Clinical trial

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

Name

ARGX-113-2008

Description

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.

Trial arms

Trial start

2022-08-18

Estimated PCD

2028-09-01

Trial end

2028-09-01

Status

Recruiting

Phase

Early phase I

Treatment

Efgartigimod IV or Efgartigimod PH20 SC

Intravenous infusion of Efgartigimod or Subcutaneous injection of Efgartigimod PH20 SC

Arms:

Efgartigimod or Efgartigimod PH20 SC

Size

Primary endpoint

Incidence of adverse events (AEs)

up to 4 years

Severity of adverse events (AEs)

up to 4 years

Incidence of serious adverse events (SAEs)

up to 4 years

Severity of serious adverse events (SAEs)

up to 4 years

Incidence of adverse events of special interest (AESIs)

up to 4 years

Severity of adverse events of special interest (AESIs)

up to 4 years

Changes in height from baseline

up to 4 years

Changes in weight from baseline

up to 4 years

Electrocardiogram - heart rate

up to 4 years

Electrocardiogram - QTcF (ms)

up to 4 years

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2', 'PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 12, 'type': 'ESTIMATED'}}

Updated at

2024-04-11

1 organization

1 product

1 indication

Organization

Argenx

Product

Efgartigimod

Indication

Myasthenia Gravis