Poland, Russian Federation
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argenxNot yet recruiting
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argenxIqvia Pty LtdRecruitingPrimary Sjögren's SyndromeBelgium, Hungary, Poland
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argenxIqvia Pty LtdRecruitingPost-COVID Postural Orthostatic Tachycardia Syndrome Postural Orthostatic Tachycardia SyndromeUnited States
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argenxNot yet recruiting
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argenxRecruitingGeneralized Myasthenia GravisUnited Kingdom, Spain, United States, Austria, Germany, Belgium, Italy, Netherlands, France, Georgia, Poland, Canada
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argenxIqvia Pty LtdActive, not recruitingPostural Orthostatic Tachycardia SyndromeUnited States
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Shanghai Zhongshan HospitalNot yet recruitingMyasthenia Gravis