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

Generalized Myasthenia Gravis Clinical Trial

— ADAPT Jr +  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05374590
• Other study ID #: ARGX-113-2008
• Status: Recruiting
• Phase: Phase 2/Phase 3
• Start date: August 18, 2022
• Est. completion date: September 2028

Study information

• Verified date: June 2024
• Source: argenx
• Contact: Sabine Coppieters, MD
• Phone: 857-350-4834
• Email: ClinicalTrials[@]argenx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: September 2028
• Est. primary completion date: September 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

Biological:
• Efgartigimod IV or Efgartigimod PH20 SC
Intravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC

Locations

Country	Name	City	State
Belgium	UZ Antwerpen	Antwerp
Netherlands	Leiden University Medical Center	Leiden
Poland	Wielospecjalistyczna Poradnia Lekarska Synapsis	Katowice
Poland	Uniwersyteckie Centrum Kliniczne WUM, Centralny Szpital Kliniczny	Warszawa
United Kingdom	Great Ormand Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital - Pediatric Neurology	London
United Kingdom	Oxford University hospitals NHS Foundation Trust-Oxford Children's Hospital	Oxford
United States	University of Virginia	Charlottesville	Virginia
United States	Ann and Robert H. Lurie Childrens Hospital of Chicago	Chicago	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Countries where clinical trial is conducted

United States,  Belgium,  Netherlands,  Poland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of adverse events (AEs)		up to 4 years
Primary	Severity of adverse events (AEs)		up to 4 years
Primary	Incidence of serious adverse events (SAEs)		up to 4 years
Primary	Severity of serious adverse events (SAEs)		up to 4 years
Primary	Incidence of adverse events of special interest (AESIs)		up to 4 years
Primary	Severity of adverse events of special interest (AESIs)		up to 4 years
Primary	Changes in height from baseline		up to 4 years
Primary	Changes in weight from baseline		up to 4 years
Primary	Electrocardiogram - heart rate		up to 4 years
Primary	Electrocardiogram - QTcF (ms)		up to 4 years
Secondary	Incidence of antidrug antibodies (ADAs) against efgartigimod		up to 4 years
Secondary	Prevalence of antidrug antibodies (ADAs) against efgartigimod		up to 4 years
Secondary	Incidence of antibodies against recombinant human hyaluronidase PH20 (rHuPH20)		Up to 4 years
Secondary	Prevalence of antibodies against recombinant human hyaluronidase PH20 (rHuPH20)		Up to 4 years