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Efgartigimod Expanded Access for Generalized Myasthenia Gravis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04777734
Expanded Access Status : Approved for marketing
First Posted : March 2, 2021
Last Update Posted : April 29, 2022
Sponsor:
Information provided by (Responsible Party):
argenx

Brief Summary:

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).


Condition or disease Intervention/treatment
Generalized Myasthenia Gravis Biological: efgartigimod

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Study Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: An Expanded Access Program for Efgartigimod Treatment in Patients With Generalized Myasthenia Gravis



Intervention Details:
  • Biological: efgartigimod
    an intravenous infusion of efgartigimod
    Other Name: ARGX-113

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

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:

    1. 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
    2. 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04777734


Locations
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United States, Illinois
Investigator site 12 - Approved for Marketing
Chicago, Illinois, United States, 60611
United States, Kentucky
Investigator site 16 - Approved for Marketing
Lexington, Kentucky, United States, 40506
United States, Michigan
Investigator site 1 - Approved for Marketing
Ann Arbor, Michigan, United States, 41809
United States, New York
Investigator Site 6 - Approved for Marketing
New Hyde Park, New York, United States, 11042
United States, Pennsylvania
Investigator site 14 - Approved for Marketing
Philadelphia, Pennsylvania, United States, 19104
United States, Virginia
Investigator site 2 - Approved for Marketing
Henrico, Virginia, United States, 23233
Sponsors and Collaborators
argenx
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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT04777734    
Other Study ID Numbers: ARGX-113-EAP-2101
First Posted: March 2, 2021    Key Record Dates
Last Update Posted: April 29, 2022
Last Verified: April 2022
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases