Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis

NCT ID: NCT05374590

Last Updated: 2025-12-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Clinical Phase

PHASE2/PHASE3

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-08-18

Study Completion Date

2029-09-30

Brief Summary

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The purpose of this trial is to evaluate the long-term safety of efgartigimod IV and efgartigimod PH20 SC administered to participants with gMG in the antecedent studies, ARGX-113-2006 and ARGX-113-2207, respectively.

Participants will receive efgartigimod IV or efgartigimod PH20 SC, using the dose administered in the antecedent studies. Participants who have not reached the age of 18 can remain in the study until efgartigimod becomes commercially available in the respective country or available through another continued access program for gMG. Participants who have reached the age of 18 can remain in the study for a maximum of 2 years, until efgartigimod becomes commercially available in the respective country or available through another continued access program for gMG, whichever comes first.

Detailed Description

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Conditions

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Generalized Myasthenia Gravis gMG

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Efgartigimod or Efgartigimod PH20 SC

Patients receiving Efgartigimod IV treatment or Efgartigimod PH 20 SC treatment

Group Type EXPERIMENTAL

Efgartigimod IV or Efgartigimod PH20 SC

Intervention Type BIOLOGICAL

Intravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC

Interventions

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Efgartigimod IV or Efgartigimod PH20 SC

Intravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

* The participant completed ARGX-113-2006, defined as:

1. The participant reached End of Trial in trial ARGX-113-2006 or End of Study in ARGX-113-2207 and agreed to participate in the ARGX-113-2008 trial.
2. The participant qualifies for retreatment in trial ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required Intertreatment Period (IP) visits within the ARGX-113-2006 trial's timeframe.
* Either the participant or the participant's legally authorized representative can understand the requirements of the trial and provide written informed consent/assent, and willingness and ability to comply with the trial protocol procedures.
* Contraceptive use for sexually active participants should be consistent with local regulations for those participating in clinical studies.

Exclusion Criteria

* Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, or the participant intends to become pregnant during their participation in the study.
* Discontinued early from ARGX-113-2006 or ARGX-113-2207 treatment.
* Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at study entry not sufficiently resolved in the investigator's opinion or known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of gMG or put the participant at undue risk.
Minimum Eligible Age

2 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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argenx

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital

Chicago, Illinois, United States

Site Status

University of Virginia (UVA) Health - Developmental Pediatrics Clinic

Charlottesville, Virginia, United States

Site Status

Universitair Ziekenhuis Antwerpen

Edegem, , Belgium

Site Status

UZ Gent

Ghent, , Belgium

Site Status

Childrens Hospital of Eastern Ontario

Ottawa, , Canada

Site Status

AP-HM- Hôpital de La Timone

Marseille, , France

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Hadassah Medical Center- Ein Kerem

Jerusalem, , Israel

Site Status

Leids Universitair Medisch Centrum

Leiden, , Netherlands

Site Status

Uniwersyteckie Centrum Kliniczne w Gdansku

Gdansk, , Poland

Site Status

Wielospecjalistyczna Poradnia Lekarska Synapsis

Katowice, , Poland

Site Status

Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM

Warsaw, , Poland

Site Status

Hospital Universitari i Politecnic La Fe de Valencia

Valencia, , Spain

Site Status

Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital - Pediatric Neurology

London, , United Kingdom

Site Status

Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital

Oxford, , United Kingdom

Site Status

Countries

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United States Belgium Canada France Germany Israel Netherlands Poland Spain United Kingdom

Other Identifiers

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2023-507379-23-00

Identifier Type: CTIS

Identifier Source: secondary_id

ARGX-113-2008

Identifier Type: -

Identifier Source: org_study_id