Clinical trial NCT05374590: Evaluating Long-term Safety of Efgartigimod Administered Intravenously and Efgartigimod PH20 Administered Subcutaneously in Children with Generalized Myasthenia Gravis
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
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE2
PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2022-05-16
Enrollment: 12
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

Participants are eligible to be included in the trial only if all of the following criteria apply:

1. The participant completed ARGX-113-2006 or ARGX-113-2207, 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.
2. 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.
3. Contraceptive use for sexually active participants should be consistent with local regulations for those participating in clinical studies. A participant is of childbearing potential if, in the opinion of the investigator, he/she is biologically capable of having children.

   1. Contraceptive requirements for male participants are presented in Section 10.4.2.2.
   2. Female adolescents of childbearing potential (FAOCBP) must have a negative urine pregnancy test at trial entry (TP1V1 or IP0V1)

Exclusion Criteria:

Participants are excluded from the trial if any of the following criteria apply:

1. Female adolescents of childbearing potential (FAOCBP): Pregnancy or lactation, or the participant intends to become pregnant during their participation in the study.
2. Discontinued early from ARGX-113-2006 or ARGX-113-2207 treatment.
3. A known hypersensitivity reaction to efgartigimod or any of its excipients.
4. Any of the following medical conditions:

   1. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at study entry not sufficiently resolved in the investigator's opinion.
   2. Known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalized myasthenia gravis (gMG) or put the participant at undue risk

Primary outcomes:
measure: Incidence of adverse events (AEs)
timeFrame: up to 4 years
measure: Severity of adverse events (AEs)
timeFrame: up to 4 years
measure: Incidence of serious adverse events (SAEs)
timeFrame: up to 4 years
measure: Severity of serious adverse events (SAEs)
timeFrame: up to 4 years
measure: Incidence of adverse events of special interest (AESIs)
timeFrame: up to 4 years
measure: Severity of adverse events of special interest (AESIs)
timeFrame: up to 4 years
measure: Changes in height from baseline
timeFrame: up to 4 years
measure: Changes in weight from baseline
timeFrame: up to 4 years
measure: Electrocardiogram - heart rate
timeFrame: up to 4 years
measure: Electrocardiogram - QTcF (ms)
timeFrame: up to 4 years
Secondary outcomes:
measure: Incidence of antidrug antibodies (ADAs) against efgartigimod
timeFrame: up to 4 years
measure: Prevalence of antidrug antibodies (ADAs) against efgartigimod
timeFrame: up to 4 years
measure: Incidence of antibodies against recombinant human hyaluronidase PH20 (rHuPH20)
timeFrame: Up to 4 years
measure: Prevalence of antibodies against recombinant human hyaluronidase PH20 (rHuPH20)
timeFrame: Up to 4 years
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ann and Robert H Lurie Children's Hospital of Chicago - Main Hospital
state: Illinois
city: Charlottesville
country: United States
facility: University of Virginia (UVA) Health - Developmental Pediatrics Clinic
state: Virginia
city: Edegem
country: Belgium
facility: Universitair Ziekenhuis Antwerpen
city: Marseille
country: France
facility: AP-HM- Hôpital de La Timone
city: Leiden
country: Netherlands
facility: Leids Universitair Medisch Centrum
city: Gdańsk
country: Poland
facility: Uniwersyteckie Centrum Kliniczne w Gdansku
city: Katowice
country: Poland
facility: Wielospecjalistyczna Poradnia Lekarska Synapsis
city: Warszawa
country: Poland
facility: Centralny Szpital Kliniczny - Uniwersyteckie Centrum Kliniczne WUM
city: London
country: United Kingdom
facility: Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital - Pediatric Neurology
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals NHS Foundation Trust - John Radcliffe Hospital Children's Hospital
Study officials:
References: