Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Efgartigimod IV or Efgartigimod PH20 SC.
- Who it may be relevant to
- Registry conditions: Generalized Myasthenia Gravis, gMG. Basic parameters: 2 years — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Belgium, Canada, France, Germany +5
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Long-term, Single-Arm, Open-label, Multicenter Trial to Evaluate Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children With Generalized Myasthenia Gravis
Overview
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.
Interventions
- Biological Efgartigimod IV or Efgartigimod PH20 SC
Intravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC
Primary outcome measures
- Incidence of AEs, SAEs and AESIs [Time frame: Up to 4 years]
Secondary outcome measures (2)
- Incidence of ADAs against efgartigimod [Time frame: Up to 4 years]
- Incidence of antibodies against rHuPH20 [Time frame: Up to 4 years]
Eligibility criteria
Inclusion criteria
- The participant completed ARGX-113-2006, defined as:
- 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.
- 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 3 centers
- Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital — Chicago
- Atrium Health Neurology Specialty Care — Charlotte
- University of Virginia (UVA) Health - Developmental Pediatrics Clinic — Charlottesville
Poland · 3 centers
- Uniwersyteckie Centrum Kliniczne w Gdansku — Gdansk
- Wielospecjalistyczna Poradnia Lekarska Synapsis — Katowice
- Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM — Warsaw
Belgium · 2 centers
- Universitair Ziekenhuis Antwerpen — Edegem
- UZ Gent — Ghent
United Kingdom · 2 centers
- Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospi — London
- Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hosp — Oxford
Canada · 1 center
- Childrens Hospital of Eastern Ontario — Ottawa
France · 1 center
- AP-HM- Hôpital de La Timone — Marseille
Germany · 1 center
- Universitätsklinikum Essen — Essen
Israel · 1 center
- Hadassah Medical Center- Ein Kerem — Jerusalem
Netherlands · 1 center
- Leids Universitair Medisch Centrum — Leiden
Spain · 1 center
- Hospital Universitari i Politecnic La Fe de Valencia — Valencia
Identifiers
NCT: NCT05374590 · ARGX-113-2008 · 2023-507379-23-00