Evaluating Long-term Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 12

Inclusion Criteria

1. The participant completed ARGX-113-2006, defined as:
a. The participant reached EoT in trial ARGX-113-2006 and agreed to participate in the ARGX-113-2008 trial.
b. The participant qualifies for retreatment in trial ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required IP visits within the ARGX-113-2006 trial's timeframe (ie, between TPnV1 to IPnV4).
2. Either the participant or the participant’s legally acceptable representative (LAR) can understand the requirements of the trial and provide written informed consent/assent (including consent/assent for the use and disclosure of research-related health information), and demonstrates a willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).
3. Contraceptive use by males and females must be consistent with local regulations for those participating in clinical trials and:
a. Male participants agree not to donate sperm from the time the informed consent form (ICF) is signed until the end of the trial.
b. Female adolescents of childbearing potential (FAOCBP; defined in Section 10.4.1.1 of the protocol ) must have a negative urine pregnancy test at trial entry (TP1V1 or IP0V1).
The contraceptive requirements for male participants are described in Section 10.4.2.2. The contraceptive requirements for FAOCBP are described in Section 10.4.2.1.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Participants are excluded from the trial if any of the following criteria apply:
1. FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of investigational medicinal product (IMP)
2. Discontinued early from ARGX-113-2006 treatment due to: pregnancy, receiving prohibited medication, participating in another trial with an investigational product, or the occurrence of a life-threatening or an investigational medicinal product-related AE, as assessed by the investigator
3. A known hypersensitivity reaction to efgartigimod or any of its excipients
4. Received a live-attenuated vaccine fewer than 4 weeks before trial entry.
5. Any of the following medical conditions:
a. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at trial entry
b. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of gMG or put the participant at undue risk

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the safety and tolerability of efgartigimod IV;Secondary Objective: To evaluate the immunogenicity of efgartigimod IV ;Primary end point(s): - Incidence and severity of adverse events (AEs)<br>- Incidence and severity of serious adverse events (SAEs)<br>- Incidence and severity of adverse events of special interest (AESIs)<br>- Changes in laboratory test results, vital signs, height and weight, and<br>electrocardiogram (ECG) results;Timepoint(s) of evaluation of this end point: Once a month

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Incidence and prevalence of antidrug antibodies (ADAs) against<br>efgartigimod ;Timepoint(s) of evaluation of this end point: No specific timepoints, during the entire study<br>

Updated 11/20/2023

Evaluating Long-term Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis

Recruitment & Eligibility

Study & Design

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